181 RNA Therapies Companies - Worldwide

4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine.

aceRNA Technologies Co., Ltd. is a biotech startup established in 2018 based on RNA design technology developed in synthetic RNA biology research by Prof. Hirohide Saito at Kyoto University. We are developing "smart gene therapy” (mRNA-based and virus-base) that enables control of therapeutic transgene expression in cell-type and -state specific manner. By that, we are committed to develop new therapeutic approach, such as in vivo cell programming.

ADARx Pharmaceuticals is a newly launched next-wave genetic medicine company focusing on oligonucleotide therapeutics for inhibition, degradation and base editing of mRNA transcripts. With RNA as the target of our drug discovery, we are capable of working on a wide range of diseases and are currently focusing on genetic, cardiometabolic, and central nervous system (CNS) diseases. We are driven by our mission to turn cutting-edge science into life-saving therapeutics and our desire to provide hope for patients with intractable disease.

Ainos Inc. is a diversified healthcare company focused on the development of novel point-of-care testing (POCT), low-dose VELDONA interferon therapeutics, women's health, pneumonia, Ainos Pen, AI Nose, and other medical technologies. The company was founded in 1984 and is engaged in developing innovative medical technologies for point-of-care testing and safe and effective treatments for various health conditions.

AIRNA, a biotech company pioneering RNA editing therapeutics to restore the health of patients with rare and common diseases. AIRNA’s experienced team is aiming to advance a pipeline of RNA editing therapeutics driven by its powerful and flexible RNA editing platform, RESTORE+.

Alida Biosciences is an early-stage, VC backed startup in San Diego focused on developing novel genomic analysis tools. The company’s technology will advance the understanding of RNA biology and provide actionable information for improving human health.

Our strategy is to develop pharmacologically optimized drug candidates, which are first assessed in healthy volunteers and patients as monotherapy prior to their use in combination regimens designed to achieve improved treatment outcomes in patients. Our initial areas of focus are viral and liver diseases where our team can leverage their in-depth knowledge and expertise to develop potentially best-in-class combination regimens for each disease area with significant unmet medical need.

Alltrna is the first transfer RNA (tRNA) platform company, and our mission is to unlock tRNA biology and pioneer tRNA therapeutics to regulate the protein universe and resolve disease. Founded at Flagship Labs in 2018, Alltrna has mapped tRNA biology to systematically design tRNA medicines and encode a completely new, unifying approach to treating both rare and common human diseases driven by shared genetic mutations. Alltrna’s platform combines internal expertise and proprietary machine learning tools to unlock the entire tRNA biology space. We have an unprecedented opportunity to advance a single tRNA medicine to restore disrupted protein production, regardless of target, for thousands of diseases with the same underlying genetic mutation.

Alnylam is the world's leading RNAi therapeutics company and the first and only company to bring RNAi-based medicines to market. We are developing RNAi (RNA interference) as an innovative, entirely new class of medicines to treat rare genetic, cardio metabolic, acute hepatic infectious and central nervous system (CNS) and ocular diseases. Alnylam was founded in 2002 based on a Nobel Prize-winning breakthrough in biology – the discovery of RNAi, and a bold vision that this discovery could be used to silence disease-causing genes upstream of today’s medicines. We are relentless in our pursuit of new treatments because we believe that RNAi therapeutics can be used to treat many diseases for which treatment options do not exist or are inadequate. Alnylam is turning scientific possibility into reality - in 2018, the FDA and EMA approved of our first product, ONPATTRO (patisiran) which is also the first-ever approved RNAi therapeutic. We now have three additional medicines on the market: GIVLAARI (givosiran), OXLUMO (lumasiran) and LEQVIO (inclisiran) in partnership with Novartis). Our robust pipeline of investigational medicines includes multiple programs in late-stage and early-stage clinical development. We are a global and diverse company of more than 1,700 people. We pride ourself on fostering a fun, inclusive and dynamic work environment where employees can recognize their fullest potential. That's why we've been named a Science Magazine Top Employer 3 years in a row ('19-'21), a Boston Globe Top Place to Work 7x in a row ('15-'21) and one of Fast Company's Best Workplaces for Innovators for 2021.

Altamira Therapeutics (former Auris Medical) is dedicated to developing therapeutics that address important unmet medical needs. The Company is currently active in three areas: • the development of RNA therapeutics for extrahepatic therapeutic targets (OligoPhore™ / SemaPhore™ platforms; preclinical), • nasal sprays for protection against airborne viruses and allergens (Bentrio™; commercial) or the treatment of vertigo (AM-125; Phase 2) • the development of therapeutics for intratympanic treatment of tinnitus or hearing loss (Keyzilen® and Sonsuvi®, Phase 3). The Company was founded in 2003, under the name Auris Medical, and is headquartered in Hamilton, Bermuda with its main operations in Basel, Switzerland. The shares of Altamira Therapeutics Ltd. trade on the NASDAQ Capital Market under the symbol “CYTO.”

Altogen Labs is a biology CRO company providing GLP preclinical research services, tox studies for IND applications, RNAi and gene silencing services, xenograft testing services (100+ tumorigenic cell lines), in vivo siRNA delivery and biodistribution, development of stable cell lines, and many more. Altogen Labs is a GLP compliant laboratory that provides preclinical research and biotechnology contract research services (CRO) to pharma, biotechnology companies, universities, and cancer research centers worldwide. Our services include pharmacology and toxicology assays (IC50), cell banking, generation of stable cell lines, RNAi gene silencing and other in vitro services. Altogen Labs provides following in vivo services for efficacy studies in the drug discovery phase: rodent xenograft models, teratoma formation and analysis services, cancer disease animal models, in vivo siRNA delivery and tissue targeting, pharmacokinetics (PK) and pharmacodynamics (PD) services, immune response and biomarker analysis. Altogen Labs recently developed an `active bioremediation`​ process based on an approach using the most potent oil-degrading bacteria found at multiple oil spill sites in Texas. This technology allows remediation of large amounts of hydrocarbon-contaminated liquids or soil. The genomes of both microorganisms were recently sequenced, which revealed that both strains were previously unreported in the literature. Altogen Labs filed bioremeddiation patent application in 6/2012.

Launched in February 2024, Alys Pharmaceuticals is an innovation leader in immuno-dermatology, co-founded by Medixci and world-leading dermatology and scientific experts. Originating from the aggregation of six asset-centric Medixci compannies, Alys boasts a robust pipeline of innovative programs and platforms targeting multiple dermatological indications. With a vision to transform the treatment paradigm for several dermatology indications of significant prevalence and major unmet medical need, Alys aspires to redefine the landscape of dermatological treatments.

Amaroq Therapeutics is a New Zealand-based biotechnology company focused on developing a new class of therapeutics that target lncRNA in cancer. Long non-coding RNAs are a relatively recently discovered class of molecules often referred to as “dark matter” of the human genome. These naturally occurring molecules do not code for proteins but fulfill important regulatory functions in the cell that could be exploited as a new therapeutic approach for multiple indications. Amaroq’s research team has discovered lncRNA molecules that are highly expressed in cancer cells, and once removed, cancer cell growth slows. Amaroq Therapeutics is developing innovative therapeutics to target lncRNAs for cancer treatment. Amaroq Therapeutics is funded by the Medical Research Commercialisation Fund, managed by life science venture capital firm Brandon Capital Partners, Otago Innovation Ltd, University of Otago, Cure Kids, and NZ Innovation Booster. The Company has been spun out of research from the University of Otago in Dunedin, New Zealand.

APITBIO is a biotech company that specializes in antibody therapeutics research and development.

Reprogramming DNA methylation in disease conditions by RNA therapeutics APTADIR Contact us NEWS Press Release Reprogramming DNA methylation by RNA therapeutics A novel therapeutic approach for untreatable cancers and rare diseases Aberrant methylation by DNA methyltransferase 1 (DNMT1) of certain genes may cause abnormal cell development and lead to disease. Current therapies targeted to inhibit

Arbutus Biopharma Corporation, a biopharmaceutical company, engages in the discovery, development, and commercialization of a cure for patients suffering from chronic Hepatitis B virus (HBV) infection in the United States. Its HBV product pipeline consists of AB-729, a proprietary subcutaneously-delivered RNA interference product candidate, which in an ongoing Phase Ia/Ib clinical trial targeted to hepatocytes that inhibits viral replication and reduces various HBV antigens using novel covalently conjugated GalNAc delivery technology; and AB-836, an oral capsid inhibitor that suppresses HBV DNA replication. The company’s research and development programs include HBV RNA destabilizers, an orally active agent to destabilize HBV RNA, which leads to RNA degradation and to reduction in HBV proteins; oral PD-L1 inhibitor to enable reawakening patients’ HBV-specific immune response; and small molecule antiviral medicines to treat coronaviruses, including COVID-19. It has strategic alliance, licensing, and research collaboration agreements with Marqibo; Gritstone Oncology, Inc.; and Alnylam Pharmaceuticals, Inc. and Acuitas Therapeutics, Inc. The company was formerly known as Tekmira Pharmaceuticals Corporation and changed its name to Arbutus Biopharma Corporation in July 2015. Arbutus Biopharma Corporation is headquartered in Warminster, Pennsylvania.

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR® mRNA Technology (sa-mRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus developed Kostaive®, the first self-amplifying messenger RNA (sa-mRNA) COVID vaccine in the world to be approved. Arcturus has an ongoing global collaboration for innovative mRNA vaccines with CSL Seqirus, and a joint venture in Japan, ARCALIS, focused on the manufacture of mRNA vaccines and therapeutics. Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), along with its partnered mRNA vaccine programs for SARS-CoV-2 (COVID-19) and influenza. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (over 400 patents and patent applications in the U.S., Europe, Japan, China, and other countries).

Argo Biopharma is a clinical stage biotech company that committed to developing a new generation of siRNA drugs to provide better treatments for global patients. In the past two years, Argo biopharma has built a rich and differentiated pipeline of siRNA drug candidates for a wide range of indications, utilizing its industry-leading siRNA platform technology known as RADS (RNA molecules with superior Activity, Durability, and Safety). Led by an experienced and professional team, Argo Biopharma has demonstrated scientific excellence in siRNA drug discovery and strong execution in advancing potential therapeutic products from discovery to clinical stage.

ARQGenetics offers custom-designed gene panels to provide accurate real-time gene expression data.

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

ARTHEx Biotech is a spin-off biotech company from the University of Valencia (Spain) focused on the research and development of novel oligonucleotide therapies that modulate microRNA to treat diseases with unmet medical needs. ARTHEx has developed its proprietary technology, the ENTRY ™ platform, that allows fast and flexible development of investigational miR-modulating oligonucleotides that are designed to enter relevant tissues in pharmacological quantities, in order to treat diseases in which microRNAs play a key role. The first investigational product from this platform is ATX-01, an antimiR in preclinical development for Myotonic Dystrophy Type 1 (DM1). DM1 is an orphan disease affecting more than 900,000 people worldwide (estimated prevalence 1/8,000) with no cure, nor approved treatment to date. ATX-01 is a first-in-class disease-modifying therapy that addresses the cause of DM1 with a dual and unique mechanism of action, which targets micro-RNA-23b a key factor in the disease pathogenesis. To learn more about Arthex Biotech, please visit us at www.arthexbiotech.com

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs.

We are a biotechnology company pioneering new treatment options for neurodegenerative diseases by utilizing our proprietary RNA interference platform. We were founded by Anastasia Khvorova, Ph.D., Craig Mello, Ph.D., and Neil Aronin, M.D., of the RNA Therapeutics Institute at the University of Massachusetts Medical School, with a Series A funding exclusively by F-Prime Capital. We are headquartered in Boston, Mass

Developed a proprietary platform that holds the potential to discover novel structure targets across the transcriptome and for the design of RNA-targeted small molecules, RNA-based medicines and RNA tools.

Specialty oncology-focused biopharmaceutical company based in Malvern, PA with a broad pipeline encompassing novel best in class selective HDAC6i immunomodulators and a game changing myeloid based discovery platform. Avstera focuses on foundational tumor biology science, while methodologically breaking barriers using cutting-edge approaches in order to deliver impactful treatments to cancer patients.

At baseclick GmbH we believe that bioconjugation is not just a method to connect (bio)molecules, but a tool to develop valuable applications. So, a new strategy was needed: click chemistry! " Nobel Prize 2022, the basis of the baseclick patented nucleic acid modification technology". We are a dynamic, highly professional team and enabling life science researchers to overcome challenges and create better solutions in diagnostics, therapeutics, and vaccines development through our proprietary click chemistry. Our mission is to develop and provide the best nucleic acid labeling applications and reagents for the life science market. For further information please visit our website https://www.baseclick.eu/ https://www.baseclick.eu/our-vaccine-strategy-and-its-benefits/

Basking is developing the first in class agent (BB-031) targeting von Willebrand Factor (VWF) along with a direct acting reversal agent (BB-025) to immediately the pharmacological effect of BB-031 in the event of bleeding. BB-031 is an RNA aptamer optimized for rapid onset of action and short duration of effect. Basking will initiate a Phase 2 trial in acute ischemic stroke in late 2023 and is exploring further application in pulmonary embolism in preclinical studies. The technology is based on two decades of translational research on RNA aptamers as therapeutic agents for cardiovascular diseases.

Shanghai BDgene Therapeutics Co., Ltd. was established in 2018. The founder team includes top scientists in the field of gene therapy, industrial experts with a background in international pharmaceutical companies, toxicology and safety pharmacology experts with preclinical drug evaluation experience.

BeBetter Medicine Technology Co., LTD. is a rapid growth novel drug discovery and development company located in Guangzhou, China. It was founded in 2012 by overseas returnees and aims to create advanced novel medicines to treat cancer and other severe diseases. The company has strong expertise and outstanding facilities to conduct comprehensive pharmaceutical research from drug design, synthesis to clinical development. Currently there are eight innovative drug candidates with own IP rights are in clinical or IND stages. Among them, BEBT-908 has entered the pivotal phase II trial for the treatment of lymphoma. BEBT-209 and BEBT-109 are in the phase Ib/II trial for the treatment of breast cancer and lung cancer, respectively. The company is expected to have a number of new drugs on the market in the next few years.

Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference (RNAi) with gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. Benitec is using its technology, called DNA-directed RNA interference (ddRNAi) to develop a pipeline of product candidates for the treatment of several chronic and life-threatening human diseases , including oculopharyngeal muscular dystrophy (OPMD) and hepatitis B. By combining the specificity and gene silencing effect of RNAi with gene therapy, ddRNAi has the potential to produce long-lasting silencing of disease-causing genes from a single administration, which could minimize the requirement for patients to take regular doses of medicine. Benitec’s objective is to become the leader in discovering, developing, clinically validating and commercializing ddRNAi-based therapeutics for a range of human diseases with high unmet clinical need or large patient populations and, as a result, provide a better life for patients with these diseases.

Beta Hatch is industrializing insect agriculture within a regenerative food system. We make protein for plants and animals in a zero waste system using at our mealworm ranch in Cashmere, WA. Our circular production system uses waste heat from a neighboring data center to condition our insect grow rooms, in collaboration with the Washington Clean Energy Fund.

BIOCAD is Russia's leading innovative biotechnology company; it combines a world-class research and development center, ultra-modern pharmaceutical and biotechnological manufacturing facilities, as well as preclinical and clinical research infrastructure compliant with international standards. BIOCAD is one of the world`s few full-cycle drug development and manufacturing companies, from new molecule discovery and genetic engineering to large-scale commercial production and marketing support. BIOCAD`s medicines are dedicated to treat complex health conditions such as cancer, HIV and Hepatitis C infections, multiple sclerosis and other disorders.

BioEcho Life Sciences is a specialized solution provider for the extraction and analysis of nucleic acids. We create disruptive technologies, products, and workflows that make downstream processing of nucleic acids easier and faster, significantly increase throughput, and deliver reliable results. Our EchoLUTION technology enables the fastest DNA and RNA extraction on the market - in just one single step. It reduces the associated plastic consumption by up to 70 %. Customers in molecular diagnostics, academic and biopharmaceutical research, plant and animal breeding rely on our products: ready-to-use kits, high-throughput solutions for automation platforms, nucleic acid extraction services and process optimization for molecular biology laboratories. BioEcho, located in Cologne, Germany, was founded in 2016 by leading industry experts and is certified according to ISO 9001 and ISO 13485. During the pandemic, our Viral RNA/DNA Swab kit was used in up to 15 % of Covid PCR tests in Germany and 50 % of tests in Austria. Through our European direct Sales team, our global distributor network, and our international online shop we serve a worldwide growing customer base with best-in-class products and services. BioEcho. The Nucleic Acid Experts. Follow us on LinkedIn so that we can keep you posted and get in touch via contact@bioecho.de.

RNAi

BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules.   Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here:  https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html

BIORCHESTRA is an innovative biotech company focusing on research and development of ribonucleic acid (RNA) based therapeutics. Our goal is to change the therapeutic paradigm from treating the symptoms to providing fundamental cure of the diseases. As we are making steady progress in the clinical development, we seek global partners to collaborate in the areas of research and development (R&D) and the commercialization.

Borealis Biosciences was founded on the premise that a convergence of scientific and translational breakthroughs is enabling the field of RNA medicines to treat kidney diseases and beyond. The company is backed by founding investor Versant Ventures and Novartis AG, and has a strategic collaboration with the pharma.

Brii Biosciences is a biotechnology company developing therapies to address major public health challenges where patients experience high unmet medical needs, limited choice and significant social stigmas. With a focus on infectious and central nervous system diseases, we have built a robust pipeline of potential treatment options based on patient insights and experiences, and are advancing these differentiated investigational therapies to address patient choice. Led by a visionary and experienced leadership team, Brii Bio has deep scientific expertise and a proven ability to progress therapeutic assets from discovery to commercial approval on a consolidated timeline. Established in 2018, Brii Bio now has operations in key biotech hubs, including Raleigh-Durham, the San Francisco Bay Area, Beijing and Shanghai. On July 13, 2021, the Company was officially listed on the Hong Kong Stock Exchange under the stock code 2137.HK.

Cambridge Nucleomics is a biotechnology company that specializes in fast and easy RNA measurement at scale with single-molecule accuracy.

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology.

Cardior Pharmaceuticals is a leading clinical-stage biopharmaceutical company pioneering the discovery and development of RNA-based therapeutics designed to prevent, repair and reverse diseases of the heart. Cardior’s therapeutic approach uses distinctive non-coding RNAs as an innovative platform for addressing the root causes of cardiac dysfunctions. The company aspires to bring transformative therapeutics and diagnostics to patients and thereby make a lasting impact on the treatment of cardiac diseases worldwide.

Maintaining musculoskeletal health is the keystone of overall health as we get older. As we age our physical condition gradually deteriorates, leading to loss of strength, fitness and mobility. In most cases, this isn’t due to a simple decision to stop exercising. It is due to the breakdown of musculoskeletal function caused by disease. Musculoskeletal diseases such as osteoarthritis, tendinopathy and lower back pain are the primary causes of disability and early retirement in people over the age of 65. Currently, there are no approved disease-modifying drugs for these diseases, meaning that physical decline in old age is inevitable. At Causeway, we believe that physical decline in old age is not inevitable and in indeed may be reversible. To address this, we are developing a range of therapies that treat common MSK diseases that will allow us to remain fit and active into our nineties and beyond. Staying active as we get older also reduces the risk of developing osteoporosis, dementia, diabetes, obesity, cancer and cardiovascular disease.

Ceria Therapeutics develops innovative drug products for inflammatory disorders. We intend to transform lives by eliminating the consequences of inflammation.

Channavix Therapeutics, LLC is developing a portfolio of novel, non-addictive, non-opioid analgesics that provide multi-week pain relief with local administration. The business and scientific team that has been assembled for Channavix has a long history of working in the field of pain management and drug development. Channavix is dedicated to the discovery of novel therapeutics for neurological disorders such as pain, with a mission to improve patient outcomes and address the global opioid problem.

Chimerna Therapeutics is a biotech company focused on RNA therapeutics. Their platform for generating circular RNA overcomes the major limitations of RNA therapeutics, such as stability and abundance.

Chimeron Bio is a biotechnology company focused on developing personalized gene therapies, including self-amplifying RNA vaccines and therapeutics designed on its proprietary technology.

CircNova, Inc. is a biotechnology company that leverages our unique AI NovaEngine™ to generate, analyze and identify circular RNA for therapeutic development.

Circular Genomicsis abiotechnology and genomics company with a vision to use circular RNAs as novel and improved biomarkers for the diagnosis and better treatment of psychiatric and neurological diseases

Founded by pioneering executives and scientists in RNAi, City Therapeutics is advancing next-generation engineering of small interfering RNAs (siRNAs) – the “trigger” molecules that mediate RNAi – to improve and expand the reach of RNAi-based medicines. City’s mission is to build the leading next-generation RNAi therapeutics company, unlocking RNAi’s transformative potential to help patients with a wide range of diseases.

Comanche is a biopharmaceutical company developing novel siRNA molecules for the treatment of preeclampsia. Our purpose is to lower the risks of pregnancy and prematurity worldwide by safely sustaining natural pregnancy and allowing for fetal maturation. We envision a world where all pregnant women have access to safe and effective therapies that are evidence-based, cost-effective and sustainable. Our mission is to prevent or treat preeclampsia at its root cause.

CureVac (Nasdaq: CVAC) is a pioneering multinational biotech company founded in 2000 to advance the field of messenger RNA (mRNA) technology for application in human medicine. In more than two decades of developing, optimizing, and manufacturing this versatile biological molecule for medical purposes, CureVac has introduced and refined key underlying technologies that were essential to the production of mRNA vaccines against COVID-19, and is currently laying the groundwork for application of mRNA in new therapeutic areas of major unmet need. CureVac is leveraging mRNA technology, combined with advanced omics and computational tools, to design and develop off-the-shelf and personalized cancer vaccine product candidates. It also develops programs in prophylactic vaccines and in treatments that enable the human body to produce its own therapeutic proteins. Headquartered in Tübingen, Germany, CureVac also operates sites in the Netherlands, Belgium, Switzerland, and the U.S. Further information can be found at www.curevac.com.

DESTINA Genomics Ltd. is a biotech company founded in Edinburgh in 2011 by Hugh Ilyine, Juan J. Diaz-Mochon and Mark Bradley. In July 2012, it was created the Spanish subsidiary DESTINA Genomica S.L. with the goal to accelerate a long-term growth plan. The company is focused on the application and validation of its patented state-of-the-art technologies. DESTINA has created a unique and reliable chemistry for highly specific detection of nucleic acids. It can be used to identify any known target nucleic acid sequence, and in particular can directly detect small RNAs, without the multiple steps required by current methods. DESTINA mission is to become a leading manufacturer and supplier of custom chemical reagents and technologies for ‘best in class' nucleic acid based biomarker assays. DESTINA's technology introduces cheaper, more accurate and much more meaningful Nucleic Acid Testing tools.

DiamiR developsand diagnostictechnology based on analysis of brain-enriched microRNA biomarkers in blood plasma.

DxTerity is bringing the power of precision medicine to autoimmune disease. We are pioneering the use of RNA-based immune system profiling to better understand the root causes of immune mediated conditions. Our goal is to provide more personalized care for autoimmune sufferers and empower patients with more detailed information about their condition so they can better navigate their complex health journey.

Eclipsebio develops first-in-class technologies, analyses, and platforms for the development of tomorrow’s RNA-based and RNA-targeting therapies. With our extensive experience in supporting early-stage basic research to evaluating preclinical vaccines and gene therapies, we provide unparalleled support for obtaining deep insights into RNA and therapeutic biology. We offer our solutions as end-to-end partnerships, including custom assay development, for biopharma and biotech companies and as a la carte services. Our areas of support include: - The optimization and characterization of RNA-based vaccines and therapeutics through our eMERGE platform - Multiomic characterization of small molecule and small oligonucleotide targets - Robust validation of on-target, and the identification of off-target, effects from small molecule and small oligonucleotide drugs - Deep profiling of RNA for groundbreaking discoveries in academic research Contact us today to discover how we can provide you with the insights needed for RNA success.

Eleven Therapeutics is a biotechnology company leading the AI revolution in nucleic acid therapeutics. They harness the power of combinatorial chemistry and AI to transform RNAi drug development into a programmable process.

Eli Lilly and Company discovers, develops, and markets human pharmaceuticals worldwide. It offers Basaglar, Humalog, Humalog Mix 75/25, Humalog U-100, Humalog U-200, Humalog Mix 50/50, insulin lispro, insulin lispro protamine, insulin lispro mix 75/25, Humulin, Humulin 70/30, Humulin N, Humulin R, and Humulin U-500 for diabetes; and Jardiance, Trajenta, and Trulicity for type 2 diabetes. The company provides Alimta for non-small cell lung cancer (NSCLC) and malignant pleural mesothelioma; Cyramza for metastatic gastric cancer, gastro-esophageal junction adenocarcinoma, metastatic NSCLC, metastatic colorectal cancer, and hepatocellular carcinoma; Erbitux for colorectal cancers, and various head and neck cancers; Retevmo for metastatic NSCLC, medullary thyroid cancer, and thyroid cancer; Tyvyt for relapsed or refractory classic Hodgkin’s lymph and non-squamous NSCLC; and Verzenio for HR+, HER2- metastatic breast cancer, node positive, and early breast cancer. It offers Olumiant for rheumatoid arthritis; and Taltz for plaque psoriasis, psoriatic arthritis, ankylosing spondylitis, and non-radiographic axial spondylarthritis. The company offers Cymbalta for depressive disorder, diabetic peripheral neuropathic pain, generalized anxiety disorder, fibromyalgia, and chronic musculoskeletal pain; Emgality for migraine prevention and episodic cluster headache; and Zyprexa for schizophrenia, bipolar I disorder, and bipolar maintenance. Its Bamlanivimab and etesevimab, and Bebtelovimab for COVID-19; Cialis for erectile dysfunction and benign prostatic hyperplasia; and Forteo for osteoporosis. The company has collaborations with Incyte Corporation; Boehringer Ingelheim Pharmaceuticals, Inc.; AbCellera Biologics Inc.; Junshi Biosciences; Regor Therapeutics Group; Lycia Therapeutics, Inc.; Kumquat Biosciences Inc.; Entos Pharmaceuticals Inc.; and Foghorn Therapeutics Inc. Eli Lilly and Company was founded in 1876 and is headquartered in Indianapolis, Indiana.

enGene Inc. has developed a highly flexible nucleotide (DNA and RNAi) delivery technology that targetsmucosal tissues and can be used to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels. Our platform technology has the ability to significantly impact diseases of mucosal tissues such as the gastrointestinal tract, lung and bladder as well as provide systemic release of proteins from the gut to treat diabetes, anemia, hemophilia and others.

Envisagenics was founded in 2014 as a spinout of Cold Spring Harbor Laboratory. Our mission is to reduce the complexity of biomedical data to accelerate the development of innovative therapeutic solutions through RNA splicing analytics and artificial intelligence. We focus on the discovery of RNA therapeutics. Over 30 million people in the US suffer from genetic diseases or cancer that could be caused by mutations affecting RNA splicing. 370 human diseases are known to be caused by splicing errors, and more remain to be discovered. Our breakthrough technology, SpliceCore, is a cloud-based platform that is experimentally validated to predict drug targets and biomarkers through splicing discovery from RNA-seq data, using Artificial Intelligence. We are using our software to identify splicing errors causative of diseases, identify the right drug-targets and develop RNA therapeutics through partnerships and collaborations with Biopharma. Our in-silico RNA therapeutics discovery platform replaces expensive drug-target selection and lead design with efficient computer simulations, decreasing time, cost, and failure risk of drug development programs.

Designer Sponge RNAs for Cellular Health Restoration

Fighting the world’s most devastating diseases through the innovative power of targeted oral mRNA vaccines.

Eterna Therapeutics is developing breakthrough mRNA cell engineering technologies to repair cellular dysfunction and treat a range of therapeutic indications. We and our strategic partners are advancing innovative nucleic acid and cell therapies that offer the hope of radically improving the health outcomes of patients with high unmet medical needs. We are committed to creating a world in which patients and their families have access to effective, life-changing treatments for serious illnesses.

Driving discovery and solving challenges for partners across LNP formulation and RNA chemistry | www.etherna.be | www.ethernamanufacturing.com We are an RNA technology company, with over 30 years’ experience, offering professional partnership to pharma and biotech with an emerging or established interest in RNA therapeutics. Our expert team supports start-up, early stage and late stage projects by using our proprietary platforms across RNA chemistry, LNP formulation and Process Technology, to drive new pipeline development or resolve manufacturing challenges for our partners.

Exai Bio is a next-generation liquid biopsy company. Our mission is to enable a world where cancer can be detected early, diagnosed accurately, treated in a personalized and targeted way, and ultimately cured. The company's proprietary RNA and artificial intelligence-based liquid biopsy platform delivers clinical insights into cancer biology to enable the earliest, most accurate diagnosis of cancer. Exai Bio was founded with technology from the UCSF laboratory of Dr. Hani Goodarzi, and consists of veteran leaders in the RNA biology, liquid biopsy, and artificial intelligence/machine learning fields from both academia (UCSF, Stanford, MIT) and industry (Bluestar, CareDx, Evidation, Google, 23andMe, Deep Genomics). Exai Bio is supported by leading life sciences and tech investors including Section 32, Casdin Capital, and Two Sigma Ventures, who have been integral to advancing cancer diagnostics, and artificial intelligence and machine learning technologies. We know that people are our greatest asset and we are rapidly building a rock-star team of talent. Our people are driven by the mission, inspired by the vision of the future, and are committed to living our core values (integrity, openness, teamwork, exploration, and kindness) that connect us to our work and to one another. What We Value Our core values represent what we stand for as a team. This is what we believe in. Everyday, we strive to align our behaviors to these core values in everything we do. Integrity – Our thoughts and actions are true to science and to people. We do the right thing. Openness – We recognize and respect our differences – and embrace learning from them. Teamwork – We are team players who trust and respect each other. Exploration – We are explorers, putting curiosity into action. Kindness – We are kind whenever possible – and we believe it's always possible.

ExcepGen is a venture-backed, early stage biotechnology company located in San Francisco. The company is focused on solving fundamental underlying problems in drug discovery and applying those solutions to create a new generation of therapeutics.

Excision BioTherapeutics is a private company focused on developing advanced gene editing therapeutics to address the medical need for the treatment of life-threatening disease caused by viral infections. Our mission is to advance gene editing therapeutics for our indications into safe and efficacious medicines that will greatly improve the lives of infected individuals around the world.

Our mission is to deliver mRNA technology innovations to revolutionize future therapies and the prevention of many diseases. We invent, develop, manufacture and apply our solutions for the benefit of pharmaceutical partners, and to improve people’s lives. With passion, ExploRNA Therapeutics develops ground-breaking innovations of mRNA as a therapeutic platform, applies those solutions in unique therapeutic programs, and delivers technology, therapeutic platform, and therapies to pharmaceutical partners.

Flamingo is pioneering RNA-targeted therapies for oncology with state-of-the art chemistries and a clinical-stage pipeline targeting undruggable transcription factors and splice variants. Flamingo Therapeutics has a Phase II trial (PEMDA-HN) evaluating the STAT3 targeting agent danvatirsen, in combination with pembrolizumab, in patients with head and neck squamous cell carcinoma (HNSCC). Flamingo has an alliance with Ionis Pharmaceuticals and is supported by well-known biotechnology investors including Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Eurazeo - Kurma Partners, Perceptive Advisors, PMV, Pontifax, Sphera, funds managed by Tekla Capital Management LLC and VIB.

Fluent BioSciences, an Illumina company, is a biotechnology company with a mission to accelerate the understanding of biology and disease through simple, cost effective and scalable solutions for every laboratory from research to the clinic. Fluent’s breakthrough Pre-templated Instant Partitions (PIPseq™) technology enables near-instantaneous self-assembly of individual cells or molecules into millions of uniform partitions without the need for complex instrumentation or expensive consumables. This powerful platform enables extremely sensitive and unbiased preparation of proteins and nucleic acids for a broad range of discovery and disease applications including single-cell RNA sequencing (scRNA-Seq).

Geneoscopy is a life sciences company with the mission to empower patients and providers to transform gastrointestinal health through innovative diagnostics. Geneoscopy's lead diagnostic uses stool-derived eukaryotic RNA (seRNA) to detect colorectal cancer and precancerous adenomas. This device was awarded a Breakthrough Device Designation from the FDA for its ability to reduce morbidity associated with colorectal cancer through advanced adenoma detection. Our initial prospective clinical study demonstrates that the diagnostic can detect these lesions with greater sensitivity than all existing noninvasive screening tests, thereby, serve as a potential aid in early detection and prevention of cancer. Geneoscopy is also developing diagnostics for the diagnosis and management of inflammatory bowel disease in partnership with biopharmaceutical companies and leading academic research institutions.

Gennova Biopharmaceuticals Ltd. is a biotechnology company based in Pune, Maharashtra, India. The company specializes in the research, development, production, and commercialization of biotherapeutics and vaccines aimed at treating life-threatening diseases in areas such as cardiovascular, neurology, nephrology, and oncology. Gennova employs advanced recombinant DNA technologies and innovative bio-manufacturing practices to create effective biotherapeutic solutions. The company utilizes various expression platforms, including bacterial, mammalian, and yeast-based systems, to produce its recombinant products. Gennova has successfully commercialized seven products, including Tenectase, a biosimilar for treating Acute Ischemic Stroke, and HAMSYL, a treatment for acute lymphoblastic lymphoma offered at a lower price point for better accessibility. With a strong focus on research and development, Gennova is recognized as a preferred partner in the vaccine sector for research and cGMP manufacturing. The company employs around 598 people and has reported a revenue of $2.8 million, positioning itself as a notable player in the Indian biotechnology landscape.

Gradalis is a late-stage clinical biotechnology company developing immunotherapies for multiple cancer indications including an upcoming Phase 3 registration trial in patients with ovarian cancer. The company’s proprietary bi-shRNA therapy platform, which has been proven in its clinical studies, can be utilized to silence a broad range of genes and is applicable to multiple cancer types. About Vigil Vigil® is the first cellular therapy to show positive survival benefits in a randomized controlled clinical study of patients with solid tumors. Vigil is a self-navigating cellular immunotherapy that addresses the major deficits of leading cancer treatments. Vigil uses all of a patient’s tumor-related antigens to identify cancer cells and then enhances the training of the immune system to recognize and attack those cells. Gradalis’s proprietary bi-shRNA platform accomplishes this via three key functions: (1) it blocks furin, the enzyme that generates the immunosuppressive cytokines TGF beta types 1 and 2; (2) it activates effector cells including antigen-presenting dendritic cells and CD8+ T cells via the production of GM-CSF; and (3) it induces an immune response to the full complement of neo-antigens from the patient’s tumor via intradermal administration. In a Phase 2b trial, Vigil showed a positive trend in the primary endpoint of recurrence-free survival in the overall population and a significant improvement in recurrence-free survival and overall survival in a pre-planned subgroup analysis of Stage III/IV newly diagnosed ovarian cancer patients with the BRCA wild type (BRCA-wt) molecular profile. The company is preparing to initiate a clinical study intended for product registration in patients in this subgroup. Additionally, Phase 1 results in an all-comer trial have shown positive signals of activity in multiple tumor types.

Multivalent RNAi Platform, Delivery

At Harness Therapeutics we have one mission – physiological upregulation of targets that have the promise to provide disease-modifying therapeutics for neurodegenerative diseases. We use a range of mRNA-targeted oligonucleotide-based methodologies to alter the post-transcriptional regulation of protein synthesis and so increase the target protein in a controlled manner. Our focus on neurodegenerative disease is driven by our passion to develop truly disease-modifying therapies for diseases that affect so many people, yet for which there are few if any therapies that can slow or halt the progression of the disease. The ability to effect controlled increases of protein levels is essential in being able to safely drug targets with narrow therapeutic windows, something that existing platforms cannot achieve. This is critical for neurodegenerative disease as there are now a number of targets that have strong genetic validation linked to disease progression but require controlled increases rather than knock-down or over-expression. Our goal is to open up the target space for neurodegenerative disease and be able to advance effective treatment solutions for the tens of thousands of patients and their families who are facing the certainty of irreversible disease progression, loss of quality of life, and ultimately death. Our name reflects our approach, built and refined since our formation in 2020; harnessing the body’s endogenous cellular processes controlling protein synthesis to achieve controlled increases in protein levels in the safest possible manner. We use our deep expertise in RNA biology/bioinformatics and neuro cell biology to probe the intricate regulation of each target and identify multiple upregulation opportunities. Our multi-year experience focussed on physiological upregulation has allowed us to develop bespoke assays and analytical techniques to screen for constructs that drive small increases in protein, which is a key challenge in this space. Our sole focus on neurodegeneration has allowed us to build deep expertise in the biology of neurons and our targets, and the best possible models for disease – critical in being able to de-risk and translate candidates to the clinic. Neurodegenerative disorders are chronic conditions that destroy parts of the nervous system over time, especially the brain. They result in progressive loss of cognitive and motor function and eventually, death. The most common neurodegenerative diseases include Alzheimer’s disease, Parkinson’s disease, Amyotrophic lateral sclerosis (ALS), Motor neuron disease (MND), Huntington’s disease (HD), Spinal muscular atrophy (SMA), and Spinocerebellar ataxia (SCA). It is estimated that more than 50 million people worldwide are affected by neurodegenerative diseases and this number is increasing as overall life expectancy increases. Most of these conditions are much more likely to happen in people over 65, but some conditions like Huntington’s disease and ALS often appear much earlier.

HAYA Therapeutics is a precision medicines company developing RNA-guided programmable therapeutics targeting the regulatory genome for serious health conditions including cardiovascular disease and cancer. The company is using its innovative cell-state modifying platform to gain novel insights into the biology of long non-coding RNAs (lncRNAs), the ‘dark genomes’ information processing features and ‘source code’. HAYA’s lead therapeutic candidate is HTX-001, an antisense oligonucleotide targeting Wisper, which is a tissue and cell-specific cardiac lncRNA known to play a role in heart failure. The company is also developing a pipeline of lncRNA-targeting candidates for the cell-specific treatment of diseases in other tissues, including the lungs and the microenvironment of solid tumor cancers. Headquartered at the life sciences park Biopôle in Lausanne, Switzerland with laboratory facilities at JLABS @ San Diego, HAYA is led by a world-class team of experts in programmable and precision RNA-targeted therapeutics and is supported by a strong investor consortium. HAYA’s mission is to identify and develop breakthrough disease-modifying therapeutics with the potential for greater efficacy, safety, and accessibility than existing treatments.

hC Bioscience is an innovator and pioneer of protein editing using context agnostic engineered tRNA. Their First in Class approach for tRNA drug products includes a variety of delivery approaches, with the goal of improving the lives of patients with the development of first-in-class tRNA-based therapeutics targeting cancer and rare diseases.

Heqet Therapeutics is a company that develops groundbreaking genetic medicines to reverse the damage of ischemic heart disease, the leading cause of death worldwide. Their therapeutic approach aims to address the underlying drivers of heart disease.

Highlight, formerly known as Bioncotech Therapeutics S.L, is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Its lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn ‘cold’ tumors ‘hot’ and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA.

Honeycomb Biotechnologies is an early-stage company focused on making scalable solutions for storage and single cell genomic analysis of precious clinical samples. We enable translation of the rich biological information encoded in clinical specimens into high-resolution digital information, which can be queried and analyzed with sequencing. The resulting exponential growth in knowledge about the state of each person’s health and disease will enable the identification of new therapies and better targeting of current medicines in the era of precision medicine. Our first product is the “HIVE” – a single-use disposable device that is used (without any Honeycomb-specific instrumentation or equipment) for capturing, storing, and analyzing biological samples with single-cell resolution.

Hybridize Therapeutics is a spin-off from the Leiden University Medical Center and is a therapeutic company focused on developing kidney-protecting modalities for untreated kidney diseases.

Immorna is a rapidly expanding biotechnology company, focusing on the development of mRNA-based therapeutics and vaccines. Immorna is utilizing multiple mRNA platforms, including conventional, self-replicating and circular mRNA. Since it’s founding in 2019, Immorna has built a robust CMC platform for mRNA synthesis, purification, and analytical testing that is well suited for clinical and commercial development. In addition, with its state-of-the-art screening tools, Immorna has developed an arsenal of mRNA delivery vehicles, including polymers and lipid nanoparticles featuring multiple proprietary ionizable cationic lipids suitable for intramuscular, intravenous and tissue-targeting delivery. Immorna has a growing intellectual property portfolio and a diverse mRNA development pipeline spanning cancer immunotherapy, infectious diseases, rare genetic diseases, and medical cosmetology, and quickly advancing its oncology and infectious disease drug candidates into their clinical stages.

InnoSIGN commercializes OncoSIGNal mRNA-based tests that measure the activity of signal transduction pathways in cancer and immune cells to predict how patients will respond to targeted drugs.

Innovac Therapeutics is a biotechnology company dedicated to developing innovative vaccines for patients with unmet medical needs, using our proprietary RNA technology.

invIOs is a private biotech company focusing on discovery and development of novel cancer therapeutics that empower and educate the immune system to fight cancer. We leverage our deep expertise in immune-system activation to advance a focused pipeline of individualized and targeted approaches against solid tumors. Our focus is on earlier-stage development, with a sweet spot in bringing assets through discovery and pre-clinical development and into clinical trials. We launched in 2022 as a spin-out bundling the innovative oncology assets of APEIRON Biologics. Our highly motivated team of 35 people has deep expertise across the biotechnology value chain, with in-house expertise in R&D, clinical development, CMC, IP, scouting and more. All of our approaches are designed with a single goal: To transform patients' lives, so that cancer doesn’t.

Founded in 2012, Jiangsu Recbio Technology Co., Ltd. (hereinafter referred to as Recbio or the Company; stock code: 2179.HK) is an innovative vaccine company driven by self-developed technologies. We are dedicated to the R&D, production and commercialization of innovative vaccines, leveraging our core technology platforms (novel adjuvant, protein engineering, immunological evaluation). With robust R&D capacity, the Company has developed high-value innovative vaccine portfolios consisting of ten-odd differentiated vaccines, covering cervical cancer, shingles, COVID-19, TB and other high-burden diseases. Our core product REC603, a recombinant 9-valent HPV vaccine in Phase III clinical trial and has a leading research and development progress. Apart from that, ReCOV, a recombinant COVID-19 vaccine, has been recognized as one of the most competitive next-generation COVID-19 vaccines in the world. Recbio has a clear commercialization strategy aiming to penetrate the diversified global vaccine market.

Judo Bio is a biotechnology company focused on developing precision therapeutics against genetic disorders. The company has raised a total funding of $20M over 1 round and is known for its innovative approach to drug development.

Junevity is a biotech rewinding complex metabolic disease. Based on 6 years of breakthrough research at UCSF, Junevity's REWIND platform identifies high-level regulators of metabolic disease with known pharmacological pathways. Junevity is advancing multiple therapeutic programs towards a new era of medicines.

Kerna Labs is unlocking the full potential of mRNA as the universal toolkit for genetic medicine.

Leal Therapeutics is a biopharmaceutical company that focuses on developing novel therapeutics for patients with major disorders of the central nervous system.

MalarVx is a bio-pharmaceutical company located in theCenter for Infectious Disease Researchfacility in Seattle, Washington.MalarVxwas founded to address the myriad challenges of creating a safe, affordable, effective malaria vaccine using innovative new technologies discovered through research.They are focused on the development of in vitro culture systems for the malaria parasite which will lead to the development of an effective whole-parasite vaccine. Theycollaborate with researchers in non-profit institutions, university infectious disease programs, foundations committed to solving diseases of the third world, and for-profit pharmaceutical and medical devices companies.

The MU Genomics and Bioinformatics cores provide centralized genomic, bioinformatic and biostatistical services to investigators at universities and colleges throughout the state. The Genomics Core currently provides five services to research programs and individual investigators:high throughput next generation sequencing (NGS) to support whole genome, whole exome, RNA-Seq, single cell RNA-Seq, microbiome and global chromatin and methylation studiesbiostatistical and bioinformatic support for NGS projectsaccess to DNA/RNA sequence analysis softwareautomated Sanger DNA sequencing, genotyping and RNA/DNA quality assessmentaccess to shared instrumentation such as plate readers, real time thermal cyclers, Agilent Bioanalyzers, fluorimeters, and spectrophotometers

MDimune - The ultimate goal of MDimune Inc. is to provide an innovative platform technology that can revolutionize the paradigm of human therapeutics by achieving target-specific delivery of effective drugs. The precision of this novel technology will enable safer and more effective treatments of many debilitating human diseases and bring hope to patients.

We develop first-in-class RNA-based therapies, with lead programs in Head & Neck Squamous Cell Carcinoma (HNSCC) and Facioscalpulohumeral Muscular Dystrophy (FSHD). Our approach to drug design revolves around using genomic patient data to create highly tailored therapeutics – the right drug for the right patient. Our proprietary DREAmiR™ platform utilizes genomic and outcome data from thousands of patients to identify underlying genetic changes that cause their disease, and then creates a novel RNA therapeutic that can directly target and fix that genetic abnormality.

Diagnostics We build innovative diagnostic and monitoring tools that empower people to make better decisions about their health. Drug Discovery We translate early-stage research into customized RNAi and CRISPR Cas9 animal and cell models to accelerate drug development. Therapeutics We identify and validate new drug targets, unleashing the commercial potential of RNAi to benefit people on a global scale. Established in 2010 to harness the power of RNAi and genetic engineering technologies developed by our founders in the labs of the world's leading experts at Cold Spring Harbor Laboratory, Howard Hughes Medical Institute, and Harvard Medical School, Mirimus creates innovative research and clinical technologies to address health issues on a global scale. Today, Mirimus markets multiple unique and high-value drug discovery and diagnostic products and services, and is advancing a pipeline of therapeutic candidates in partnership with leading biopharma companies to create novel high-impact health care solutions that benefits society on a global scale. Mirimus' ability to leverage its cutting-edge science and proprietary technology platforms to rapidly create new, affordable technology and products at scale is core to the way we work. We embrace diversity and creativity and have a proven track record of innovative problem solving—which led to our recognition as a $1M grand prize winner of the XPRIZE competition by developing a radically affordable, rapid, non-invasive methodology for mass COVID-19 testing—driving us to make better drugs and detect disease earlier. At Mirimus, our ingenuity, respect for our colleagues' ideas and creativity, executional agility, and a commitment to global health equity combine to create a unique culture of innovation that delivers measurable progress.

Mirxes is a Singapore-headquartered biotechnology company whose mission is to save lives through early, actionable, and personalized diagnoses across the care continuum. We are Asia-centered, starting with Southeast Asia, and have global ambitions. We have research, development, manufacturing, and clinical diagnostic operations in Singapore, USA, Japan, and China. We have sales and distribution networks worldwide. Our flagship product is GASTROClear, the world’s first molecular blood test for early detection of gastric cancer, able to detect around 90% of patients with Stage I and II cancers. We continue to build a comprehensive cancer portfolio with early detection tests for lung and thyroid cancer (2022), colorectal and breast cancer (2023), liver and ovarian cancer (2024). We are concurrently building a precision oncology portfolio to provide clinical insights for the complete patient journey. MiRXES was spun off from A*STAR’s Bioprocessing Technology Institute in 2014 to commercialize an industry-leading qPCR-based technology for microRNA detection. Our ID3EAL discovery tool and workflow has been adopted by top academic and industry partners globally and is backed by the world’s first industry standard for microRNA-base diagnostics. Our versatile platform technology and capabilities have broad applications in the discovery of biomarkers and diagnosis of diseases. We are starting with early cancer detection, but we will also be addressing unmet clinical needs in cardiovascular, metabolic, and infectious diseases. We responded to COVID-19 by leveraging our capabilities to mass produce and distribute over 5 million units of the Fortitude COVID-19 RT-PCR test. This made-in-Singapore diagnostic test, developed by A*STAR and Tan Tock Seng Hospital, has been deployed in 13 Singapore hospitals and clinical labs and exported to over 45 countries worldwide. Post-COVID, we will leverage this network to commercialize multi-cancer early detection tests.

Mitotherapeutix was created to take advantage of advancements in RNA chemistry and its use in developing siRNA based drugs that can target otherwise difficult to address disease targets. Our initial gene target is a novel protein that controls metabolic activity/efficiency in the mitochondria. The ability to control mitochondrial activity is significant because > 90% of the energy of the cell comes from the mitochondria. This gene target is a negative regulator. Thus, in the presence of the regulator, metabolism is slowed down and the removal of the regulator safely increases metabolism. We have explored the function of this metabolic regulator in different tissues and disease states and have found that turning off the regulator can have a positive effect in disease treatment. Targeting this gene product creates the opportunity of producing a drug that can treat a wide array of diseases.

At Moderna, we believe messenger RNA, or mRNA, is the “software of life.” Every cell in the body uses mRNA to provide real-time instructions to make the proteins necessary to drive all aspects of biology, including in human health and disease. Given its essential role, we believe mRNA could be used to create a new category of medicines with significant potential to improve the lives of patients. We are pioneering a new class of medicines made of messenger RNA, or mRNA. The potential implications of using mRNA as a drug are significant and far-reaching and could meaningfully improve how medicines are discovered, developed and manufactured.

NeoCura is a high-tech enterprise that relies on AI technology to create an RNA technology platform and conduct R&D of innovative drugs. The company has built multi-omics big data acquisition platforms and multiple bioomics databases. It uses AI and bioinformatics technology to conduct in-depth drug target mining and fully automated drug design for innovative RNA technology platform upgrades and drug research. It has established leading R&D centers and production centers in Beijing, Shenzhen, and Guangzhou to support pipelines development, platform upgrades, and clinical demand.

Developing RNA therapeutics for disease modification neurological disorders

NextRNA focuses on lncRNAs, which account for the majority of ncRNAs. Their approach is motivated by observations where disregulated interactions between lncRNAs and RNA-binding proteins (RBPs) are implicated in multiple disease areas, including oncology, immunology, and neurology. They are elucidating the proteins that lncRNAs interact with and what cellular processes they affect and designing small molecules to drug these lncRNA-RBP interactions.

Nitto BioPharma is a clinical stage biopharmaceutical company located in San Diego focused on the discovery and development of novel RNA-based therapeutics for the treatment of fibrosis and other unmet medical needs. The company offers unique drug and siRNA delivery solutions by designing novel biodegradable delivery vehicles with the combined ability to target specific cells.

Nosis Biosciences delivers breakthrough receptor-targeted RNA medicines with cutting-edge physics-aware deep learning, single cell biology, and high-throughput chemistry

Nutcracker Therapeutics is an RNA therapeutics company that has combined the power of advanced engineering with high-precision biosynthesis to deploy a complete RNA therapeutics platform. Armed with this high-tech advantage, we have developed a wholly owned pipeline of RNA therapeutic programs and established partnership initiatives with top clinical investigators at several leading institutions across the globe. With our platform’s ability to accelerate the development of life-changing RNA therapeutics, we seek to advance breakthrough RNA therapies at high velocity through all stages of development across a variety of indications. Our technology platform has the potential to significantly reduce costs and cycle times for RNA therapeutic development, with dramatic advantages in capacity scaling and point-of-care delivery over other RNA manufacturing approaches.

Ochre Bio is a biotechnology company developing RNA therapies for chronic liver diseases. Using a combination of genomic deep phenotyping, precision RNA medicine, and testing in live human donor livers, Ochre is developing therapies for important liver health challenges, from increasing donor liver supply to reducing cirrhosis complications. To learn more, please visit www.ochre-bio.com.

OliX Pharmaceuticals is a clinical stage pharmaceutical company developing therapeutics based on the science of RNAi to address a wide variety of illnesses. The Company has developed core platforms (asymmetric siRNA, cell-penetrating asiRNA) targeting locally administrable diseases, such as hypertrophic scars, hair loss, and age-related macular degeneration (AMD). OliX's third RNAi therapeutic platform, GalNAc-asiRNA, targets multiple liver diseases, including NASH/diabetes and HBV. For the general welfare of human beings, OliX is constantly endeavoring to become a leader in the global biotechnology industry by innovating and expanding its pipeline, even to the undruggable diseases.

Oncovir, Inc is a pharmaceutical corporation dedicated to the development of nucleic-acid-based clinical therapies for cancer, infectious, immune, and degenerative disorders. We promote Hiltonol® (poly-ICLC) as an experimental viral mimic and broad activator of innate and adaptive immunity. While initially developed as an interferon inducer, Hiltonol® has much broader biological effects in humans, including specific antiviral, immune activating, vaccine adjuvant, and antitumor actions.

Orbital Therapeutics is building a first-in-kind platform at the intersection of RNA technology, delivery methods, data science and automation to develop an expansive portfolio of RNA-based medicines to treat disease in humans.

Orna Therapeutics is a biotechnology company reshaping RNA therapeutics. We are developing the leading circular RNA technology platform (oRNA®) and unprecedented lipid nanoparticle (LNP) delivery solutions – unlocking the therapeutic possibilities of circular RNA and pioneering the next chapter of safe and effective in vivo therapies for the treatment of disease. Website: www.ornatx.com

Pantherna Therapeutics is a privately held mRNA therapeutics company developing novel first-in-class therapies for indications with high unmet medical need. Pantherna’s development programs are based on two innovative and proprietary PTX platform technologies, offering robust mRNA expression constructs (PTXmRNA) and lipid nanoparticle (LNP) delivery vehicles (PTX_LNP) for localized expression of therapeutic proteins. PAN004 is Pantherna´s first development candidate derived from this platform, representing a defined mRNA-LNP formulation for selective mRNA delivery and expression of a Tie2-agonist in the vascular endothelium of the lung. Pantherna strives for expanding its pipeline with various novel mRNA-LNP candidates enabling the therapeutic use of mRNA beyond the vascular endothelium in distinct tissues such as skeletal muscle or immune cells.

PDX Pharmaceuticals is an IND-enabling stage biopharmaceutical company. We develop novel combination and immunotherapies to treat cancer by bringing complementary biological pathways together to achieve synergistic clinical benefit. Our therapeutic candidates bridge the innate and adaptive immune systems by generating profound CD8+ T cell production. Our pipelines are patented AIRISE (Augmenting Immune Responses and Inhibiting the Suppressive Environment of Tumors) and ARAC (Antigen Release Agent and Checkpoint Inhibitor) pipelines that generate anti-tumor immunity when given locally (AIRISE) or systemically (ARAC) by relying on patient' s own tumor in situ as the source of antigens.

Phalanx Biotech Group provides genomics research products and services worldwide to academic, pharmaceutical and biotech communities. We developed the OneArray® line of high-quality and affordable microarrays for expression profiling of whole genomes, microRNA, and custom targets. Our service specialists offer comprehensive services including sample preparation, expression analysis, and qPCR validation. In 2017, Phalanx is pleased to announce that it has added Next Generation Sequencing Services (NGS) and corresponding NGS Bioinformatics to its service offerings.

Phio Pharmaceuticals Corp. (Nasdaq: PHIO) is a clinical stage biotechnology company whose proprietary INTASYL™ RNAi technology makes immune cells more effective in killing tumor cells. INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics. INTASYL drugs precisely target specific proteins that reduce the body’s ability to fight cancer, without the need for specialized formulations or drug delivery systems. Phio was co-founded by Nobel Laureate Craig Mello, who was awarded the Nobel prize in 2006 for his discovery of RNAi, which has broad therapeutic applications. Our founders opened up a whole new field of biology when they discovered RNAi, and we continue their pioneering efforts today through our work with INTASYL. We’re committed to discovering and developing innovative cancer treatments for patients, creating new pathways for a cancer-free future. For additional information, visit the Company's website, www.phiopharma.com. #INTASYL #siRNA #RNAi #Phio #immunooncology

Neutralizing Antibody Discovery

Pinion Immunotherapeutics is a Maryland based biotechnology company passionate about innovating safer and more efficient RNA preventive vaccines for infectious diseases and therapeutic vaccines for cancer.

Poolbeg Pharma plc is committed to the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, with a growing emphasis on rare and orphan diseases. Its model focusses upon developing its exciting clinical assets and commercialising approved and marketed drugs to fund the development of its robust pipeline of innovative products, thereby driving significant value creation. Poolbeg is led by an experienced leadership team with a history of delivering significant shareholder value. The team has been strengthened by the appointment of three former members of the Amryt Pharma plc leadership team, with the intention of repeating Amryt's success and generating near term revenues. Poolbeg's clinical programmes target large addressable markets including cancer immunotherapy-induced CRS, infectious disease, and metabolic conditions such as obesity with the development of an oral GLP-1R agonist. It uses a cost-effective development philosophy to generate high quality human data to support partnering and further development. Its AI-led infectious disease programmes analyse unique data from human challenge trials to identify clinically relevant drug targets and treatments, leading to faster development and greater commercial appeal.

Prolifagen Therapeutics is a biotech startup dedicated to the prevention of heart failure in patients with large myocardial infarcts. The company is focused on cardiac regeneration based on microRNA technology.

We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for rare genetic diseases. We focus our drug development mainly on a group of blinding disorders affecting the retina, called inherited retinal diseases.

Providence Therapeutics Holdings Inc is a clinical-stage biotechnology company focused on drug development and platform development. The Providence mRNA Medicines platform is comprised of four integrated platforms: 1) mRNA Medicines Platform, 2) NextGen lipid nanoparticles (LNP), 3) Manufacturing Platform and 4) Regulatory Platform. Providence’s mRNA and NextGen LNP platform offer an end-to-end solution for designing codon-optimized ultrapure mRNA that can be formulated in application optimized LNP. The Providence proprietary NextGen LNP, consists of rationally designed lipids that have been tested for suitability for programs in oncology, infectious diseases and gene delivery. Our scalable, efficient platform demonstrates differentiated capabilities in mRNA design, manufacturing, and formulation processes, as well as specific organ and tissue targeting. For more information, please visit providencetherapeutics.com

Creation of optimal nucleic acid medicine for disease treatment with proprietary technology PURMX's solution is a drug discovery seed of small RNA nucleic acid drugs that fine-tune many target genes and deliver unprecedented therapeutic effects. PURMX already possesses a large amount of functional RNA screening data, which is being further expanded. The first stage of innovative nucleic acid medicine seeds is MIRX002. MIRX002 is a nucleic acid medicine for malignant pleural mesothelioma. We have completed non-clinical trials and are conducting investigator-initiated clinical trials at Hiroshima University Hospital starting in 2021.

Ractigen Therapeutics is an early-stage biotech company committed to bringing to market first-in-class therapies designed to selectively restore the expression of therapeutic genes silenced in diseased cells. Its core technology is based on a paradigm-shifting discovery known as RNA activation (RNAa). Founded in 2016 by the pioneers in the field, Ractigen is developing a rich pipeline of candidate medicines for patients with unmet need.

Radar Therapeutics is a biotech company developing programmable precision therapeutics using molecular RNA sensors. The company has raised $13.4 million in seed funding to support its work in developing smart, programmable medicines. Radar Therapeutics is also the winner of the first-ever Amgen Bakar Labs Diversity, Inclusion, and Belonging Award. The company is building a business around medicines that use RNA sensors—mRNAs that gate their expression based on other RNAs in the cell.

RealSeq Biosciences’ core expertise includes innovative proprietary technologies for bias-free small RNA/miRNA NGS library construction, targeted NGS tools, and cf-RNA analysis (liquid biopsy) that form the basis of the Company’s life science research programs and product development.

Recorna Bio is a researcher and developer of RNA-edited drugs, focusing on drug discovery and development by targeting RNA editing cutting-edge technology. The company has completed millions of USD in Angel round funding and collaborates with other RNA companies to discover novel therapies for lung disease.

Regulus Therapeutics Inc. (Nasdaq:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is located in San Diego, California and is led by a seasoned executive team experienced in corporate management, business, science, drug discovery and development. Regulus'​ scientific advisory board consists of world-class scientists and some of the foremost authorities in the field of microRNA research.

Rejuvenation Technologies is a biotechnology company that developed nucleoside-modified TERT mRNA to safely and rapidly extend telomeres. The company is focused on developing mRNA therapies to extend the healthspan by preventing and treating chronic age-related diseases.

Replicate Bioscience is amplifying the power of RNA therapeutics by pioneering novel self-replicating RNA (srRNA) technology. Replicate’s off-the-shelf srRNAs contain two components: virally derived genetic code to drive controlled and self-limiting amplification, and the RNA encoding therapeutic proteins. Our library of viral vectors, selected for driving robust and sustained protein expression and orders-of-magnitude improved performance over linear mRNA, allow for the development of treatments in applications including oncology, infectious disease, and autoimmunity. Differentiated by a team of srRNA experts, a customizable library of synthetic srRNA vectors, and end-to-end development capabilities, Replicate is uniquely positioned to finally expand the reach of RNA treatments toward widespread use in infectious disease, immuno-oncology, autoimmune disease, and more.

Resalis Therapeutics’ transformative metabolic disease approach targets a master regulator of key pathways underlying obesity and fatty liver disease. The company is applying its unique understanding of the non-coding RNA drug modality and lipid metabolism to develop its lead program, RES-010, into a safe and convenient treatment providing disease-modifying therapeutic impact including durable weight loss and reduction of hepatic steatosis. Building on robust preclinical evidence, Resalis will rapidly bring RES-010 into clinical trials for a range of metabolic disorders.

RiboNova is a privately-held biopharmaceutical company based in the Lankenau Institute for Medical Research near Philadelphia, Pennsylvania. Their product pipeline includes a Phase-2 ready small molecule drug for the treatment of all forms of genetically-confirmed mitochondrial disease and a proprietary drug discovery platform that targets transfer RNA with novel precision medicines for the treatment of mitochondrial and other diseases.

Spun out of Yale University in 2020, RIGImmune is a platform biopharmaceutical company developing a novel investigational class of RNA immunotherapies, and advancing complimentary targeted delivery systems, for the broad-spectrum treatment of viral diseases and cancer. Led by biopharma industry veteran, President Susan Sobolov, PhD, RIGImmune’s products act to specifically modulate RIG-I, a host surveillance pathway that triggers the innate immune system to enhance the patients’ intrinsic response to disease. RIGImmune was launched in 2020 by Yale Professors Anna Marie Pyle, PhD and Akiko Iwasaki, PhD, who are also Scientific Advisors for the company. Pyle co-discovered the RIG-I receptor family and conducted many of the first structural and biochemical investigations on RIG-I. Pyle is also a specialist in RNA structure, and design. In collaboration with Iwasaki, Pyle designed the stem loop RNAs for selective targeting of RIG-I using crystal structure data of RIG-I complexed with RNA and developed them as antitumor and anticancer compounds.

RNAConnect, Inc. is a company focused on the development of life science reagents to help further RNA sciences. They promise groundbreaking solutions for exploring the real-world diversity of RNA sequence and structure.

At RNAimmune, we use mRNA technology to help people live full and healthy lives. Our primary focus is development of vaccines against cancer and respiratory viruses. Our expertise in design and production of mRNA vaccines is supported by research and development efforts toward novel lipids, targeted delivery and antigen engineering. We collaborate with the world for the health of everyone.

RNatives is a biotechnology company developing precision RNA-based gene regulating therapies by leveraging its RNAIntelTM platform which identifies small non-coding RNAs that bind directly to gene promoters to regulate gene expression. The Company is developing novel RNA-based therapies to treat ocular, CNS, and metabolic diseases, as well as cancer. The Company's technology is based on years of academic research published by Turunen et al.

Rona Therapeutics is a pioneering RNA therapeutics platform company dedicated to discovery and development of modular and programmable RNA medicines to deliver to patients around the world. The company is founded by industry veteran with deep expertise in RNA therapeutics program execution and world leading scientific advisors with rigorous biology understanding in the RNA space.

RosVivo Therapeutics is a biotechnology company that develops microRNA-based therapeutics for type 2 diabetes, obesity, and fatty liver disease. They have signed a Material Transfer Agreement for first-in-class diabetes treatment with Eli Lilly and Company.

RTLGenomics is a full service sequencing company offering sequencing approaches including barcoding, metagenomicsand transcriptomics. A sister lab, MicroGen, is a CAP accredited clinical diagnostic laboratory.

Rznomics is developing safe and effective biopharmaceuticals in oncology, degenerative disease, and genetic disease based on RNA platform technology called trans-splicing ribozyme. Acknowledged by bio-focused institutions, Rznomics has raised \23.7 billion from series A& B and recently raised \37.2 billion from series C, resulting in a total of \60.9 billion of fundraising. Also, Rznomics received \8 billion for the new drug development research funding from the government. In 2022 1H, Rznomics’ RZ-001 IND submission for the HCC treatment was approved by MFDS, and the clinical trial for the RZ-001 is currently running. In addition to the RZ-001, Rznomics has competitive and innovative pipelines, such as glioblastoma multiforme, Alzheimer’s disease, genetic ocular disease (retinitis pigmentosa), and circular RNA technology. Rznomics is also actively partnering with global companies to discuss collaboration opportunities in co-research, co-development, and licensing.

SanegeneBio is an emerging RNAi-based company developing innovative RNA interference (RNAi) therapeutics driven by the cutting-edge delivery technology. Founded in 2021, SanegeneBio was propelled by a team of industry-leading experts and has operations in both the US and China. Since its inception, SanegeneBio has successfully established proprietary chemical modification platform, hepatic and extrahepatic delivery platforms, enabling tissue-specific delivery of a wide range of RNA therapeutics to efficiently knock down disease-causing genes. Our fast-growing RNAi portfolio covers a broad range of therapeutic areas including cardiovascular and metabolic diseases, immunology-related diseases, and nervous system diseases. Among them, our first RNAi drug has entered the clinical stage, and several pipelines are in progress simultaneously. SanegeneBio is committed to creating transformational RNAi medicines through striving for scientific innovation to address unmet medical needs worldwide and change the lives of patients and families.

Sapu Bioscience is an immuno-oncology company dedicated to the development of first in class RNA therapeutics as well as small molecule drugs against cancer and infectious diseases. OT-101, the lead immuno-oncology drug candidate of Sapu Bioscience, is a first-in-class anti-TGF-βRNA therapeutic that exhibited single agent activity in relapsed/refractory cancer patients in clinical trial settings. OT-101 also has activity against SARS-CoV-2. Sapu Bioscience is seeking to leverage its deep expertise in drug development to improve treatment outcomes and survival of cancer patients with a special emphasis on rare pediatric cancers. Sapu Bioscience has rare pediatric designation for DIPG (OT-101).

Scipher Medicine is building the future of patient treatment. Most patients who are prescribed blockbuster therapies today don’t respond to the treatment, costing the health care industry billions in wasted drugs while patients continue to suffer. Scipher's platform identifies which drug will work based on the patient's fundamental disease biology, and not based on symptoms, disease classification, or medical bias. A simple test predicts which drug he/she will respond to, ensuring that most optimal treatment is prescribed from day one. The molecular data generated by our tests is then used to fuel novel target discovery to address a clear unmet medical need in patients who do not respond to any existing therapy.

At Sensible Biotechnologies, we are turning living cells into cost-efficient factories for manufacturing high-quality mRNA to unlock the full potential of mRNA therapeutics and vaccines. 

SeqLL is a biotechnology company that specializes in Quantitative RNA & Specialty DNA Sequencing, offering True Single Molecule Sequencing technology.

Shape Therapeutics is a biotechnology company that combines breakthroughs in AI, RNA technology, and synthetic biology to create safe, effective, and accessible medicines to help as many patients as possible. The company was founded with the mission to make programmable RNA medicines available for everyone. Shape Therapeutics is pioneering the field of programmable RNA Medicines.

Silexion Therapeutics is a pioneering clinical-stage oncology-focused biotechnology company engaged in discovering and developing proprietary treatments for KRAS-driven cancers. The company's platform technology is addressing difficult-to-treat solid tumors. Silexion's lead product candidate, SIL-204, consists of locally administered small interfering RNAs, or siRNA, in an extended-release formulation, as a first-line treatment of locally advanced pancreatic cancer patients combined with standard-of-care chemotherapy. SIL-204 is a siRNA product candidate following Phase 1 and Phase 2 clinical trials with Silexion's first-generation siG12D-LODER, also referred to as LODER™. Results from the LODER™ Phase 2 clinical trial showed a trend for differences between treatment groups with the LODER arm suggesting an overall survival advantage of 9.3 months. Silexion commitment to Pushing the boundaries of therapeutic advancements in the field of oncology is focused on its second-generation microparticle delivery system, SIL-204 a newly formulated product that will improve clinical efficacy. Silexion plans to conduct a Phase 2/3 trial of SIL-204 in locally advanced pancreatic cancer patients that harbor the KRAS G12D and G12V mutations. Silexion Therapeutics Ltd. is headquartered in Israel and partnered with well known GMP compliant siRNA facility, LGC Axolabs group located in Petaluma California, and worldwide experts extended-release formulation facility, EVONIK Corporation located in Birmingham, Alabama to upscale manufacturing.

Sireau Labs is a new biotech based in Cambridge, UK, that is using mRNA technology to develop ground-breaking treatments for rare genetic diseases. Sireau Labs is engineering solutions for rare diseases with science that is at the forefront of precision medicine. We are in a daily race to save lives stolen or impacted by rare disease.

Sirius is developing transformative siRNA therapeutics for patients with chronic diseases globally. Founded in 2021, Sirius established an innovation center in the United States and a translational medicine center in China dedicated to state-of-the-art solutions for the treatment and management of chronic diseases.

siRNAgen Therapeutics is a next-generation RNAi platform products company focused on immunology and central nervous system (CNS) diseases. Their proprietary SAMiRNA™ platform leverages modular chemistry to overcome the challenges around delivery. SAMiRNA™ platform has been instrumental in the creation of innovative drugs such as SRN-001, which holds immense promise in treating fibrotic diseases globally. With a firm commitment to innovation and collaboration, siRNAgen continues to push the boundaries of scientific discovery to change the lives of patients worldwide.

We are an RNA delivery and therapeutics company. Our proprietary Bio-Courier® technology addresses the limitations of other RNA delivery technologies through the hybridization of organic materials with inorganic bioabsorbable silicon. Our Bio-Courier portfolio includes silicon stabilized hybrid lipid nanoparticles (sshLNP) that improve the stability, safety, and transfection efficiency of RNA. We use sshLNP to develop a pipeline of RNA therapeutics for rare genetic disorders and are maximizing the potential of our technology through research partnerships. Led by founder and leading biomaterials specialist Dr Suzanne Saffie-Siebert, SiSaf is a venture capital backed private company. Headquartered in Guildford, UK, we have fully integrated research labs and bio-analytical facilities, a scalable technology to fast-track development, and a growing patent estate with extensive freedom to operate.

We drive RNA research with tailored molecular tools. We are from Munich and provide researchers with highly complex & advanced genetic tools. Our core expertise is RNA interference, bioinformatics, and RNA research tools for RNA-Seq and Ribo-Seq. Distinguished by their exceptional targeting specificity and efficiency, our gene function analysis tools include - siPOOLTM for reliable gene silencing, raPOOLTM for targeted RNA affinity purification and riboPOOLTM for efficient ribosomal RNA depletion. Our technology combines high complexity oligo pooling (“Pack Hunter” approach), proprietary design algorithms, and quality production to achieve highly precise and rapid results. siTOOL’s reagents are used by Pharma for target discovery and validation and are mentioned in a growing list of academic publications. Comprised entirely of scientists, siTOOLs provides outstanding technical support and offers bespoke experimental and bioinformatic services, making it a favoured research partner for accelerating scientific discoveries and drug development.

SKIP Therapeutics develops RNA-based therapies for rare genetic disorders and common diseases – utilizing a proprietary computational discovery engine to optimize target selection and therapeutic design. We employ sequence-based molecules, called Antisense Oligonucleotides (ASO), to restore function of mutated genes in rare genetic disorders or to modify functionality of key genes in major disease pathways. Convergence of SKIP’s novel computational discovery engine alongside strong experimental capabilities form a unique venture in the landscape of RNA-based therapies. Thousands of rare genetic disorders affect millions of people worldwide, however the small number of patients for each disease, further segregated by patient-specific mutations, has hindered development of dedicated treatments. Recent advancements in RNA-therapeutics offer an expanded molecular toolkit that can be deployed rapidly and at low costs to restore protein function of mutated genes. Identifying sub patient-populations that could potentially be suitable for treatment with RNA-based therapeutics and matching molecular treatment-strategies across the vast landscape of known disease-causing mutations could be assisted by computational methods. To facilitate development of dedicated treatments, Skip Therapeutics has developed a bioinformatic pipeline that analyzes disease-causing mutations and recognizes the best molecular strategy (if any) to restore protein function, using splice modulating antisense oligonucleotides (ASOs). Analyzing mutation data at scale, enables identification of treatable patient cohorts and optimizes target selection. Top candidates from the computational analyses are experimentally validated and pursued for therapeutic development.

The Integrated Genomics Operation (IGO) at Memorial Sloan Kettering (MSK) is a collaborative core facility dedicated to generating high-quality genomics data for basic, translational, and clinical research projects. The core is focused on Illumina Next-Generation Sequencing (NGS) while also providing comprehensive genomics services including pathological review of tissue samples, fragment analysis, Sanger sequencing, and much more.

SomaGenics is a privately held biotech company with offices and laboratories located in Santa Cruz, CA. We specialize in small RNA technologies, including innovative RNAi-based therapeutics using sshRNA molecules, and diagnostics/biomarker detection (NGS, quantitative PCR) of small or fragmented RNA, including microRNA.

Starna Therapeutics focuses on extrahepatic targeted delivery technology and is committed to developing innovative drugs with RNA as the core to solve unmet clinical needs. The core team comes from well-known pharmaceutical companies, universities and research institutes. The team has profound capabilities in nucleic acid drug discovery, delivery technology and formulation development.

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases. The company is pursuing an entirely new approach to treating severe diseases. We are initially focused on diseases of the central nervous system and the eye, with an emphasis on haploinsufficiencies, which are diseases caused by a loss of about half of the normal protein function. Rather than replace, repair or edit faulty genes, we aim to increase – or stoke – protein output from healthy genes to restore functional protein levels using our proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform. In all, Stoke has identified ~6500 genes with a TANGO signature, representing broad potential for our approach to help people with severe diseases. The early results have been promising. Stoke’s first medicine in development, STK-001 has demonstrated positive Phase 1/2 results for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy that is associated with a number of debilitating non-seizure comorbidities. Behind that, is STK-002, a potential treatment for autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Clinical studies of STK-002 are anticipated to start in 2024. The company is pursuing potential treatments for three additional neurodevelopmental disorders, including Syngap1 and Rett syndrome, in collaboration with Acadia Pharmaceuticals. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts.

Established in 2007, Suzhou Ribo Life Science Co., Ltd. (Ribo) is a clinical stage company devoted to the development of innovative RNAi technologies and oligonucleotide therapeutics as an effort to address unmet clinical needs in the world with the tools, capacity and RNA therapeutics that we build up in China. Fueled by our mission to make China's oligonucleotide therapeutics serve the world, we seek. Based on its cutting edge RNAi technologies, Ribo has established oligonucleotide therapeutic platform that vertically integrates the complete set of technologies to support the full life cycle of oligonucleotide therapeutics from early R&D to commercialization.

Switch Therapeutics is an emerging preclinical stage biotechnology company pioneering a new type of medicine that integrates nucleic acid nanotechnology and RNA interference (RNAi) science with the goal of treating a range of diseases – affecting the central nervous system and systemic indications – with significant unmet needs. Switch’s novel gene knockdown approach is based on technology developed by renowned researchers in the field of RNA from Caltech, Harvard and City of Hope. Based on the scientific discovery of Switch’s co-founders, the company has developed a novel proprietary platform known as CASi (Conditionally Activated siRNAs) that combines advantageous properties of both single and double-stranded RNAs in a single molecule, allowing for cell selective RNAi activity. To date, Switch has raised $52M in funding through Series A, and the company’s South San Francisco based team has continued to grow as its research has advanced.

Sylentis is an European biotechnological company founded in 2006. Sylentis focuses on researching new therapeutic approaches based on gene silencing. Our company is specialised in developing ophtalmologic therapies via interference RNA, a powerful tool for rational drug design. Most advanced product of the company are treatments for glaucoma and dry eye syndrome.

SynerK's founders are RNA-targeted therapy industry veterans with decades of drug development experience. Our expertise covers from discovery, preclinical and clinical development to commercial launch. Millions suffer from life threatening diseases, yet only few benefits from current treatment. There is so much we understood but also much more to understand. We strive to find a way to treat disease where a genetic defect is present. One patient at a time, the number will add up.

SYTE.bio is a Synthetic Biology Start-Up company developing precision DNA & RNA-based therapeutics and vaccines. The company has been incorporated as a Delaware C Corp in 2021 (SYTE.bio, Inc), and has labs & offices in Argentina's headquarter, that has been operating since 2017. SYTE.bio is pioneering the development of life-changing treatments for severe diseases and vaccines to treat and prevent diseases at the molecular and cellular level using engineered DNA, improving life through innovation. SYTE.bio’s powerful redosable DNA & RNA delivery platforms will unleash the potential of personalized genetic medicine in multiple potential applications, such as Gene Therapy (Oncology, Monogenic Diseases, Cardiovascular Diseases, Vaccines, Orphan Diseases), Cell Therapy (CAR-T Therapy, Induced Pluripotent Stem Cells (iPSC)) and Gene Editing (CRISPR/Cas, TALEN, ZFN).

Takara Bio USA, Inc. (formerly Clontech Laboratories, Inc.), a Takara Bio company, develops, manufactures, and distributes a wide range of life science reagents under the Takara®, Cellartis®, and Clontech® brands. Our products support applications including stem cell research; NGS; gene discovery, regulation, and function studies; protein expression and purification; RNAi and gene editing; and plant and food research.

At TAmiRNA we drive innovation through microRNA and RNAi technology to solve unmet clinical needs. Circulating microRNAs are novel minimal-invasive biomarkers that fulfil the requirements of valuable markers. TAmiRNA excels in the identification and development of microRNA biomarkers for diagnosis and prediction of disease and treatment response with a special focus on musculoskeletal, cardiovascular and metabolic diseases. TAmiRNA is a biotech R&D company based in Vienna, Austria. ---- Privacy Policy and Imprint can be accessed by clicking "Contact us" ----

Employs an iterative biophysics and computational engine that addresses the challenges and limitations that have historically hampered efforts to drug RNA. Utilizes NMR (nuclear magnetic resonance spectroscopy)-derived ligand binding data to guide molecular dynamics simulations and build accurate structural models.

Pioneering tRNA therapeutics to modulate mRNA function and cure a broad range of genetic diseases.

Thera Neuropharmainvestigates the use of small molecules and RNA-based compounds as therapeutics to treat Amyotrophic Lateral Sclerosis (ALS)andTraumatic Brain Injury (TBI).

About Tiba Biotech Tiba Biotech is a pre-clinical stage biotechnology company revolutionizing the design and delivery of a safer, more effective and affordable generation of nucleic acid products for human and animal health. Tiba’s innovative RNA vaccine platform disrupts existing design, delivery and bio-manufacturing processes while enabling the rapid development of highly effective vaccines against multiple diseases. Privately held, Tiba is headquartered in Cambridge, Massachusetts, emanated from the Massachusetts Institute of Technology’s Koch Institute, and has strategic partnerships with the U.S. Army Medical Research Institute of Infectious Diseases and a growing list of leading research institutions. To learn more about Tiba, visit www.tiba.bio.

More specifically, Trajectum Pharma is developing novel mRNA-based products, formulated as lipid nanoparticles for the field of rheumatoid arthritis and other inflammatory diseases. Trajectum Pharma is developing a potentially curative, first in class treatment, for which initial proof-of-concept for the treatment of RA has been established.The initial results in preclinical disease models are extremely promising. The long-term vision of the company is to develop an off-the-shelf vaccine for RA, using the principle of tolerance induction. The company has strong ties with Utrecht University, Department of Immunology and Infectious Diseases, Faculty of Veterinary Medicine (Prof. Femke Broere). There is an ongoing co-operation in the field of mRNA technology and lipid nanoparticle formulations with the Weissman Lab at UPenn (Philadephia, PA) (Prof. Drew Weissman).

TransCode's lofty goal is delivering a cancer-free future for all cancer patients. Our core belief is that cancer can be overcome through the intelligent design and efficient delivery of targeted therapeutics. Our lead therapeutic candidate, TTX-MC138, targets microRNA-10b, or miRNA-10b, considered the master regulator of metastatic cell viability in a range of cancers, including breast, pancreatic, ovarian, colon cancer, glioblastomas, and several others. Our other preclinical programs include two solid tumor programs, TTX-siPDL1, an siRNA-based modulator of programmed death-ligand 1, or PD-L1, and TTX-siLIN28B, an siRNA-based inhibitor of RNA-binding protein LIN28B. TransCode also has three cancer agnostic programs, TTX-RIGA, an RNA- based agonist of the retinoic acid-inducible gene I, or RIG-I, targeting activation of innate immunity in the tumor microenvironment; TTX-CRISPR, a CRISPR/Cas9-based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells; and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines that activate cytotoxic immune responses against tumor cells. Until now, targeting biomarkers that are the primary drivers of cancer onset, progression and recurrence have been locked behind the challenge of delivery – cytosolic delivery to engage these known targets. TransCode believes it has overcome this barrier, opening the door to a broad array of cancer therapies -- a disease with the highest unmet need that affects everyone. It is through the design and optimization of this technology that TransCode has devised an elegant solution -- TTX -- a proprietary delivery platform for the transport of targeted therapeutics to cancer cells. With their world class team and know how, TransCode has a rapidly expanding platform of drug candidates designed to target a variety of tumor indications with the goal of long-term treatment survival.

Translation-X is a spin-off project at the Biozentrum, University of Basel. We are developing novel pharmacological tools to reduce core symptoms of autism by targeting RNA translational machinery.

We are a biotechnology company focused on restoring cells’ youthful vigor to repair damage caused by the aging process. Using a proprietary ERA™ Platform, we develop mRNA medicines that are specially formulated to instruct specific cells in the body to fight disease or repair damaged tissue. We do this by reprogramming the epigenome to restore cell function that people often lose as they age. This approach can be used to revitalize different cells throughout the body – we are researching its applicability across multiple therapeutic areas, including dermatologic conditions, immunology, osteoarthritis and ophthalmology.

Our Therapies Restore Balance. We are explorers leading the revolution in RNA drug discovery to develop an entirely new class of therapies. We are identifying the chemical motifs to potently and selectively modulate RNA regulatory networks to treat the most challenging diseases.

Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community

VaxEquity is a biotechnology company amplifying the impact of RNA vaccines and therapeutics, through using its next generation self-amplifying RNA (saRNA) platform. The Company uses saRNA to provide greatly enhanced protein expression, combining better tolerability with slower release and thermostability. VaxEquity is pioneering a next generation approach by modifying its saRNA to modulate the immune response and thereby overcoming current limitations. The flexibility of the Company’s platform ensures that its broad pipeline of assets can be designed, developed, and manufactured rapidly and at scale. VaxEquity was founded in 2020 by Professor Robin Shattock, the Head of Immunology of Infection at Imperial College London and is led by a world class team who are pioneers in the field of RNA.

Vernagen aims to discover and develop mRNA based therapeutics using highly advanced mRNA technologies. Our current pipeline includes vaccine platforms targeting various emerging pathogens and biologics platforms aiming to replace current protein based therapeutics. Our mRNA platforms employ multiple innovative chemical and biological components to enhance their therapeutic efficacy and safety.

Versameb AG is a privately held biotechnology company focusing on discovering and developing innovative RNA-based drugs for modulation of protein expression, including the ability to simultaneously influence several therapeutic targets, in a controlled manner, with one molecular construct, and cellular targeting. Based in Basel and fully operational from 2018, the company is led by an experienced scientific and leadership team with proven expertise in drug discovery and development from lab bench to patient. Versameb’s proprietary technology platform, VERSagile, optimizes the application of functional RNA in different disease contexts – making RNA druggable in new therapeutic areas others have been unable to solve. The pipeline includes lead candidate programs in Stress Urinary Incontinence (SUI) and solid tumors. Versameb is working towards the completion of a first in-human/proof-of-concept clinical study while advancing its platform.

Vir Biotechnology, Inc. (in Europe, Humabs BioMed, https://bit.ly/46lQGN2) is an immunology company focused on powering the immune system to address unmet patient needs in infectious disease and beyond. Our powerful R&D engine combines our proprietary monoclonal antibody (mAb) platform with machine learning and artificial intelligence-enhanced capabilities, enabling us to engineer our rich database of human antibodies and deliver transformative medicines for diseases with significant global burden. Our current pipeline consists of multiple clinical candidates targeting hepatitis B and hepatitis delta viruses and human immunodeficiency virus, including a potential functional cure for chronic hepatitis B and treatment for chronic hepatitis delta. Our portfolio of broad, potent investigational monoclonal antibodies also targets influenza A and B viruses, COVID-19, respiratory syncytial virus/human metapneumovirus and human papillomavirus, and our investigational T cell vaccine aims to prevent HIV. Explore our full pipeline: https://bit.ly/3tr7FPm We collaborate with some of the world’s leaders in industry as well as government agencies and non-profit organizations enabling us to rapidly advance cutting-edge scientific discoveries and innovative technologies with the potential to benefit people around the world. We are proud to have been ranked the fastest-growing company in North America on the 2022 Deloitte Technology Fast 500™. Join us as we pursue our mission to transform lives. It has come to our attention that there have been various recruitment scams targeting job seekers on LinkedIn. Vir Biotechnology, Inc. is committed to your privacy and will never request sensitive personal information including banking details through email, text message, social media or video software platforms. We also will never request a financial commitment from a candidate as part of the application process. Learn more about Vir’s recruiting process: https://bit.ly/461EgJN

Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting two global Phase 3 clinical trials (THRIVE and THRIVE-2) to evaluate the safety and efficacy of VRDN-001 in patients with active and chronic TED. Viridian’s goal is to advance VRDN-001 as a potential best-in-class intravenous therapy followed by VRDN-003 as a potential first- and best-in-class subcutaneous therapy for the treatment of TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases.

We provide innovative gene editing reagents, screening services and engineered model systems for the global R&D market. Our success is based on the proprietary 3Cs technology.

We are developing a platform for discovery of RNA-targeting small molecules to treat genetic diseases, neurodegeneration and cancer. We combine a proprietary high-throughput screening platform with machine learning models and unique datasets to accelerate development of drugs for previously undruggable targets. https://wayfinderbio.com

At the Wyss Institute, we leverage recent insights into how Nature builds, controls and manufactures to develop new engineering innovations - a new field of research we call Biologically Inspired Engineering. Our scientists, engineers and clinicians, who come from Harvard's Schools of Medicine, Engineering, Arts & Sciences, Design, and Education as well as 12 collaborating academic institutions and hospitals, work alongside staff with industrial experience in product development to engineer transformative solutions to some of the world’s greatest problems. By emulating biological principles of self assembly, organization and regulation, we are developing disruptive technology solutions for healthcare, energy, architecture, robotics, and manufacturing, which are translated into commercial products and therapies through formation of new startups and corporate alliances.

Single-cell and Bulk RNA-sequencing services.

La Laiterie du Berger est une entreprise familiale sénégalaise, créée en 2006. Notre mission est de collecter le lait frais des éleveurs de notre pays, pour offrir chaque jour aux consommateurs sénégalais des produits laitiers, au lait local et naturel, connus sous la marque Dolima. C’est ce qui fait que la Laiterie du Berger n’est pas une entreprise comme les autres, et que le yaourt Dolima n’est pas un yaourt comme les autres. Notre engagement s’incarne dans notre mission sociale : construire une filière sénégalaise plus forte, avec un élevage plus productif, en mesure d’approvisionner le marché local. Il s’agit aussi de plus en plus d’aller vers la nutrition, pour apporter la santé par une alimentation saine et accessible dans notre pays où cet enjeu est si grand.

Ziphius Vaccines is a biopharmaceutical company, dedicated to develop saRNA vaccines and therapeutics. Ziphius focuses on both vaccines for infectious diseases and gene supplementation therapies for rare genetic disorders. Since our founding in 2019, Ziphius has worked to develop and validate its self-amplifying RNA and LNP platforms. Currently, these platforms are being used to rapidly expand our pipeline and move forward multiple preclinical development programs towards the clinic.