171 RNA Therapies Companies - Worldwide

Emeryville, California, United States of America

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.

Kyoto, Japan

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.

San Diego, California, United States of America

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.

San Diego, California

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.

Cambridge, Massachusetts, United States

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+.

11535 sorrento valley road, san diego, california, united states

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.

South San Francisco, California, United States of America

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.

Cambridge, Massachusetts

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.

Cambridge, Massachusetts, United States of America

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.

Hamilton, Bermuda

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.”

Austin, Texas, United States

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.

Boston, MA, United States

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.

Auckland, New Zealand

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.

Milan, Italy

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

Vancouver, Canada

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.

San Diego, California, United States of America

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).

Shanghai, CN

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.

Bastrop, Texas, United States

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

Pasadena, California, United States of America

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.

Paterna, Valencia, Spain

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

Boston, Massachusetts, United States of America

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.

Boston, Massachusetts, United States

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

South San Francisco, California, United States

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.

Philadelphia, Pennsylvania

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.

Munich, Germany

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/

Columbus, OH

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, China

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.

8th Floor, No. 25, Yayingshi Road, Science City, Luogang District, Guangzhou, CN

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.

Hayward, California, United States

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.

Cashmere, Washington, United States

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.

st. Svyazi, 34 litter A, Strelna, Saint Petersburg, 198515, RU

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.

Köln, Germany

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.

Nantong, China

RNAi

Mainz, Germany

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

Yusong, Taejon-jikhalsi, South Korea

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.

Vancouver, CA

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.

Durham, North Carolina, United States

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, United Kingdom

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

Newbury Park, California, United States of America

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.

Hanover, Germany

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.

Glasgow, Glasgow City, United Kingdom

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.

Tucson, Arizona, United States

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

Pittsford, NY, United States

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.

New York

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.

Philadelphia, Pennsylvania, United States

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

Detroit, Michigan, United States

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

Albuquerque, New Mexico, United States

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

Cambridge, MA

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.

Concord, Massachusetts, United States of America

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.

Tübingen, Baden-Württemberg, Germany

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.