List of RNA Therapies Companies in United States - 108
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4D Molecular Therapeutics 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. |
ADARx Pharmaceuticals 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. |
Ainos 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. |
AIRNA 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+. |
Alida Biosciences 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. |
Aligos Therapeutics Inc. 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. |
Alltrna 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. |
Alnylam Pharmaceuticals 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. |
Altogen Labs 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. |
Alys Pharmaceuticals 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. |
Arcturus Therapeutics 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). |
ARQ Genetics Bastrop, Texas, United States | ARQGenetics offers custom-designed gene panels to provide accurate real-time gene expression data. |
Arrowhead Pharmaceuticals 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. |
Ascidian Therapeutics 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. |
Atalanta Therapeutics 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 |
Atomic AI 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. |
Avstera Therapeutics 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. |
Basking Biosciences 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. |
Benitec Biopharma 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. |
Beta Hatch 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. |
Brii Biosciences 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. |
Capsida Biotherapeutics 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. |
Ceria Therapeutics Tucson, Arizona, United States | Ceria Therapeutics develops innovative drug products for inflammatory disorders. We intend to transform lives by eliminating the consequences of inflammation. |
Channavix Therapeutics 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. |
Chimerna Therapeutics 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. |
Chimeron Bio 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. |
![]() CircNova 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. |
Circular Genomics 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 |
City Therapeutics 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. |
Comanche Biopharma 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. |
DiamiR Monmouth Junction, New Jersey, United States | DiamiR developsand diagnostictechnology based on analysis of brain-enriched microRNA biomarkers in blood plasma. |
DxTerity Diagnostics Rancho Dominguez, California, United States | 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. |
Eclipse Bio 5770 Oberlin Dr, San Diego, California 92121, US | 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. |
Eli Lilly & Company Indianapolis, Indiana, United States | 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. |
Envisagenics New York, New York, United States | 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. |
Esperovax Plymouth, United States | Fighting the world’s most devastating diseases through the innovative power of targeted oral mRNA vaccines. |
Eterna Therapeutics Cambridge, Massachusetts, United States | 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. |
Exai Bio 3350 W Bayshore Rd, Palo Alto, California 94303, US | 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 San Francisco, CA | 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 San Francisco, California, United States of America | 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. |
Fluent BioSciences Watertown, Massachusetts, United States | 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 2220 Welsch Industrial Ct, St. Louis, MO 63146, US | 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. |
Gradalis Carrollton, Texas, United States | 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. |
Halo-Bio RNAi Therapeutics, Inc. Seattle, Washington, United States | Multivalent RNAi Platform, Delivery |
hC Bioscience Cambridge, Massachusetts, United States | 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. |
Honeycomb Biotechnologies Waltham, Massachusetts, United States | 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. |
Innovac Therapeutics Cambridge, Massachusetts, United States | Innovac Therapeutics is a biotechnology company dedicated to developing innovative vaccines for patients with unmet medical needs, using our proprietary RNA technology. |
Judo Bio 300 Technology Square, 8th Floor, Cambridge, MA 02139 | 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 135 mississippi street, san francisco, california, united states | 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 San Francisco, California, United States | Kerna Labs is unlocking the full potential of mRNA as the universal toolkit for genetic medicine. |
Leal Therapeutics New York, New York, United States | Leal Therapeutics is a biopharmaceutical company that focuses on developing novel therapeutics for patients with major disorders of the central nervous system. |
MalarVX Seattle, Washington, United States | 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. |
Marshall University Genomics Core Facility Huntington, West Virginia, United States | 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 |
miRecule Gaithersburg, Maryland, United States of America | 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. |
Mirimus 760 Parkside Ave, Brooklyn, New York 11226, US | 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. |
Mitotherapeutix Farmington, Connecticut, United States | 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. |
Moderna Cambridge, Massachusetts, United States | 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. |
NextRNA Therapeutics Boston, Massachusetts, United States | 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 11558 Sorrento Valley Road, Suite 3, San Diego, CA 92121, United States | 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. |
Nutcracker Emeryville, California, United States | 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. |
Oncovir Washington, District of Columbia, United States of America | 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 Cambridge, Massachusetts, United States | 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 Cambridge, Massachusetts, United States | 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 |
PDx Pharmaceuticals Portland, Oregon, United States | 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 6150 Lusk Blvd Ste. B100, Suite 160, San Diego, CA 92121, US | 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 Marlborough, Massachusetts, United States | 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 |
Phylex Biosciences Del Mar,USA | Neutralizing Antibody Discovery |
Pinion Immunotherapeutics 12358 Parklawn Dr, Suite 230, Rockville, Maryland 20852, US | 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. |
Prolifagen Therapeutics Villanova, Pennsylvania, United States | 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. |
Radar Therapeutics Berkeley, California, United States | 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 Santa Cruz, California, United States | 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. |
Regulus Therapeutics San Diego, California, United States | 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 Mountain View, California, United States | 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 San Diego, California, United States of America | 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. |
Ribonova Wynnewood, Pennsylvania, United States | 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. |
RIGImmune Inc. Farmington, Connecticut, United States | 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 433 Temple St, New Haven, CT 06511, United States | 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. |
RNAimmune Gaithersburg, Maryland, United States | 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. |
RosVivo Therapeutics Reno, Nevada, United States | 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 Lubbock, Texas, United States | 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. |
Sanegene Bio Woburn, Massachusetts, United States | 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 Therapeutics Agoura Hills, California, United States | 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 Waltham, Massachusetts, United States | 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. |
Sensible Biotechnologies 2093 philadelphia pike, claymont, delaware, united states | 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 Woburn, Massachusetts, United States | SeqLL is a biotechnology company that specializes in Quantitative RNA & Specialty DNA Sequencing, offering True Single Molecule Sequencing technology. |
Shape Therapeutics Seattle, Washington, United States | 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. |
Sirius Therapeutics San Diego, California, United States | 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 Cambridge, Massachusetts, United States | 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. |
Sloan Kettering Integrated Genomics Operation New York, New York, United States | 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 Santa Cruz, California, United States | 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. |
Stoke Therapeutics Bedford, Massachusetts, United States | 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. |
Switch Therapeutics San Francisco, California | 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. |
SynerK Concord, Massachusetts, United States | 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 Boston, Massachusetts, United States of America | 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. Mountain View, California, United States | 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. |
TargetRNA Dracut, Massachusetts, United States | 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. |
Tevard Biosciences Cambridge, Massachusetts, United States | Pioneering tRNA therapeutics to modulate mRNA function and cure a broad range of genetic diseases. |
Thera Neuropharma Berywyn, Pennsylvania, United States | Thera Neuropharmainvestigates the use of small molecules and RNA-based compounds as therapeutics to treat Amyotrophic Lateral Sclerosis (ALS)andTraumatic Brain Injury (TBI). |
Tiba Cambridge, Massachusetts, United States | 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. |
TransCode Therapeutics Newton, Massachusetts, United States | 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. |
Turn Biotechnologies Mountain View, California, United States of America | 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. |
Twentyeight-Seven Therapeutics 490 Arsenal Way, Watertown, Massachusetts, USA, 02472 | 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. |
Vernagen Tucker, Georgia, United States | 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. |
Vir Biotechnology San Francisco, California, United States | 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 Therapeutics Waltham, Massachusetts, United States | 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. |
Wayfinder Biosciences 4000 Mason Rd, Seattle, Washington 98195, US | 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 |
Wyss Institute 30 Dunster St, Cambridge, Massachusetts 02138, US | 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. |
Zata Pharmaceuticals Worcester, Massachusetts, United States | 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. |