OPEN COMPANY INTELLIGENCEPublicly Listed Biopharma · Companies
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25Wave Life Sciences is a U.S.-domiciled biotechnology company developing therapeutic technologies that act on RNA. Its research includes RNA editing, gene silencing and other oligonucleotide-based approaches designed to alter disease-related biological pathways. The company has interests in rare genetic disorders and selected metabolic and neurologic diseases. Wave is headquartered operationally in Cambridge, Massachusetts, and its common stock trades on Nasdaq under WVE. Following a 2026 redomiciliation, its Delaware parent succeeded an earlier Singapore-incorporated issuer without creating a second independent listing.
26Arrowhead Pharmaceuticals develops RNA interference medicines intended to reduce the production of disease associated proteins. Its research uses delivery technologies designed to bring RNA therapeutics into selected tissues, with programs in cardiometabolic, liver and other serious diseases. The company is headquartered in Pasadena, California, and maintains research operations in Madison, Wisconsin. Arrowhead has both internally developed and partnered programs, so commercial and development rights can vary by medicine. Its investor relations site provides the authoritative current securities information and ongoing company disclosures.
27Dyne Therapeutics is a U.S. clinical-stage biotechnology company developing medicines for genetically driven neuromuscular disorders. The company's scientific approach focuses on delivering therapeutic molecules to muscle and related tissues, including oligonucleotide technologies designed to modify disease-associated processes. It works on rare inherited conditions for which delivery to affected tissue is an important challenge in drug development. Based in Waltham, Massachusetts, Dyne is listed on Nasdaq under DYN. Its therapeutic research spans candidate design, delivery-system optimization and clinical evaluation in defined neuromuscular patient populations. Dyne's development strategy is centered on disease biology and the potential benefits of targeted drug delivery.
28Biocytogen is a Beijing-headquartered biotechnology company developing platforms and therapeutic antibody assets for drug discovery and collaborative development. Its RenMice and related antibody technologies support fully human antibodies, multispecific formats and antibody-drug conjugate programs. The business also supplies research models and preclinical services, but its disclosed agreements include therapeutic antibody co-development and out-licensing, so it should not be categorized as a pure contract research provider. Biocytogen has public share listings on the Shanghai Stock Exchange STAR Market and the Hong Kong Stock Exchange.
29Intellia Therapeutics is a U.S. biotechnology company developing medicines based on genome editing, including CRISPR-based technologies. Its research aims to create genetic interventions for serious diseases, with drug development programs evaluating targeted editing in relevant tissues. The company's therapeutic interests include rare inherited and protein-mediated conditions. Intellia is headquartered in Cambridge, Massachusetts, and its common stock trades on Nasdaq under NTLA. It operates as a research-driven therapeutic developer rather than a contract-service or diagnostic-only company. This entry summarizes enduring modality and disease-area characteristics without claiming that investigational therapies are approved.
30Beam Therapeutics is a United States biotechnology company developing precision genetic medicines using base editing technology. Base editing aims to make targeted changes to DNA without relying on the same double stranded cutting mechanism used by some other gene editing approaches. Its development work includes serious inherited diseases, including conditions affecting blood and liver metabolism. Founded in 2017, Beam has its headquarters in Cambridge, Massachusetts, and manufacturing operations in North Carolina. The company publishes official investor disclosures and clinical updates through its website.
31Legend Biotech is a publicly traded biotechnology company focused on developing and commercializing engineered cell therapies, particularly chimeric antigen receptor T-cell approaches. Its drug development combines cell engineering, translational science, clinical studies and commercial collaborations. The company has a major United States operating presence and a global headquarters in Bridgewater, New Jersey, while its listed issuer has a separate corporate legal structure. Legend trades on Nasdaq under LEGN. Its work centers on cancer therapies, including hematologic malignancies, with additional research into next-generation cell approaches.
32Everest Medicines is a Hong Kong-listed biopharmaceutical company engaged in developing, manufacturing and commercializing innovative medicines. The company works with internally developed capabilities and external partnerships to bring therapies to patients, with activities spanning several disease areas. Its portfolio and development strategy have included immunology, infectious diseases, renal conditions and other therapeutic needs. The official company website identifies its Hong Kong listing and provides investor disclosures alongside business information. Its work connects therapeutic development with manufacturing capabilities and commercial partnerships.
33Amgen is a U.S. biotechnology company that researches, develops, manufactures and commercializes medicines for serious diseases. Its discovery and development work includes biologic medicines, antibodies, other protein-based approaches and selected targeted therapeutics. The company operates across oncology, inflammatory disease, cardiovascular and metabolic disorders, and rare conditions. Headquartered in Thousand Oaks, California, Amgen is listed on Nasdaq. Its integrated capabilities extend from molecular research and translational development to manufacturing and medicine supply. Amgen combines in-house scientific platforms with external research collaborations to pursue treatment approaches in disease areas where substantial patient needs remain.
34BioMarin Pharmaceutical is a U.S. biotechnology company focused on medicines for rare and genetically defined conditions. It develops, manufactures and commercializes therapies informed by genetic and disease biology, including enzyme-based and other targeted treatment approaches. The company is based in San Rafael, California, and its shares are traded on Nasdaq under BMRN. BioMarin's long-term focus includes skeletal and metabolic conditions as well as other rare disorders where specialized drug development and manufacturing capabilities are important.
35Arcus Biosciences is a U.S. clinical-stage biopharmaceutical company developing differentiated drug candidates for cancer and immune-mediated diseases. Its research combines medicinal chemistry, established disease biology and selected antibody-based capabilities, with an emphasis on molecules and treatment combinations that could improve patient outcomes. Arcus is headquartered in Hayward, California, and its shares trade on the New York Stock Exchange under RCUS. The company conducts clinical and discovery-stage research in oncology and has expanded work toward immune diseases. This concise profile emphasizes the issuer's scientific focus and identity.
36Arcutis Biotherapeutics is a U.S. biopharmaceutical company focused on medical dermatology and therapies for inflammatory skin conditions. Its research and commercial activities address immune-driven dermatologic diseases through targeted therapeutic mechanisms and formulation approaches suitable for different patient needs. The company is headquartered in Westlake Village, California, and its stock trades on Nasdaq under ARQT. Arcutis combines drug development, regulatory work and commercialization in dermatology, including approved therapies and investigational uses that may evolve over time.
37Allogene Therapeutics is a U.S. clinical-stage biotechnology company developing allogeneic, or donor-derived, engineered cell therapies. Its AlloCAR T approach aims to develop off-the-shelf cellular medicines, using gene engineering and specialized cell-manufacturing methods. Research focuses on cancer and selected autoimmune diseases, while development programs evaluate the safety and activity of different cellular constructs. The company is based in South San Francisco, California, and its common stock trades on Nasdaq under ALLO. This entry identifies the company's therapeutic modality and disease areas without treating evolving clinical results as permanent facts.
38Agios Pharmaceuticals is a U.S. biopharmaceutical company focused on developing and commercializing medicines for rare blood disorders. Its science builds on cellular metabolism and related disease pathways, seeking targeted therapeutic approaches for inherited hematologic conditions. The company's development activities combine clinical research, regulatory work and commercialization of prescription medicines. Agios is based in Cambridge, Massachusetts, and is listed on Nasdaq under AGIO. This company directory records the issuer's core therapeutic specialization, rather than offering a complete record of individual treatment indications, regulatory decisions or quarterly performance.
39Abeona Therapeutics is a U.S. biotechnology company developing and commercializing genetic medicines for serious diseases with significant unmet medical needs. Its capabilities encompass gene therapy and engineered cell therapy approaches, particularly in rare disorders. The company links translational research, clinical development, specialized production and patient access work to support advanced therapies. Abeona's common stock is listed on Nasdaq under ABEO. Genetic and cellular interventions are central to its technology strategy, distinguishing its drug-development business from diagnostic testing and contract research services. Such treatments require dedicated processes for product characterization, manufacturing, clinical evaluation and patient delivery.
40BioCryst Pharmaceuticals is a U.S. biotechnology company focused on developing and commercializing targeted medicines for rare diseases. Its scientific capabilities emphasize small-molecule discovery and pharmacological approaches selected for specific disease mechanisms. BioCryst is particularly associated with hereditary angioedema and maintains research interests in additional rare conditions. The company operates from Research Triangle Park, North Carolina, and its common shares are listed on Nasdaq under BCRX. BioCryst combines medicinal chemistry and biological research with development and commercialization capabilities. Its focus on rare diseases shapes its approach to therapeutic discovery, clinical development and interactions with specialized patient communities.
41Acrivon Therapeutics is a U.S. biotechnology company developing targeted small-molecule medicines using its predictive precision proteomics approach. Its AP3 platform combines cellular signaling measurements with drug discovery and clinical biomarker strategies. The company has focused primarily on oncology and has expanded its work into immunology and inflammatory diseases. Based in Watertown, Massachusetts, Acrivon is listed on Nasdaq under ACRV. The company's approach seeks to connect drug mechanisms with patient selection and clinical development decisions.
42Axsome Therapeutics is a U.S. biopharmaceutical company developing and commercializing medicines for central nervous system disorders. Its research focuses on pharmacological interventions for neurological and psychiatric conditions, supported by clinical development and commercialization operations. The company's therapeutic interests include disorders affecting mood, cognition, sleep and related neural functions. Axsome is headquartered in New York and its shares are listed on Nasdaq under AXSM. Its work spans the identification of treatment approaches, clinical assessment of therapies and delivery of medicines to patients with neurological or psychiatric needs. This combination of research and commercial capabilities defines its biopharmaceutical operating model.
43Cabaletta Bio is a U.S. biotechnology company developing engineered T-cell therapies for autoimmune diseases. The company's scientific work combines cellular engineering with clinical development intended to alter harmful immune responses. Its research interests span rheumatologic, inflammatory and antibody-mediated autoimmune disorders, rather than concentrating solely on oncology. Cabaletta is headquartered in Philadelphia, Pennsylvania, and its shares trade on Nasdaq under CABA. The technology involves modifying immune cells for therapeutic purposes and assessing their effects in defined patient populations. Cabaletta's focus places it within the broader field of cell-based medicines and immune-system modulation for serious disease.
44Alumis is a U.S. biopharmaceutical company developing targeted medicines for immune-mediated and inflammatory diseases. Its research includes orally administered small-molecule approaches designed to modulate signaling pathways involved in autoimmune conditions. The company pursues therapeutic opportunities in dermatology and systemic immune disorders, supported by translational science and clinical development capabilities. Alumis is based in the San Francisco Bay Area and is listed on Nasdaq under ALMS. The company is classified here by its durable research focus rather than the current phase or outcome of any particular investigational program.
45Alector is a U.S. biotechnology company developing therapeutic approaches for neurodegenerative diseases. Its research combines neuroscience, immune biology and technology intended to improve the delivery of therapeutics to the brain. The company is based in South San Francisco, California, and its common shares trade on Nasdaq under ALEC. Alector's drug discovery activities target biological mechanisms that may contribute to neurodegeneration, and the company works through internal development and selected external collaborations. Its treatment candidates and development priorities can change as research progresses.
46Arbutus Biopharma is a biotechnology company focused on discovering and developing therapeutics for chronic hepatitis B virus infection. The company pursues approaches intended to address viral persistence and improve outcomes for people living with hepatitis B. Its research includes RNA-based methods and other antiviral strategies, and the company collaborates with partners on selected technology and intellectual property programs. Arbutus is incorporated in British Columbia, Canada, while its principal executive offices are in Warminster, Pennsylvania, United States. Its common shares are registered on Nasdaq under ABUS.
47Alto Neuroscience is a U.S. clinical-stage biotechnology company developing precision medicines for neuropsychiatric disorders. Its research strategy uses patient biomarkers and related analytical approaches to identify groups that may respond to particular therapies. The company's therapeutic emphasis includes depression and other disorders of the central nervous system, with development work involving targeted small-molecule candidates. Alto is based in Mountain View, California, and trades on the New York Stock Exchange under ANRO. The company's scientific approach is centered on matching treatments to patient biology rather than relying only on broad diagnostic categories.
48Akeso is a listed Chinese biopharmaceutical company focused on the research, development, manufacturing and commercialization of innovative medicines. Its work includes monoclonal antibodies, bispecific and multispecific antibodies, and other therapeutic platforms. Cancer and immune-mediated diseases are prominent areas of its research. The company operates an integrated model that connects early discovery with clinical development and commercial production. Its official corporate profile identifies the Hong Kong Stock Exchange listing and describes its technology platforms.
Coverage expands from sourced records; this database does not claim all biopharma companies.
