Alnylam Pharmaceuticals

Ionis Pharmaceuticals, Inc.0JDI · PharmaceuticalsCarlsbad, United States

76% matchPublic

Ionis Pharmaceuticals, Inc. is a U.S.-based biotechnology company specializing in the discovery and development of RNA-targeted therapeutic agents. The company's commercialized portfolio features SPINRAZA, a treatment for spinal muscular atrophy (SMA) in both pediatric and adult patients. It also offers TEGSEDI, an injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, and WAYLIVRA, approved for familial chylomicronemia syndrome and familial partial lipodystrophy. Beyond its marketed therapies, Ionis is actively progressing numerous drug candidates through pivotal Phase 3 clinical trials. These late-stage programs include Eplontersen, a monthly self-administered subcutaneous injection targeting all types of TTR amyloidosis; Olezarsen, designed for patients with severe hypertriglyceridemia (SHTG); Donidalorsen, for hereditary angioedema; ION363, aimed at amyotrophic lateral sclerosis; Pelacarsen, for individuals with established cardiovascular disease and elevated lipoprotein(a); and Tofersen, developed to inhibit superoxide dismutase 1 production. Furthermore, Ionis is exploring treatments across a broader spectrum of therapeutic areas, such as metabolic, infectious, renal, ophthalmic diseases, and cancer. The company maintains a significant strategic collaboration with Biogen Inc. and holds various collaboration and licensing agreements with other prominent pharmaceutical partners, including AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc. Founded in 1989, Ionis Pharmaceuticals, Inc. operates from its headquarters in Carlsbad, California.

Sarepta Therapeutics, Inc. operates as a biopharmaceutical firm with products already available on the market. Its primary objective is the research and advancement of genetic treatment modalities, encompassing RNA-targeted therapeutics and gene therapies, specifically for the management of rare diseases. The company's commercial offerings include EXONDYS 51, an injectable medication prescribed for Duchenne muscular dystrophy (DMD) patients whose genetic mutation in the dystrophin gene is responsive to exon 51 skipping. Similarly, VYONDYS 53 is provided for individuals with DMD who can benefit from exon 53 skipping. Sarepta is also actively developing several investigational therapies: AMONDYS 45, a product candidate that utilizes phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to bypass exon 45 of the dystrophin gene. SRP-5051, a peptide-conjugated PMO engineered to bind to exon 51 of dystrophin pre-mRNA. SRP-9001, a gene therapy initiative focused on micro-dystrophin for DMD. SRP-9003, a gene therapy program targeting limb-girdle muscular dystrophies. The company has established collaborative relationships with various partners, including F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio. Sarepta was incorporated in 1980 and maintains its corporate headquarters in Cambridge, Massachusetts.

Enanta Pharmaceuticals, Inc. is a biotechnology firm dedicated to discovering and advancing small molecule therapies designed to combat a range of viral infections and various liver conditions. Its research and development efforts are primarily focused on addressing pathogens such as respiratory syncytial virus (RSV), SARS-CoV-2, human metapneumovirus (hMPV), and the hepatitis B virus (HBV). The company also holds a strategic partnership and licensing agreement with Abbott Laboratories. This collaboration involves the joint identification, development, and market introduction of specific HCV NS3 and NS3/4A protease inhibitor compounds, including known agents like paritaprevir and glecaprevir, which are utilized in the management of chronic hepatitis C virus. Founded in 1995, Enanta Pharmaceuticals, Inc. operates from its principal office located in Watertown, Massachusetts.

Apellis Pharmaceuticals, Inc.PharmaceuticalsUnited States

68% matchPrivate

Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company headquartered in Waltham, Massachusetts. Founded in 2008, the public company discovers, develops, and commercializes targeted therapeutic treatments for autoimmune and inflammatory diseases.

BioCryst Pharmaceuticals, Inc. is a biotechnology company focused on discovering and developing novel, orally administered, small-molecule therapeutics. The company currently markets two key products: Peramivir injection: An intravenous neuraminidase inhibitor, sold under the brand names RAPIVAB, RAPIACTA, and PERAMIFLU, used for the treatment of acute uncomplicated influenza. ORLADEYO: An oral serine protease inhibitor designed to manage hereditary angioedema. BioCryst's development pipeline includes several promising candidates: BCX9930: An oral factor D inhibitor in Phase II clinical trials for complement-mediated diseases. BCX9250: An oral activin receptor-like kinase-2 inhibitor undergoing Phase I clinical evaluation for fibrodysplasia ossificans progressiva. Galidesivir: An RNA dependent-RNA polymerase inhibitor, also in Phase I, aimed at treating a range of RNA viruses, including Marburg, Yellow Fever, Ebola, and Zika. The company engages in strategic collaborations and in-license agreements with numerous partners. These include pharmaceutical entities like Torii Pharmaceutical Co., Ltd., Seqirus UK Limited, Shionogi & Co., Ltd., Green Cross Corporation, and Mundipharma International Holdings Limited. Their alliances also extend to governmental and academic organizations such as the National Institute of Allergy and Infectious Diseases, the Biomedical Advanced Research and Development Authority, the U.S. Department of Health and Human Services, The University of Alabama at Birmingham, Albert Einstein College of Medicine of Yeshiva University, and Industrial Research, Ltd. Established in 1986, BioCryst Pharmaceuticals, Inc. is headquartered in Durham, North Carolina.

Catalyst Pharmaceuticals, Inc., founded in 2002 and based in Coral Gables, Florida, operates as a commercial-stage biopharmaceutical enterprise. This company is dedicated to discovering, developing, and marketing therapeutic solutions for individuals in the United States who are afflicted with uncommon, severe, and long-term neuromuscular and neurological disorders. Among its key offerings is Firdapse, an amifampridine phosphate tablet formulation approved for managing Lambert-Eaton Myasthenic Syndrome (LEMS). Catalyst also provides Ruzurgi, a specific treatment option for pediatric patients diagnosed with LEMS. Beyond its existing products, Catalyst is actively investigating additional applications for Firdapse, including its potential use in treating MuSK antibody positive myasthenia gravis, spinal muscular atrophy type 3, and hereditary neuropathy with liability to pressure palsies. The company engages in various strategic collaborations, such as licensing agreements with BioMarin Pharmaceutical Inc., and a joint development and commercialization agreement with Endo Ventures Limited concerning a generic version of Sabril tablets. Previously known as Catalyst Pharmaceutical Partners, Inc., the company adopted its current name in May 2015.