Sarepta Therapeutics, Inc.

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

71% 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.

PYC Therapeutics Limited, a drug-development company, engages in the discovery and development of novel RNA therapeutics for the treatment of genetic diseases in Australia. The company offers VP-001, a drug program, which is in Phase 1 clinical trial for the treatment of retinitis pigmentosa type 11; PYC-001, a drug candidate for treating blinding eye disease named Autosomal Dominant Optic Atrophy, which is in clinical studies; PYC-002, a drug program for the treatment of severe neurodevelopmental disorder named Phelan-McDermid Syndrome, which is in pre-clinical studies; and PYC-003 to treat autosomal dominant polycystic kidney disease, which is in early-stage clinical trials. PYC Therapeutics Limited has academic-industry collaborations with Murdoch University to support drug discovery and development efforts in the field of neurodegenerative disorders. The company was formerly known as Phylogica Limited and changed its name to PYC Therapeutics Limited in November 2019. PYC Therapeutics Limited was incorporated in 2001 and is based in Nedlands, Australia.

Black Diamond Therapeutics, Inc. is a biotechnology enterprise dedicated to discovering, advancing, and bringing to market therapeutic agents specifically designed for patients with genetically characterized tumors. A core part of its pipeline is BDTX-189, an irreversible small molecule inhibitor. This compound is engineered to neutralize oncogenic proteins driven by non-canonical epidermal growth factor receptor (EGFR) and human epidermal growth factor receptor 2 (HER2) driver mutations. The firm is also progressing BDTX-1535, a brain-penetrant inhibitor addressing a range of EGFR mutations, including conventional, inherent resistance, and acquired resistance forms. Concurrently, it is advancing BDTX-4933, another brain-penetrant inhibitor targeting oncogenic BRAF alterations across Class I, II, and III categories. Black Diamond maintains a strategic alliance with OpenEye Scientific Software, Inc. Established in 2014, the company was initially known as ASET Therapeutics, Inc. before adopting its current name, Black Diamond Therapeutics, Inc., in January 2018. Its corporate headquarters are situated in Cambridge, Massachusetts.

Solid Biosciences Inc.SLDB · BiotechCharlestown, United States

77% matchPublic

Solid Biosciences Inc. is an American biotechnology firm dedicated to creating therapeutic solutions for Duchenne Muscular Dystrophy (DMD). Its most advanced experimental drug, SGT-001, is a gene transfer treatment currently in Phase I/II clinical trials, designed to encourage the production of functional dystrophin protein in patients' muscles. The company is also progressing with SGT-003, which represents a next-generation gene transfer therapy intended for DMD. Beyond specific drug candidates, Solid Biosciences is actively developing innovative platform technologies, such as "dual gene expression," a technique that allows for multiple therapeutic genes to be bundled into a single viral vector, and researching new capsid designs. The company maintains a strategic alliance through a collaboration and license agreement with Ultragenyx Pharmaceutical Inc., focusing on the joint development and market introduction of novel gene therapies for Duchenne Muscular Dystrophy. Founded in 2013, Solid Biosciences Inc. conducts its operations from its headquarters in Cambridge, Massachusetts.

Dyne Therapeutics, Inc. is a U.S.-based biotechnology firm focused on developing advanced treatments for genetically driven muscle disorders. Utilizing its proprietary FORCE platform, which is engineered to deliver disease-modifying therapeutics, the company is actively engaged in various programs. These initiatives target conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral dystrophy, in addition to other rare skeletal, cardiac, and metabolic muscle diseases. The company was founded in 2017 and maintains its corporate headquarters in Waltham, Massachusetts.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company that engineers therapeutic agents by leveraging its proprietary adeno-associated virus (AAV) vector technology. The company's developmental efforts are concentrated across a trio of therapeutic fields: ophthalmology (eye conditions), cardiology (heart ailments), and pulmonology (respiratory disorders). Within its active pipeline, three specific product candidates have advanced into clinical trial phases, all currently undergoing Phase 1/2 clinical assessment: 4D-125 is being evaluated for the treatment of X-linked retinitis pigmentosa. 4D-110 targets choroideremia. 4D-310 is focused on addressing Fabry disease. Beyond these, two Investigational New Drug (IND) candidates are in development: 4D-150, aimed at combating wet age-related macular degeneration, and 4D-710, designed to tackle cystic fibrosis lung disease. To advance its mission, 4D Molecular Therapeutics, Inc. actively engages in research and development partnerships with entities such as uniQure, CRF, Roche, and CFF. The firm commenced operations in 2013 and maintains its headquarters in Emeryville, California.