Sarepta Therapeutics, Inc. competitors & alternatives

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

71% matchPublic

Similar pharmaceuticals company

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.

Pharmaceuticals, Biotechnology & Life Sciences

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.

Similar pharmaceuticals company

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

Biotech

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.

Biotech

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.

Biotechnology

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.

Biotech

Voyager Therapeutics, Inc. operates as a gene therapy company, concentrating its efforts on developing innovative treatments and pioneering advanced platform technologies. Its leading clinical asset, VY-AADC, is presently undergoing an open-label Phase 1 clinical trial for the management of Parkinson's disease. The company's preclinical portfolio is extensive, featuring VY-SOD102 for amyotrophic lateral sclerosis (ALS), VY-HTT01 for Huntington's disease, and VY-FXN01 for Friedreich's ataxia. Additionally, Voyager is pursuing a Tau program aimed at various tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia, alongside initiatives for spinal muscular atrophy. To advance its gene therapy product pipeline, the company has forged strategic collaboration and licensing agreements with key industry players such as Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G. These partnerships cover the research, development, and commercialization of adeno-associated virus-based gene therapy products. Voyager Therapeutics, Inc. was founded in 2013 and maintains its corporate headquarters in Cambridge, Massachusetts.

Biotech

CRISPR Therapeutics AG (CRSP) is a biotechnology firm dedicated to pioneering gene-based medicines for severe diseases. The company achieves this through its exclusive Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology, which enables precise and targeted alterations to an organism's genetic code. Its extensive pipeline includes therapeutic candidates spanning multiple medical areas, such as blood disorders (hemoglobinopathies), various cancers (oncology), regenerative medicine, and rare conditions. The company's flagship investigational therapy is CTX001, an ex vivo CRISPR gene-edited treatment. This therapy aims to benefit patients with transfusion-dependent beta-thalassemia or severe sickle cell disease by modifying their own hematopoietic stem cells to markedly boost the production of fetal hemoglobin within red blood cells. CRISPR Therapeutics is also advancing several other genetically engineered allogeneic (donor-sourced) CAR-T investigational therapies: CTX110, designed to combat cluster of differentiation 19-positive malignancies; CTX120, which targets B-cell maturation antigen for multiple myeloma that has relapsed or proven resistant to previous treatments; and CTX130, focused on Cluster of Differentiation 70 for a spectrum of solid tumors and blood cancers. Furthermore, the company is developing VCTX210, an immune-evasive, gene-edited stem cell-derived product candidate for treating type 1 diabetes. It is also pursuing various in vivo gene-editing initiatives aimed at addressing disorders affecting the liver, lungs, muscles, and central nervous system. The company has forged strategic alliances with significant partners, including Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was established in 2013 and maintains its headquarters in Zug, Switzerland.

Splice BioBiotechUnited States

69% match

Biotech

SpliceBio is a clinical-stage genetic medicines company that develops gene therapies using a proprietary Protein Splicing platform. The company focuses on treating diseases caused by mutations in large genes, with a lead clinical program targeting Stargardt disease.

Biotechnology

Beam Therapeutics Inc., founded in 2017 and based in Cambridge, Massachusetts, operates as a pioneering biopharmaceutical firm. Its core mission involves engineering precise genetic remedies to tackle a spectrum of severe human ailments, primarily within the United States. The company's developmental portfolio features several key candidates: BEAM-101 is being advanced to treat both sickle cell disease and beta thalassemia. BEAM-102 is specifically designed for addressing sickle cell disease. BEAM-201, an allogeneic chimeric antigen receptor T-cell therapy, is under investigation for individuals suffering from relapsed or refractory T-cell acute lymphoblastic leukemia. BEAM-301 is a liver-targeted candidate aimed at patients afflicted with Glycogen Storage Disease Type Ia. Beyond these flagship programs, Beam Therapeutics is also engaged in devising treatments for alpha-1 antitrypsin deficiency, various ocular conditions, and other disorders impacting the liver, muscular system, and central nervous system. To further its research and development objectives, Beam Therapeutics has forged numerous strategic alliances and partnerships. These include collaborations with Boston Children's Hospital; a research and clinical trial agreement with Magenta Therapeutics, Inc.; a licensing deal with Sana Biotechnology, Inc.; and a research partnership with the Institute of Molecular and Clinical Ophthalmology Basel. Additionally, the company maintains research collaborations with both Pfizer Inc. and Apellis Pharmaceuticals, Inc., alongside a comprehensive collaboration and license agreement with Verve Therapeutics, Inc.

Biotechnology

Rocket Pharmaceuticals, Inc., operating alongside its affiliates, is an innovative biotechnology enterprise dedicated to pioneering gene therapies for rare and severely debilitating diseases. The company's active clinical pipeline features multiple promising programs. It is currently progressing three ex vivo lentiviral vector therapies aimed at specific genetic conditions: Fanconi anemia, characterized by a bone marrow defect that hinders blood cell production; leukocyte adhesion deficiency-I, an inherited disorder resulting in a compromised immune system; and pyruvate kinase deficiency, a rare autosomal recessive red blood cell ailment leading to chronic non-spherocytic hemolytic anemia. Complementing these, Rocket Pharmaceuticals also has an in vivo adeno-associated virus program underway for Danon disease, a serious multi-organ lysosomal storage disorder that tragically often results in early mortality due to heart failure. To facilitate its extensive research and development efforts, the company has established key licensing partnerships with esteemed institutions such as the Fred Hutchinson Cancer Research Center, European research bodies including CIEMAT and its collaborators (Centro de Investigacion Biomedica En Red and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz), UCL Business PLC (in conjunction with CIEMAT), The Regents of the University of California, and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. maintains its primary corporate location in Cranbury, New Jersey.

Biotech

Korro Bio, Inc., a biopharmaceutical firm based in Cambridge, Massachusetts, is dedicated to exploring, advancing, and bringing to market genetic therapeutics. These treatments are formulated through RNA editing technology, aiming to address a wide array of medical conditions, encompassing both uncommon and widespread diseases.