4D Molecular Therapeutics, Inc.

REGENXBIO Inc. is a biotechnology company operating in the clinical stage, dedicated to developing gene therapy candidates. These therapies are designed to introduce genes into cells, aiming either to rectify genetic deficiencies or to stimulate the body's own cells to produce therapeutic proteins or antibodies to combat diseases. Central to its therapeutic development is the proprietary NAV Technology Platform, an adeno-associated virus-based gene delivery system. The company's primary investigational asset, RGX-314, is currently in Phase III clinical trials for treating wet age-related macular degeneration. Its pipeline also includes RGX-121 and RGX-111, both in Phase I/II trials for mucopolysaccharidosis type II and type I, respectively; RGX-181, which is in preclinical development for late-infantile neuronal ceroid lipofuscinosis type II disease; RGX-202, undergoing Phase I/II evaluation for Duchenne muscular dystrophy; and RGX-381, at the preclinical stage for addressing the ocular manifestations of CLN2 disease. In addition to its internal programs, REGENXBIO Inc. licenses its NAV Technology Platform to other biotech and pharmaceutical enterprises. It also maintains a collaborative and licensing agreement with Neurimmune AG for the advancement of innovative gene therapies. Founded in 2008, REGENXBIO Inc. maintains its headquarters in Rockville, Maryland.

Adverum BiotechnologiesBiotechUnited States

70% match

Adverum Biotechnologies is a clinical-stage biotechnology research company headquartered in Redwood City, California. The company focuses on developing novel gene therapies designed to redefine ocular care and treat severe eye diseases.

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.

Solid Biosciences Inc.SLDB · BiotechCharlestown, United States

70% 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.

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.

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.