uniQure N.V.

Neurogene Inc. is dedicated to engineering transformative genetic treatments for individuals and their families grappling with debilitating neurological conditions. Its current pipeline prominently features two key investigational therapies: NGN-401, an AAV9 gene therapy specifically targeting Rett syndrome, and NGN-101, which is being developed to address neuronal ceroid lipofuscinosis subtype 5 (Batten disease). The company's operations are based in New York, New York.

Amicus Inc. / DEBiotechnologyUnited States

67% match

Amicus Therapeutics, a subsidiary of BioMarin Pharmaceutical Inc., is a biotechnology company that researches and delivers medicines for individuals living with genetically defined rare diseases. The company provides patient advocacy and support services alongside its clinical development pipeline.

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.

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.

Homology Medicines, Inc.BiotechnologyUnited States

65% matchPrivate

Homology Medicines, Inc. is a clinical-stage genetic medicines company historically focused on transforming the lives of patients with rare diseases through genetic treatments.

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.