MeiraGTx Holdings plc

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.

GenSight Biologics is a clinical-stage biopharmaceutical company that develops gene therapies to treat neurodegenerative retinal diseases and central nervous system disorders. The company focuses on addressing conditions such as Leber Hereditary Optic Neuropathy (LHON), Retinitis Pigmentosa, and Geographic Atrophy using technologies like gene therapy, mitochondrial targeting sequences, and optogenetics.

Prevail Therapeutics Inc.BiotechnologyUnited States

69% matchPrivate

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York City, New York, focused on developing gene therapies for patients with neurodegenerative diseases.

Passage Bio, Inc. is a biopharmaceutical company dedicated to pioneering genetic therapies for conditions affecting the central nervous system. Their advanced pipeline includes PBGM01, designed to treat infantile GM1 gangliosidosis by utilizing a proprietary AAVhu68 capsid to deliver a functional GLB1 gene, which codes for the lysosomal acid beta-galactosidase enzyme, to both the brain and peripheral tissues. Another key program, PBFT02, employs an AAV1 capsid to introduce a functional granulin (GRN) gene, encoding progranulin (PGRN), to the brain as a treatment for FTD-GRN. Additionally, PBKR03 is under development for infantile Krabbe disease, leveraging a proprietary AAVhu68 capsid to deliver a functional GALC gene, responsible for producing the hydrolytic enzyme galactosylceramidase, to the brain and surrounding tissues. The company's portfolio also features PBML04 for metachromatic leukodystrophy, PBAL05 for amyotrophic lateral sclerosis, and PBCM06 for Charcot-Marie-Tooth Type 2A. Passage Bio maintains a significant research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program, alongside a development services and clinical supply agreement with Catalent Maryland, Inc. Established in 2017, the company's headquarters are located in Philadelphia, Pennsylvania.

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.

Coave TherapeuticsBiotechnologyFrance

68% match

Coave Therapeutics is a clinical-stage biotechnology company headquartered in Paris, France. The company focuses on developing life-changing gene therapies targeting rare ocular and central nervous system diseases.