Lexeo Therapeutics, Inc.

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.

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.

Taysha Gene Therapies, Inc.TSHA · BiotechDallas, United States

69% matchPublic

Taysha Gene Therapies, Inc. is a biotech firm specializing in the creation and market introduction of gene therapies that utilize adeno-associated virus (AAV) vectors. Its core mission is to tackle inherited diseases affecting the central nervous system (CNS). The company's development pipeline features several key programs: TSHA-120 is aimed at giant axonal neuropathy; TSHA-102 is in development for Rett syndrome; TSHA-121 and TSHA-118 are both being advanced for CLN1 disease; TSHA-105 addresses SLC13A5 Deficiency; and TSHA-101 targets GM2 gangliosidosis. Furthermore, Taysha has forged a strategic alliance with The University of Texas Southwestern Medical Center to jointly advance and bring to market innovative gene therapy solutions. Founded in 2019, the company operates from its headquarters in Dallas, Texas.

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.

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.

Abeona Therapeutics Inc. operates as a clinical-stage biopharmaceutical company, specializing in the creation of gene and cell therapies to combat critical, uncommon genetic illnesses. Their foremost developmental asset is EB-101, a personalized gene-corrected cell therapy that has progressed to Phase III clinical trials for the treatment of recessive dystrophic epidermolysis bullosa. The company's pipeline also encompasses several other programs: ABO-102, an adeno-associated virus (AAV)-based gene therapy designed for Sanfilippo syndrome type A; ABO-201, aimed at CLN3 disease; ABO-401, targeting cystic fibrosis; and ABO-50X, intended for genetic eye disorders. Additionally, Abeona actively advances AAV-based gene therapies through its proprietary AIM vector platform. Incorporated in 1974, the firm was initially named PlasmaTech Biopharmaceuticals, Inc. before officially adopting the name Abeona Therapeutics Inc. in June 2015. The company's corporate base is situated in New York, New York.