Neurogene Announces Upcoming Presentation of Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome at ASGCT Meeting
Neurogene Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Updates
Neurogene Announces Expansion for Enrollment of Rett Syndrome Gene Therapy Trial
Neurogene Announces Appointment of Julie Jordan, M.D., as Chief Medical Officer
Neurogene Announces Business Update and 2024 Outlook
Neurogene Announces Closing of Merger with Neoleukin Therapeutics
Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401
Neurogene has struck deals to fund its pipeline of gene therapies into the second half of 2026, agreeing to merge with the floundering, Nasdaq-listed Neoleukin Therapeutics and persuading investors to pump $95 million into the combined company.
NEW YORK--(BUSINESS WIRE)-- Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for NGN-101. NGN-101, Neurogene’s lead product candidate in its Batten disease franchise, is an investigational adeno-associated virus (AAV) gene therapy for the treatment of CLN5 Batten disease, a rare and fatal neurodegenerative disorder with no approved disease-modifying therapies available.
Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) for patients diagnosed with CLN5, a subtype of Batten disease.