Gene Therapy
Page 1 • 11 itemsUnlock global gene therapy market insights. Stay ahead on regulatory approvals, clinical trials, and investment opportunities in this cutting-edge biotech sector.
CRISPR Therapeutics' CASGEVY Reaches 60,000+ Eligible Patients Across 10 Countries Following Global Approvals
CRISPR Therapeutics reports CASGEVY now approved in 10 countries for severe sickle cell disease and beta thalassemia, reaching over 60,000 eligible patients worldwide.
Restore Vision Reports Positive Safety Data for RV-001 Gene Therapy in Retinitis Pigmentosa Trial
Restore Vision's RV-001 optogenetic gene therapy shows no dose-limiting toxicities in Phase I/II trial for advanced retinitis pigmentosa patients at 168 days.
Expression Therapeutics Receives FDA Fast Track and Rare Pediatric Disease Designations for Hemophilia A Stem Cell Therapy
Expression Therapeutics gains dual FDA designations for investigational autologous stem cell therapy targeting hemophilia A, advancing to Phase 2 trials.
Regeneron Announces Free Gene Therapy Otarmeni for Rare Hearing Loss in U.S. Government Agreement
Regeneron will provide Otarmeni gene therapy for free in the U.S. under new government agreement to lower drug costs for rare genetic hearing loss patients.
Wave Life Sciences WVE-007 Phase 2a Trial Gets FDA Approval for Obesity and Type 2 Diabetes Treatment
Wave Life Sciences receives FDA acceptance for WVE-007 Phase 2a multidose trial targeting obesity and type 2 diabetes, set to begin Q2 2026.
Intellia Therapeutics Submits FDA Application for Lonvo-Z, First CRISPR Gene Therapy for Hereditary Angioedema
Intellia initiates rolling BLA submission for lonvo-z, a potential one-time CRISPR treatment for hereditary angioedema, marking a breakthrough in gene therapy.
Intellia Therapeutics Achieves First-Ever Positive Phase 3 Results for In Vivo Gene Editing with Lonvoguran Ziclumeran in Hereditary Angioedema
Intellia's lonvoguran ziclumeran becomes first in vivo gene editing therapy to succeed in Phase 3, offering potential one-time cure for hereditary angioedema patients.
FDA Approves Otarmeni: First Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program
FDA approves Otarmeni (lunsotogene parvec-cwha), the first dual AAV vector gene therapy for genetic hearing loss under National Priority Voucher Program.
FDA Approves Regeneron's Otarmeni: First Gene Therapy for Genetic Hearing Loss Shows 80% Success Rate
Regeneron's Otarmeni becomes first FDA-approved gene therapy for OTOF-related hearing loss, with 80% of patients meeting primary endpoint and 42% achieving normal hearing.
Medera's AAV-SERCA2a Gene Therapy Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Cardiomyopathy
Medera Inc. receives FDA Fast Track designation for AAV-SERCA2a gene therapy targeting cardiomyopathy in Duchenne muscular dystrophy patients.
MeiraGTx to Present 3-Year AAV-hAQP1 Gene Therapy Data for Radiation-Induced Xerostomia Treatment
MeiraGTx will present 3-year Phase 1 AQUAx trial data for AAV-hAQP1 gene therapy treating radiation-induced xerostomia on April 16, 2026.