Gene Therapy
Page 1 • 12 itemsUnlock global gene therapy market insights. Stay ahead on regulatory approvals, clinical trials, and investment opportunities in this cutting-edge biotech sector.
CRISPR Therapeutics Reports Q1 2026 Results as CASGEVY Gene Therapy Gains Global Regulatory Approval
CRISPR Therapeutics reports Q1 2026 financial results highlighting CASGEVY's approval in 10+ countries for sickle cell disease and beta thalassemia treatment.
Krystal Biotech Reports Strong Q1 2026 Results with VYJUVEK Global Expansion and Two Registrational Studies Pending
Krystal Biotech announces Q1 2026 results highlighting VYJUVEK global expansion and two upcoming registrational study readouts for gene therapy pipeline.
Vertex CASGEVY Gene Therapy Reaches 60,000+ Eligible Patients Across 10 Countries as Q1 2026 Results Show Global Expansion
Vertex's CASGEVY, the first approved CRISPR gene therapy, now serves 60,000+ eligible sickle cell disease and beta thalassemia patients across 10 countries.
Stoke Therapeutics Zorevunersen Shows Promise for Dravet Syndrome Treatment Ahead of Q1 2026 Results
Stoke Therapeutics prepares Q1 2026 earnings call featuring Zorevunersen, an investigational antisense therapy targeting Dravet syndrome's underlying cause.
uniQure AMT-130 Gene Therapy Advances Toward UK Approval for Huntington's Disease Treatment
uniQure completes successful UK MHRA pre-submission meeting for AMT-130 Huntington's disease gene therapy, targeting Q3 2026 marketing authorization application.
Ray Therapeutics RTx-015 Receives EMA PRIME Designation for Retinitis Pigmentosa Treatment
Ray Therapeutics' RTx-015 gene therapy receives EMA PRIME designation for retinitis pigmentosa, accelerating regulatory pathway for vision restoration treatment.
Silexion Therapeutics Submits Phase 2/3 Trial Application for SIL204 KRAS-Targeted Pancreatic Cancer Treatment
Silexion Therapeutics successfully submits Phase 2/3 clinical trial application to Germany's BfArM for SIL204, targeting KRAS-driven pancreatic cancer.
Solid Biosciences Receives European Orphan Drug Designation for SGT-003 Duchenne Muscular Dystrophy Treatment
Solid Biosciences secures European Commission orphan drug designation for SGT-003, advancing gene therapy development for Duchenne muscular dystrophy patients.
EMA Issues Positive Opinion for Itvisma (Onasemnogene Abeparvovec) Gene Therapy for Spinal Muscular Atrophy
European Medicines Agency issues positive opinion for Itvisma, a one-time gene therapy for spinal muscular atrophy in infants and children.
RNA Therapy Clinical Trials Market Reaches $3.4 Billion as Personalized Medicine Drives Growth Through 2035
RNA therapy clinical trials market hits $3.4B driven by delivery advances, rare disease funding, and personalized medicine growth through 2035.
Center for Cancer and Blood Disorders Launches CAR T-Cell Therapy Program in Maryland, Expanding Community Access
CCBD becomes first Maryland community oncology practice to offer FDA-approved CAR T-cell therapy, significantly expanding patient access in the region.
Allogene Therapeutics Expands Cemacabtagene Ansegedleucel Phase 2 ALPHA3 Trial to South Korea and Australia
Allogene Therapeutics receives regulatory clearance to expand pivotal Phase 2 ALPHA3 trial of cemacabtagene ansegedleucel to South Korea and Australia markets.