Researchers to test gene therapy for rare diseases with a new FDA trial protocol
Schematic of “umbrella of umbrellas” master protocol clinical trial. Credit: The American Journal of Human Genetics (2025). DOI: 10.1016/j.ajhg.2025.10.006 Earlier...
therapy
Everything to Know About Cell and Gene Therapy in 2025
Is cell and gene therapy the future?Cell and gene therapy is widely regarded as a major part of the future...
Experimental gene therapy restores immune system function in children with genetic immune disorder
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained...
Gene Therapy Market Share & Trends Report, 2025-2034
Gene Therapy Market Size The global gene therapy market size was valued at USD 9 billion in 2024. The market...
REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss (Revised)
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study. This gene therapy...
REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.This gene therapy is...
REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.This gene therapy is...
Opus Genetics reports positive pediatric data from LCA5 gene therapy study
Opus Genetics, Inc. released positive data from a pediatric cohort in an ongoing phase 1/2 clinical study evaluating OPGx-LCA5, an...
New gene therapy strategy reactivates dormant genes to treat blood disorders
Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to...
