News

The American Journal of Managed Care2d
Progress and Challenges With Gene Therapy in Hereditary Hearing Loss
Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...
Fierce Pharma2d
Vinay Prasad's exit signals FDA's probable slip toward more permissive regulation of cell and gene therapy: analyst
But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...
GEN - Genetic Engineering and Biotechnology News1d
Gene Therapy Companies Set Sights on Cardiovascular Disease
The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...
Diorasis Therapeutics and Northway Biotech Announce Partnership for the Development and Manufacturing of AAV Gene Therapy for Glaucoma
Leveraging Northway Biotech’s 20+ years of CDMO expertise, Diorasis Therapeutics is advancing AAV gene therapy for glaucoma ...
Fierce Pharma1d
Untangling Sarepta’s gene therapy fallout and a growing trust deficit
Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...
Managed Healthcare Executive23h
Gene Therapy for Wet AMD May Replace Anti-VEGF Injections | ASRS 2025
Ixo-vec reduced anti-VEGF injection frequency and was well tolerated in patients with wet AMD. A phase 3 pivotal trial ...
Progress in gene therapy offers hope for long-term knee pain relief
For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...
Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
Novel Access Model For Sickle Cell Disease Gene Therapy Could Be Template
If successful, the access model for sickle cell disease gene therapies could serve as a blueprint for other such treatments ...
X-SCID gene therapy gives scientists a rare glimpse into early immune system
Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune ...