News

Falmouth teen begins human gene therapy for sickle cell. 'High risk and it's scary'
A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.
Alzheimer's gene therapy shows promise in preserving cognitive function
Researchers at the University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's ...
News-Medical.Net1h
Alzheimer's gene therapy could help protect the brain from damage and preserve cognitive function
Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's disease ...
BioSpace3h
Sarepta Wins FDA’s Platform Technology Designation, Marking ‘Critical Shift’ in Gene Therapy Development
The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...
The American Journal of Managed Care1h
Splicing Modifiers and Gene Therapy for SMA
Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...
Fierce Biotech3h
Chasing Sarepta, RegenXBio links DMD gene therapy to improved outcomes
RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...
BioPharma Dive19h
FDA to use new review tool on Sarepta’s gene therapy work
The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...
Sickle cell gene therapy offers hope to N.J. teen
New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...
Gene Therapy For Inherited Disease In The Unborn Child
In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...