A new strategy to enhance gene therapy for sickle cell disease
Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...
Promising results from phase 1 gene therapy trial for Danon disease
Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...
BioSpace23h
Regenxbio Gears Up to Challenge Sarepta in DMD as Gene Therapy Advances to Pivotal Studies
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...
FierceBiotech1d
Neurogene stops Batten gene therapy after FDA denies request
Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...
FierceBiotech2d
CSL Behring shuts down California cell and gene therapy R&D site amid group pivot
In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...
WebMD1d
First Brain-Injected Gene Therapy Approved by FDA
The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called ...
BioSpace21h
Neurogene Drops Batten Gene Therapy Program
Failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...
Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst
On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study ...
Clinical trial finds new gene editing therapy for heart condition to be safe and effective
A new type of therapy that "edits" a gene in patients with a rare heart condition has been shown to be safe and effective, ...
STAT1d
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...
MedPage Today on MSN1d
Gene Therapy Promising for Rare Genetic Cardiomyopathy
CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...
Taiwan News35m
Voyager Selects Tau Silencing Gene Therapy Development Candidate for Alzheimer’s Disease
- VY1706 is the fifth neuro gene therapy development candidate nominated leveraging Voyager’s IV-delivered, CNS-penetrant TRACER capsids - ...