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 ...
MyChesCo on MSN1d
Revolutionary Gene Therapy Approved: Kebilidi Offers New Hope for Rare Genetic Disorder
The U.S. Food and Drug Administration (FDA) has announced a groundbreaking approval of Kebilidi (eladocagene exuparvovec-tneq ...
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 ...
BioSpace1d
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 ...
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 ...
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 ...
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 ...
BioSpace23h
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 ...
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 ...
STAT2d
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 MSN6d
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...