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 MSN20h
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 ...
FierceBiotech1d
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 ...
BioSpace19h
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 ...
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 ...
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 ...
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 ...
Pharmaceutical Technology1d
Intellia’s gene editing therapy shows early potential in rare heart condition
Intellia presented data from its Phase I study of NTLA-2001 at the 2024 American Heart Association scientific meeting in ...
Science Daily5d
New discovery enables gene therapy for muscular dystrophies, other disorders
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.