Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...
In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...
Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...
The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called ...
Failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...
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 CardiomyopathyCHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...
Intellia presented data from its Phase I study of NTLA-2001 at the 2024 American Heart Association scientific meeting in ...
Medpage Today on MSN6d
First Brain-Delivered Gene Therapy Approved for AADC DeficiencyThe treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
- VY1706 is the fifth neuro gene therapy development candidate nominated leveraging Voyager’s IV-delivered, CNS-penetrant TRACER capsids - ...
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