(Bloomberg) -- A top US Food and Drug Administration official overrode reviewers to give broad approval to Sarepta Therapeutics Inc.’s gene therapy for a rare muscle disease in children ...

Gilead Sciences (NASDAQ:GILD) traded higher in the morning hours Thursday after announcing that a Phase 3 trial for its experimental therapy lenacapavir succeeded when used as a twice-yearly ...

Ionis Pharmaceuticals (NASDAQ:IONS) traded higher Tuesday after the U.S. FDA accepted to review a marketing application for its anti-lipid therapy olezarsen, targeted at a rare genetic condition ...

Earlier, the therapy had only ... age group that appeared to gain the most benefit in studies. In October, Sarepta said its confirmatory trial failed to clearly slow the disease in a yearlong study of ...

Harith Rajagopalan, M.D., Ph.D. thinks his company, Fractyl Health, may have the answer: a gene therapy that integrates ... be enough for long-term efficacy. Second, because the treatment isn ...

Taysha Gene Therapies has unfurled more data in the developmental disorder Rett syndrome, linking its gene therapy to improvements ... Taysha’s second adult patient, a 21-year-old female ...

Eli Lilly has entered into an exclusive licence agreement worth over $620m to gain rights to QurAlis’ investigational precision therapy for neurodegenerative diseases including amyotrophic lateral ...

In November 2023, the FDA issued a warning about a risk of secondary cancers—particularly blood cancers—that may be associated with CAR-T cell therapy. The warning was preceded by a rising ...

The risk of secondary cancer after CAR-T therapy, pioneered at Penn, is less than regulators feared last year, an FDA official said June 14 at cell and gene therapy conference in King of Prussia.

Global investors are increasingly interested in gaining exposure to China through secondary private equity investments amid US-China tensions and a market slump that has squashed valuations ...