Will consider data that includes "the experience of individuals," FDA commissioner says ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

The FDA is now expected to decide on the application by Aug. 23. ・The company intends to manufacture the therapy at its new gene therapy manufacturing facility in Bedford, Massachusetts, if approved.

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...

As cell and gene therapies move into more hospitals and cancer centers, health system leaders are learning that clinical ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene therapy for severe hemophilia A.

This sponsored article was provided by a partner and is not editorial content from Los Angeles Magazine or the Engine Vision Media Network. Every cell in your body holds a unique genetic code within ...

A lower court had previously ruled for Sarepta in the companies’ long-running dispute, finding that REGENXBIO’s AAV patent ...