Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from ...

The FDA has designated the viral vector rAAVrh74, which Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses in its gene therapy ...

Victoria Gray is the first person in the world to receive CRISPR, a gene-editing therapy for sickle cell disease created by ...

By reprogramming brain cells, a new gene therapy approach for Alzheimer's developed by UC San Diego researchers could address ...

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...

While heritable human genome editing is banned in the United States, across Europe and much of the world, the technology ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

Congress, convening virtually and in Milan, Italy, from June 12 to June 15, 2025, will feature a revamped program structure ...

The U.S. health secretary said people should have access to experimental therapies including unregulated uses of stem cells.

The partners further aim to raise awareness and increase early diagnosis of BAG3-associated dilated cardiomyopathy.