The Latest Breaking News from Gene Therapy

Exclusive: Alison Joseph's sons Noah and Hunter both have Duchenne muscular dystrophy. She tells Rhian Lubin the heartbreaking reality of watching one thrive while the other suffers

Researchers used evolutionary genetics and CRISPR gene editing tech to develop an innovative treatment for gout. The approach has yet to be tested in humans.

The U.S. Food and Drug Administration (FDA) on Monday extended its review timeline for REGENXBIO Inc.’s (NASDAQ:RGNX) Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.

Gene therapies hold immense promise for treating sight loss. These therapies use a modified, harmless virus to deliver therapeutic genes directly to diseased cells, helping them to function normally again.

Prasad left the agency after far-right influencer released edited audio misleadingly suggesting he was anti-Trump

Vinay Prasad resigns from the FDA after backlash over vaccine restrictions and gene therapy decisions. Political pressure cited in sudden departure.

In May, FDA Commissioner Marty Makary named Vinay Prasad the next director of the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER). Within three months, Prasad resigned.

Globally, an estimated 1.3 million women and girls living with HIV become pregnant every year, according to the World Health Organization

A patient suffering from a genetic eye disorder in Italy regained his sight thanks to a world-first experimental gene therapy.

As gene therapies continue to evolve as a promising frontier in medical treatment, regulatory scrutiny intensifies following safety concerns. Recent developments with Sarepta Therapeutics Inc (NASDAQ:SRPT) highlight the delicate balance between innovation and patient safety in the biopharmaceutical industry.

Sarepta stock soared after the FDA said the biotech company can resume shipments of a controversial gene therapy drug.