In May, a historic moment in science and medicine was captured in a single photo that circulated across news outlets ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with FDA approval application planned for early next year.

Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of SYNGAP1-related disorder, a devastating condition affecting an estimated 1 million ...

Scientists have reversed diabetic heart failure with a genetic therapy in mice and miniature human "hearts" grown from stem ...

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life. The work stems ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Anyone looking for evidence of genetic medicine’s ...

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 ...

Bayer agreed to acquire unlisted U.S. biotech firm Asklepios BioPharmaceutical Inc for as much as $4 billion in a bet on gene therapy with the help of modified viruses.

Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.

Takeda exits cell and gene therapy development amid cost and safety concerns in Japan Takeda to divest cell and gene assets ...