Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, and ...

A single dose of experimental gene therapy GS001 reduced bleeding episodes in men with severe hemophilia A, a study showed.

Solid Biosciences (SLDB) stock retains a "Strong Buy" rating, driven by robust interim data for SGT-003 in pediatric Duchenne Muscular Dystrophy [DMD]. Read the full analysis here.

Regenxbio Inc. RGNX shares are down on Wednesday after the company reported new interim data from its Phase 1/2 AFFINITY DUCHENNE trial of RGX-202. • Regenxbio stock is among today’s weakest ...

Investigating microplastics in agriculture reveals their effects on soil microbes and viruses, crucial for developing strategies for ecosystem restoration.

AFTX-201 is designed using Affinia's proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than doses of gene therapies using conventional capsids WALTHAM, ...

Alex Sundby is a senior editor at CBSNews.com. In addition to editing content, Alex also covers breaking news, writing about crime and severe weather as well as everything from multistate lottery ...

The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments. The new ...

WASHINGTON, Feb 23 (Reuters) - The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for rare and life-threatening genetic diseases, ...

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teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...