Ocugen, Inc. gene therapy outlook: key 2026 catalysts, Phase 2/3 eye-disease data, BLA plans to 2028, and risks. Click for ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders ...

Osaka, Japan - A research team at The University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’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 ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Abu Dhabi achieved a medical milestone on January 5, 2026, with the UAE's first gene-therapy injection for inherited blood disorders. CASGEVY, utilizing CRISPR-Cas9, corrects faulty DNA in conditions ...