This scan of a mouse brain shows blood vessels (red), brain cells (green), and amyloid plaques (blue). A new gene therapy treatment developed by UC San Diego works by reprogramming the behavior of ...

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

CORONA, Calif. (KABC) -- A teenage girl is alive thanks to an experimental gene therapy treatment created at UCLA. She was born with a rare genetic disorder, so any common infection could've killed ...

Hot on the heels of a $150 million fundraise, Atsena Therapeutics is back with early-stage data for the company’s eye disease gene therapy. The candidate improved the structure and function of the ...

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 ...

Even hearing the phrase “Huntington’s disease” will make a room suddenly somber. So the joy that accompanied a recent announcement of results of an experimental gene therapy for the deadly diseases ...

A Johnson & Johnson gene therapy in development for an inherited vision-loss disorder has failed a Phase 3 study, the latest clinical research setback for a rare eye disease that so far has no ...

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene-writing program, inking a deal that lets it in on a prospect for alpha-1 antitrypsin deficiency (AATD) through a $150 million ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...