PHILADELPHIA (WPVI) -- A 5-year-old from Center City has faced rare odds since birth, and now this warrior is in a real life race to rewrite his story. Pierce Hynes is battling not one, but two rare ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

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 ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. The New York pharma is ending global development and commercialization of Beqvez ...

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, ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...