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 ...

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, ...

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 ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

ELRIG has revealed Drs Stephen Ward (Cell and Gene Therapy Catapult) and Annarita Miccio (Imagine Institute) as keynote ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...

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 ...

ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the keynote speakers for Cell and Gene Therapy 2026. Drs Stephen Ward (Cell and ...

Drs Stephen Ward, CTO of Cell and Gene Therapy Catapult, and Annarita Miccio, managing director at Imagine Institute for Genetic Diseases, will lead the inaugural two-day event taking place at Hinxton ...