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 ...

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 ...

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, ...

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 ...

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 ...

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 ...

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 ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...