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

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

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 ...

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

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 ...

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 ...

Join the ELRIG conference to hear from experts on cell and gene therapy. Discover advancements shaping the future of medicine.

Eric Boodman focuses on narrative features, exploring the startling ways that science and medicine affect people’s lives. You can reach Eric on Signal at eboodman.88. Katie Palmer covers telehealth, ...

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

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 ...