Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...

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

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 ...

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 ...

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 ...

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

Codon develops curated PR solutions for transformational biotech companies at every stage to help establish brand credibility ...

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, ...

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 ...

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 ...