KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

Baby KJ was successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of ...

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

After 307 days at the Children's Hospital of Philadelphia, a local baby who was born with a rare and usually fatal disorder ...

FDA alignment on outcomes for the key Part B trial was supported by caregiver-reported milestone gains. Taysha Gene Therapies ...

KFSHRC accounts for 52.4% of all active clinical trials in Saudi Arabia.

A baby born with a rare and dangerous genetic disease is thriving after getting an experimental gene editing treatment made ...

Beam Therapeutics rises after the FDA grants orphan drug status to its gene-editing therapy BEAM-101 for sickle cell disease.

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

There are early signs that gene therapy can treat a rare neurodevelopmental condition, leaving a Brockville-area mother ...