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

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

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...

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 ...

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized ...

After 307 days at CHOP, KJ was dressed in what early studies suggest are the world’s smallest and cutest graduation ...

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

CRISPR Therapeutics CRSP is currently the only company in the world to have secured approval for a CRISPR-based gene therapy ...

KJ Muldoon, who spent nearly the first year of his life being treated at the Children’s Hospital of Philadelphia, is finally home after a gene-editing therapy gave him a second chance at life.

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