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 ...

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 ...

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

Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by synaptic dysfunction, neuronal loss and the accumulation of amyloid plaques and neurofibrillary tangles, ...

Discover the major highlights from ASCO 2025, according to researchers and drug developers who attended the convention.