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

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

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

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

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

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

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

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

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

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 ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...