Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

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

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

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

Researchers at the University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's ...

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

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

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

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

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

By reprogramming brain cells, a new gene therapy approach for Alzheimer's developed by UC San Diego researchers could address ...