Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...

A sponsor will have to demonstrate success with "with several consecutive patients with different bespoke therapies" to ...

In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory ...

Type 1 diabetes is an autoimmune disease in which the insulin-producing β cells in the pancreatic islets of Langerhans are ...

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

With a snip of a gene, doctors may one day permanently lower dangerously high cholesterol, possibly removing the need for ...

Q3 2025 Earnings Call Transcript November 4, 2025 Taysha Gene Therapies, Inc. misses on earnings expectations. Reported EPS ...

An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...

Opal Sandy underwent a gene therapy injection to her right ear in 2023. A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...