Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | ...

The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease ...

The FDA has granted Fast Track designation to AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy.

Proof-of-concept trial in a single patient shows that cells can survive transplantation without immunosuppression ...

Qure's stock has dropped by more than 40% after being asked to conduct another study of its Huntington’s disease gene therapy ...

Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one ...

Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / ...

UniQure NV’s shares plunged after US regulators said the company should conduct another study of its Huntington’s disease ...