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

UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.

In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...

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

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 ...

Recent developments of interest in cardiovascular medicine ...

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

Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / Shutterstock.com) The US Food and Drug Administration (FDA) has released the ...

Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Delaying onboarding until FDA approval reduces sunk-cost risk but can impede institutional learning and slow post-approval ...