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 ...

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

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 ...

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

Recent developments of interest in cardiovascular medicine ...

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

Precigen Inc. reports fourth-quarter results on Monday, with analysts looking for signs that the commercial launch of its first FDA-approved gene therapy is driving a material revenue ramp as the ...

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