FDA official calls UniQure's gene therapy a 'failed' treatment for Huntington's disease

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

FDA details rationale for rejecting rare disease gene therapy from Regenxbio

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...
The Earth Times

Gene Therapy Meets Regulatory Reality: The FDA–uniQure Clash Over a Promising Huntington’s Treatment

The FDA’s dispute with biotech firm uniQure over Huntington’s gene therapy highlights growing tensions between innovation, ...
Fierce Biotech

UPDATED: Senior FDA official refutes uniQure's claims amid controversy over Huntington's gene therapy

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

UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms

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

Intellia gets FDA nod to resume second gene therapy trial after safety pause

By Christy Santhosh March 2 (Reuters) - Intellia Therapeutics said on Monday the U.S. Food and Drug Administration has lifted ...
MPR

Gene Therapy Fast Tracked for BAG3-Associated Dilated Cardiomyopathy

AFTX-201 uses a proprietary capsid to deliver a human BAG3 transgene, aiming to restore cardiac function with lower doses than conventional gene therapies.

Gene therapy is transforming lives, but for many Americans it's hard to reach

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

Intellia’s CRISPR Gene Therapy Free To Move Forward After FDA Fully Lifts Hold

The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme ...
MedPage Today on MSN

Cardiologist's Fake Cancer; Gene Therapy Trial Resumes; More Impella Woes

Recent developments of interest in cardiovascular medicine ...
Devdiscourse

FDA Challenges UniQure's Huntington's Disease Gene Therapy Trial

UniQure faces a setback as the FDA demands a placebo-controlled study for its Huntington's gene therapy, rejecting the recent trial design that lacked randomized control. The FDA remains firm on its ...