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 ...

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.
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 ...

FDA defends its demand for new trial for rare brain disease therapy

A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new ...
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 ...

FDA goes on offense amid surge of criticism for recent drug decisions

In a highly unusual move for a normally buttoned-up regulatory agency, a senior official with the US Food and Drug ...
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.

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 ...

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 ...

UniQure Falls as FDA Seeks Controversial Gene Therapy Study

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