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 ...
Scientific American

Pioneering gene therapy may treat a deadly seizure disorder

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

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 ...
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 ...

Cell and Gene Therapy CDMO Market Size to be Worth USD 29.49 Billion by 2035; Growth is Driven by Increasing Number of Advanced Therapies Globally

U.S. Cell and Gene Therapy CDMO Market is projected to grow from USD 2.09 Billion in 2025 to USD 10.28 Billion by 2035 due to expanding gene and cell therapy pipelines globallyAustin, March 06, 2026 ...
MedPage Today on MSN

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

Recent developments of interest in cardiovascular medicine ...
Managed Healthcare Executive

A Q&A with Zahra Mahmoudjafari on building successful cell and gene therapy programs

Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
FOX 32 Chicago on MSN

Lurie Children’s helps lead clinical trial for gene therapy targeting Dravet syndrome

A new clinical trial led in part by a Lurie Children’s Hospital doctor is showing promise for children with Dravet syndrome, a severe form of epilepsy, and could become the first gene regulation ...
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 releases hold on second of Intellia’s ATTR-CM gene therapy trials

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 ...

First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...