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 ...

FDA push for fake Huntington’s brain surgery trial spurs ethical concerns

Subjecting participants to invasive procedures without the prospect of therapeutic benefit is unjustifiable,” says the CEO of ...
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 ...
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, ...
New Orleans City Business

Louisiana sees first ENCELTO gene therapy procedure at AVALA Hospital

AVALA Hospital, in partnership with Retina Associates New Orleans, has successfully performed Louisiana’s first ENCELTO procedure, a cell-based gene therapy for adults with macular telangiectasia type ...
BioSpace

Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
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 ...

Hope And Hurdles For Sickle Cell Gene Therapy

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...
Fred Hutch

Scientists see foam as starting point of a path to bedside gene therapy

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...
Medical Xpress

Therapeutic or Preventive Procedure:

Older women could be vulnerable to harmful inflammation from new gene therapies to treat incurable eye diseases, new research has found. The University of Bristol-led study, published in Molecular ...
FierceBiotech

Regeneron amplifies CHORD progression, sharing updated data on hearing loss gene therapy

Regeneron has posted updated clinical data on DB-OTO, providing more evidence of the gene therapy’s potential to treat a rare genetic form of deafness as it plans for a potential FDA filing later this ...
GEN

Gene Therapy Companies Set Sights on Cardiovascular Disease

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...