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 ...

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 trial to prove the therapy actually helps Huntington’s patients. The ...
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.
Stocktwits on MSN

QURE jumps 20% – analysts upgrade stock on Huntington’s disease therapy update, retail eyes $150

UniQure’s AMT-130 is a gene therapy to reduce levels of the Huntingtin protein in the brain. ・The FDA recently said that data ...
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 ...
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 ...

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 ...

Abeona Therapeutics® Provides Business Update Highlighting Building Momentum in ZEVASKYN® Commercial Launch

Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced an update on the building momentum in the Company’s launch of FDA-approved ZEVASKYN (prademagene zamikeracel), a first-of-its-kind, autologous ...
Forbes

Breakthrough Gene Therapy For Butterfly Children’s Disease

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...
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 ...