MedPage Today on MSN

FDA halts two gene therapy trials after child develops brain tumor

Studies of Hunter and Hurler syndromes on hold ...
STAT

FDA halts gene therapy trials after brain tumor found in patient

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
News-Medical.Net

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
KIMT News 3

Mayo Clinic's gene therapy breakthrough targets most-common kidney disease

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
GlobalData on MSN

Genespire to take rare disease gene therapy to clinic in 2026

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
GlobalData on MSN

FDA puts REGENXBIO gene therapy trials on hold after brain tumour

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.
News Medical on MSN

Gene therapy targeting connexin 43 shows promise across inherited heart diseases

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

Novato's Ultragenyx tries — again — to convince FDA to approve brain disease gene therapy

Emil Kakkis and Ultragenyx Pharmaceutical are taking another swing at a potentially deadly genetic disease with their ...
Yicai Global

Chinese Scientists Make Key Advance in Using Gene Therapy

(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...
The Manila Times

Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA

Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) ...
BioWorld

Gene therapy restores auditory function in deaf mice

DFNB16, a recessive form of mild-to-moderate human deafness, is caused by mutations in the STRC gene, which encodes stereocilin, a protein essential for the effective function of outer hair cells as ...
Becker's Hospital Review

FDA puts hold on gene therapy trials after brain tumor

After finding an intraventricular tumor in one clinical trial participant, the FDA placed a clinical hold on an experimental ...