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

Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...
BioPharma Dive

Regenxbio gene therapy trials suspended by FDA over safety worries

The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing ...

Adolore BioTherapeutics Announces FDA Orphan Drug Designation for its Kv7-activating rdHSV-CA8* Therapy in the Treatment of Erythromelalgia

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
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 ...
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 ...
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 ...
University of California

Could gene therapy treat a deadly heart condition that targets young athletes?

ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...

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 ...

Sarepta's Duchenne gene therapy slows disease progression at three years

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
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.
New Atlas

Gene editing takes a major step toward ending daily cholesterol pills

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
BioSpace

Sarepta’s DMD Gene Therapy Staves Off Disease Three Years After Treatment

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.