Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
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.
MedPage Today on MSN
FDA Halts Two Gene Therapy Trials After Child Develops Brain Tumor
Studies of Hunter and Hurler syndromes on hold ...
Pharmaceutical Technology on MSN
Eli Lilly outlays $1.12BN for hearing loss gene therapy partnership
Lilly and German biotech Seamless Therapeutics will develop recombinase-based treatments for hearing loss.
University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...
Stocktwits on MSN
Rare stock slips 3% today — what’s up with Ultragenyx’s gene therapy for Sanfilippo syndrome?
Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...
By Kamal Choudhury Jan 27 (Reuters) - Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a ...
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.
For the most part, Lucas Azevedo is just like most four-year-old boys – he’s active, smiles a lot and loves playing with cars ...
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