The company said that the FDA hold has impacted two experimental gene therapies, RGX-111 and RGX-121. ・REGENXBIO added that ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
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 ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...
Scientists have achieved a genetic feat once thought impossible: removing an entire extra human chromosome using gene-editing ...
In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...
An AAV vector genome integration event associated with PLAG1 overexpression was identified in a preliminary analysis of the resected tumor.
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.
Emil Kakkis and Ultragenyx Pharmaceutical are taking another swing at a potentially deadly genetic disease with their one-shot-and-done gene therapy.
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
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