A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...
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 ...
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...
Stocktwits on MSN
RGNX shares pare losses as Wall Street dismisses gene therapy clinical hold spurred selloff as an overreaction
Leerink said the premarket selloff in shares underscores the degree of surprise on the news, feeding into the "FDA ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
By Kamal Choudhury Jan 27 (Reuters) - Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a ...
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.
After finding an intraventricular tumor in one clinical trial participant, the FDA placed a clinical hold on an experimental ...
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