FDA reconsidering gene therapy for Huntington's disease after official who rejected it leaves agency
By
Beth Mole
Warm and crisp on the edges. A bagel with a bit of bite.
Summary
A gene therapy for Huntington's disease from UniQure has gained a new path toward FDA approval following the ouster of Trump administration officials, notably Vinay Prasad, who had previously rejected the therapy in a move one former FDA official called "truly evil." Huntington's disease is an inherited, incurable condition causing brain nerve degeneration, typically striking in middle age with patients often dying in their 50s and 60s. UniQure held another FDA meeting after Prasad's exit, suggesting a potential reversal of the earlier rejection.
Key quotes
· 3 pulled"Truly evil" FDA rejection of gene therapy overturned after Trump official ousted
Huntington's disease is an inherited condition that typically strikes in middle age and causes nerve cells in the brain to gradually break down
There are currently no treatments for the disease, and many afflicted die in their 50s and 60s
You might also wanna read
Huntington's disease treatment shows success in slowing progression for first time, UK trial finds
For the first time, doctors have successfully treated Huntington's disease, a devastating inherited neurodegenerative condition that kills b
bbc.com·8mo ago
FDA Approves First Dual-AAV Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program
The FDA has approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy for trea
FDA·1mo agoFDA Initiates Approval of Leucovorin Calcium for Cerebral Folate Deficiency
The FDA has initiated the approval of leucovorin calcium tablets for patients with cerebral folate deficiency (CFD), a neurological conditio
fda.gov·8mo ago
Goldman Sachs Report Questions Financial Sustainability of Curative Gene Therapies
Goldman Sachs analysts question whether curing patients is a sustainable business model for biotech companies, particularly those developing
cnbc.com·7mo ago
HHS Ends mRNA Vaccine Development Under BARDA Following Review
The U.S. Department of Health and Human Services (HHS) has announced the wind-down of its mRNA vaccine development activities under BARDA, i
hhs.gov·10mo ago
Third Death in Sarepta Gene Therapy Trial for Limb-Girdle Muscular Dystrophy
A 51-year-old patient in a Phase I study of Sarepta's gene therapy for Limb-Girdle muscular dystrophy Type 2E has died from acute liver toxi
biocentury.com·11mo ago