US FDA declines to approve Regenxbio's gene therapy for rare disease

Feb 9 (Reuters) - Regenxbio said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for ...
Fierce Biotech

FDA rejects Regenxbio's gene therapy in Hunter syndrome, leaving CEO 'concerned'

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...
BioSpace

Ultragenyx’s Gene Therapy Shows Long-Term Efficacy in Two Sanfilippo Studies

The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing ...
STAT

FDA rejects Regenxbio’s rare-disease gene therapy

The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday.
Fierce Biotech

UniQure pauses Fabry gene therapy dosing in 2 groups after toxicities emerge

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

Solid Biosciences: Positive FDA Meeting For SGT-003 Sets Up Next Milestones

Solid Biosciences Inc. is a Strong Buy driven by positive FDA alignment on phase 3 IMPACT DUCHENNE trial for SGT-003. Read ...
BioSpace

Liver Toxicities Force Pause on 2 Dose Groups of UniQure’s Mid-Stage Fabry Study

The gene therapy uses an AAV vector to restore healthy levels of the alpha-galactosidase enzyme, which is rendered dysfunctional in patients with Fabry disease, leading to the toxic build-up of lipids ...

Gene-editing therapy proves effective for Leber's hereditary optic neuropathy

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

David Liu unlocks the power of gene editing to treat rare genetic diseases

Among them was David Liu, a biochemist at the Broad Institute and Harvard University in Cambridge, Massachusetts. Liu isn’t a ...

FDA Declines to Approve Regenxbio's Hunter Syndrome Treatment

The Food and Drug Administration has declined to approve Regenxbio's gene therapy, RGX-121, for the treatment of Hunter syndrome, a rare and fatal neurodegenerative disease. The clinical-stage ...
Investing.com UK

REGENXBIO stock falls after FDA rejects gene therapy for Hunter syndrome

Investing.com -- REGENXBIO (NASDAQ:RGNX) stock fell 27% after the company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) rejecting its gene therapy RGX-121 ...

Potent DNA-recombination method developed for large cargo delivery in gene therapy

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...