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.

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 ...
Investing.com India

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 ...

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 ...
GlobalData on MSN

Eli Lilly bets on in vivo cell therapy in $2.4bn Orna Therapeutics buyout

Lilly joins the growing list of big pharma companies hoping to see success from their acquired in vivo CAR-T programmes.
PharmTech

Fresenius Kabi and Phlow Corp. Enter First US End-to-End Manufacturing Collaboration

Phlow will manufacture epinephrine API in Virginia while Fresenius Kabi formulates and fills finished doses in existing US ...
Carnegie Mellon University

Carnegie Mellon Researchers Rethink Chronic Pain

Across neuroscience, biomedical engineering and artificial intelligence, researchers from Carnegie Mellon University are exploring how pain is measured, understood and treated to support safer, more ...