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 ...
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 ...
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 ...
Investing.com South Africa

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 ...
Interesting Engineering on MSN

First-ever experimental gene therapy seeks to restore vision by rejuvenating eye neurons

Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene ...
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 ...
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 ...
BioPharma Dive

Regenxbio gene therapy trials suspended by FDA over safety worries

The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing ...
News-Medical.Net

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

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 ...

SparingVision Successfully Completes PRODYGY Trial Patient Dosing with SPVN06, its Novel Neuroprotective Gene Therapy

All patients dosed with SPVN06 in Phase I/II trial continue to demonstrate a favorable safety profileInitial data readout for this novel ...
ABC7 San Francisco

Children with genetic deafness have hearing restored with gene therapy, study finds

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...