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.
The American Journal of Managed Care

Shaping Gene Therapy Approaches in Neuromuscular Disease

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Solid Biosciences Announces Positive Feedback from Type C Meeting with FDA for SGT-003 Gene Therapy for Duchenne Muscular Dystrophy

Company aligned with FDA on Phase 3 randomized, double-blind, placebo-controlled trial design -- IMPACT DUCHENNE: Company ...
Fierce Biotech

Lilly drops 3 pipeline drugs, including gene therapy from $1B Prevail buyout

As Eli Lilly awaits an FDA approval decision for its oral GLP-1 orforglipron, the pharma has discarded three clinical-stage ...
Monitor

New gene therapy offers hope to sickle cell patients

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
News-Medical.Net on MSN

New workflow boosts nuclear delivery for safer gene therapy

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.
Medical Xpress

First-in-human trial of CRISPR gene-editing therapy safely lowers cholesterol and triglycerides

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...
Los Angeles Magazine

What Is Gene Therapy?

This sponsored article was provided by a partner and is not editorial content from Los Angeles Magazine or the Engine Vision Media Network. Every cell in your body holds a unique genetic code within ...