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 ...
The Manila Times

Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S.

Approval in severe-to-profound and profound OTOF-related hearing loss is based on pivotal results of the CHORD trial demonstrating 80% of participants achieved or surpassed a hearing level meeting the ...
Medical Xpress

New gene therapy uses Tylenol to combat genetic diseases

Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science ...
Labiotech.eu

The future of cell & gene therapy: Key trends to watch

Cell and gene therapies are moving towards correcting root causes of diseases. Let's take a look at future cell and gene therapy trends.

Goldman Sachs warned 'curing patients isn't a sustainable business model' in 2018 — 8 years later, biotech is quietly proving the bank right

Many new gene therapy treatments aren’t as profitable as predicted. But the reasons why don’t quite fit Goldman Sachs’ ...
Medscape

Gene Therapy Injections for Knee OA: Where Are We At?

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection ...
sharewise

OCGN's Gene-Agnostic Strategy Could Reset Retinal Therapy

Ocugen OCGN is pushing a modifier gene therapy platform across several retinal diseases where current options are limited, ...