Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing ...
The American Journal of Managed Care1d
Real-World Study Suggests DMD Gene Therapy Is Safe, Effective
Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...
Gene editing with adeno-associated virus vector offers hope for hereditary deafness
An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...
Good News Network3d
Groundbreaking Gene Therapy Cures 21-year-old of His Sickle Cell Anemia: ‘I’m not in pain anymore’
Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...
NC Biotech7h
Beam Therapeutics reports gene editing breakthrough
The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...
Teen’s death following Sarepta DMD gene therapy underscores a risk seen for decades
The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...
Gene-Therapy Maker Sarepta Tanks After Patient Death
Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...
The News International6d
Investors shift focus from gene therapy to lucrative weight-loss drugs
Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...
University of California7d
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
Fierce Pharma3d
Bharat Biotech enters cell and gene therapy market with $75M production investment: reports
With a storied history in vaccine development, India's Bharat Biotech is shaking things up and answering the call of advanced ...
The American Journal of Managed Care9d
Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...