Gene Therapy Loses Luster as Investors Eye Quicker Returns From Weight-Loss Drugs
In the last year, some drugmakers have pulled back from the sector, including Pfizer, which recently stopped selling its gene ...
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 ...
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 Biotech6h
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 ...
Science Daily2d
Gene Therapy News
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...
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 ...
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 ...
DNA-loaded lipid nanoparticles are poised to bring gene therapy to common chronic diseases
A breakthrough in safely delivering therapeutic DNA to cells could transform treatment for millions suffering from common ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
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 ...
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 ...
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 ...