Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
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 ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...
Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
BioPharma Dive2d
Novartis builds case for new SMA gene therapy
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
University of California2d
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 ...
Upgrading Beam Therapeutics: Promising BEAM-302 Data Opens Door For Durable Gene Therapy
Beam Therapeutics reported promising Phase 1/2 data for BEAM-302, demonstrating durable gene correction for AATD. See why I ...
The American Journal of Managed Care4d
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 ...
GEN - Genetic Engineering and Biotechnology News2d
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
GEN - Genetic Engineering and Biotechnology News3d
DMD Patient Dies After Treatment with Sarepta Gene Therapy
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
The American Journal of Managed Care3d
STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children
Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...
Sarepta says teen died after its gene therapy treatment
Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for a rare muscular dystrophy.