Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.
As Eli Lilly awaits an FDA approval decision for its oral GLP-1 orforglipron, the pharma has discarded three clinical-stage drugs, including a gene therapy from its $1 billion acquisition of Prevail ...
Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotypeBLA resubmitted to U.S. FDA in January 2026; ...
Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of ...
A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...
Clinical Trials Arena on MSN
Ultragenyx’s gene therapy shows sustained improvements in MPS IIIA
There are currently no approved disease-modifying therapies for MPS IIIA.
It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...
Market OverviewThe global cell and gene therapy market is undergoing rapid and transformative growth, reflecting its increasing importance in modern healthcare.The market is projected to expand at a ...
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results