Becker's Hospital Review

FDA approves 1st gene therapy for Wiskott-Aldrich syndrome

FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.
MedPage Today on MSN

Gene therapy wins FDA approval for life-threatening immunodeficiency

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...
BioSpace

FDA Greenlights First Gene Therapy for Rare Pediatric Disease

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

FDA approves first gene therapy for rare immune disorder

The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...
STAT

Gene therapy spurs improvement in rare neuromuscular disease

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

Teen Beats Deadly Leukemia With Life-Saving Gene-Edited Cell Therapy

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...
NJBIZ

FDA approves Novartis’ Itvisma gene therapy for SMA

Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...
Fierce Pharma

ASH: CSL proves efficacy of hemophilia B gene therapy Hemgenix holds up after 5 years

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...
BioSpace

Novartis Expands Spinal Muscular Atrophy Gene Therapy Market With FDA’s Itvisma Greenlight

The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor ...
Fierce Biotech

Regeneron makes $150M bet on Tessera's rare disease gene writing prospect

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene writing program, inking a deal that lets it in on a ...
FiercePharma

FDA restricts bluebird bio gene therapy Skysona after blood cancer reports

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...
News-Medical.Net on MSN

EpilepsyGTx raises $33 million Series A to develop single dose gene therapy for focal refractory epilepsy

EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, today announced it has raised $33 million in a Series A financing ...