Nature

‘Giant step forward’ for Huntington’s — the scientist behind the first gene therapy

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
MedPage Today on MSN

Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders

Exa-cel is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of gene editing technology through which patients' ...
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 ...

UK doctors develop groundbreaking new gene therapy to treat blood cancers

Doctors in the United Kingdom developed a groundbreaking new gene therapy that is giving hope to patients with aggressive ...

World-first gene therapy reverses 'incurable' leukemia in some patients

CAR7, has shown remarkable success in reversing an aggressive form of leukemia. This world-first treatment uses gene ...
Fierce Pharma

Italian nonprofit lands FDA approval for first gene therapy to treat rare genetic disease

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

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 ...
MedPage Today

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 ...
STAT

In a first, a nonprofit wins FDA approval for rare disease gene therapy

The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
BioPharma Dive

Hemophilia gene therapies are struggling on the market, even as innovation soars

Hemophilia gene therapies arrived a few years ago with blockbuster expectations. But now, one therapy has been pulled from ...

She Was Born Without an Immune System. Gene Therapy Saved Her Life.

Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
Medscape

First Patient With Recessive Dystrophic Epidermolysis Bullosa Treated With New Gene Therapy

The first-of-its kind gene therapy uses a patient’s skin cells to create sheets to treat the open wounds caused by the rare, ...