Fierce Pharma

Italian nonprofit lands FDA approval for 1st 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 ...
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-induced CAR T cells for myeloma could alter treatment landscape

MRD-negative status in first four heavily treated patients with "off the shelf" product ...
Fierce Healthcare

Aradigm launches out of stealth with platform for cell, gene therapy benefits

Aradigm Health, a new benefits platform aimed at easing the financial sting of coverage for cell and gene therapies, has ...
MedPage Today on MSN

Gene Therapy Maintains Efficacy in Hemophilia B Out to 5 Years

Etranacogene dezaparvovec meets the benchmarks for success, researcher says ...
STAT

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

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

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 ...
BioSpace

FDA Minutes Confirm Challenging Path Ahead for UniQure’s Huntington’s Gene Therapy

Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...
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 ...
MedPage Today on MSN

Gene Therapy Wins FDA Approval for Life-Threatening Immunodeficiency

In Wiskott-Aldrich syndrome trials, therapy was associated with decreases in bleeding, inf ...

FDA approves first gene therapy for rare immune disorder

Dec 9 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and ...
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, ...