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 ...
MedPage Today on MSN

Gene Therapy-Induced CAR T Cells for Myeloma Could Alter Treatment Landscape

ORLANDO -- A gene therapy that could transform CAR T-cell treatment achieved rapid and potentially durable measurable ...

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 on MSN

Gene Therapy Maintains Efficacy in Hemophilia B Out to 5 Years

Based on initial positive results from HOPE-B, etranacogene dezaparvovec became the first gene therapy approved for adults ...
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 ...
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 ...
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 ...
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 ...
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.
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 ...
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 ...

Experimental gene therapy treatment created at UCLA gives 'bubble girl' a new life

An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.