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 ...
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 ...
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 ...
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 ...
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 ...
Fitgurú on MSN

Incurable cancer vanishes: Radical new gene therapy clears disease in 64% of patients

A pioneering genetic treatment is saving lives where chemotherapy failed, offering new hope for children and adults with ...
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 ...

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 ...
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 ...
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 ...
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, ...