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 ...
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 ...
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 ...
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 ...

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 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 ...
ABC7 KABC on MSN

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.
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 ...
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 ...
Science Daily

Gene-edited CAR-T cells erase aggressive T-cell leukemia

A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...