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 ...
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 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 ...

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 ...
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.

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

This gene therapy costs $5 million a dose. Is it worth the price?

A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...