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 ...

Fondazione Telethon Announces FDA approval of Waskyra(TM) (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome

ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...
The American Journal of Managed Care

Hurdles Remain Despite Progress in Gene Therapy for DMD

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Boy with rare condition amazes doctors after world-first gene therapy

Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.
Reuters

Roche 'steps up' for gene therapy with $4.3 billion Spark bet

ZURICH (Reuters) - Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to "step up", Chief Executive ...
News Medical

The Latest Advances in Cell and Gene Therapy

Cell and gene therapies (CGTs) represent the cutting edge of drug discovery. 1 These are amongst the most advanced medical treatments, specifically designed to correct the root genetic cause of an ...
The Conversation

Gene therapy can be less effective in women – and my research in mice brings us one step closer to understanding why

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...
Saint Louis University

Ethical, Legal and Social Implications: Bringing Gene Therapies to the Clinic

Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...
Hosted on MSN

'Will it really work?': Young sickle cell patient among the first to start new gene therapy

WASHINGTON — For Wedam Minyila, hospital rooms have always meant blinding pain. “Like someone is jamming a knife in me,” he said. But for a brief moment on a recent December morning, Wedam, 19, who ...

Study offers real-world data on commercial implementation of gene therapies for sickle cell disease, beta thalassemia

A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...
New Atlas

95% of kids with “bubble boy” disease cured by one-time gene therapy

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...
Finanznachrichten

Fondazione Telethon Announces FDA approval of Waskyra (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome

WAS affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. The therapy represents a major scientific and clinical achievement, offering new hope for patients ...