FDA approves first gene therapy for rare immune disorder

Dec 9 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and ...

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 ...

Children born with deadly immune flaw get powerful new gene therapy option

Gene therapy uses the patient's own blood stem cells, which were genetically modified to include functional copies of the WAS gene, the FDA said.
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 ...
Labroots

Novel Gene Therapy Treats T Cell Leukemia

Scientists at the University College London (UCL) have developed a novel therapy that helps treat patients with T cell acute ...
Good News Network

Patients Thought Untreatable with Rare Disease Dramatically Improve with Common Gene Therapy

The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...
News Medical

Gene activity can predict severe side effects from melanoma treatment

An activity pattern in certain genes responsible for building proteins known as spleen tyrosine kinases can predict which melanoma patients are likely to have severe side effects from immunotherapy ...
pharmaphorum

FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon ETS' Waskyra has become the first gene therapy in the US for patients with Wiskott-Aldrich syndrome (WAS) ...
STAT

Gene therapy spurs improvement in rare neuromuscular disease

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

Abeona Therapeutics® Announces First Patient Treatment with ZEVASKYN® Gene Therapy

Treating our first patient is a proud moment for Abeona and a testament to the tireless resolve of our team,” said Vish ...

Gene therapy improves movement in kids with spinal muscular atrophy

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
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 ...