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Scientists Discover Way to Send Information into Black Holes Without Using Energy

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FDA Approved Worlds First Gene Therapy For Rare Immune Disorder Wiskott-Aldrich Syndrome

In a groundbreaking move, the U.S. Food and Drug Administration (FDA) has approved etuvetidigene autotemcel, marketed as Waskyra, the first-ever gene therapy for Wiskott-Aldrich syndrome (WAS). This milestone brings new hope to patients and families affected by this rare and life-threatening immune disorder.

A Transformative Treatment for a Rare Disease

Wiskott-Aldrich syndrome is a genetic condition that mainly affects boys, occurring in 1 to 10 per million. It causes severe problems with the immune system and blood clotting, leading to:

  • Frequent and serious infections

  • Easy bleeding and bruising

  • Eczema and other skin issues

Until now, treatment options were limited—primarily supportive care or stem cell transplants from matched donors. Waskyra changes that landscape.

How the New Gene Therapy Works

The therapy uses the patient’s own blood-forming stem cells, which are:

  1. Collected from the patient

  2. Genetically corrected in the lab to add a functional version of the WAS gene

  3. Returned to the patient to rebuild a healthier immune system

This approach reduces the risks associated with donor stem cell transplants, including rejection.

Strong Clinical Benefits

The FDA approval was based on data from two open-label studies and an expanded access program involving 27 patients. The results were impressive:

  • 93% reduction in severe infections within the first 6–18 months

  • 60% reduction in moderate and severe bleeding within the first year

  • Improved overall survival and quality of life

  • No treatment-related serious side effects or signs of insertional oncogenesis (a concern in earlier gene therapy trials)

Most side effects were mild to moderate, including rash, vomiting, diarrhea, and respiratory infections.

Who Can Receive Waskyra?

The therapy is approved for:

  • Children 6 months and older

  • Adults with confirmed mutations in the WAS gene

The approval was granted to Fondazione Telethon ETS, an Italian nonprofit dedicated to rare disease research.

A New Era for Rare Disease Treatment

FDA chief medical and scientific officer Vinay Prasad emphasized the agency’s flexible approach in reviewing data for rare conditions, including evidence from expanded access programs.

The approval of Waskyra (etuvetidigene autotemcel) marks a major step forward not just for WAS patients, but for the entire field of gene therapy. It showcases how precision genetic medicine can reshape the future of treatment for rare and complex diseases.

Provided by Reuters

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