Gene Therapy Wins FDA Approval for Life-Threatening Immunodeficiency

FDA Flexibility Leads to First Approved Gene Therapy for Rare Blood & Immune System Disorder

Waskyra is the first FDA-approved gene therapy for the rare disease Wiskott-Aldrich syndrome and the first such approved product from a non-profit applicant, Fondazione Telethon. Data from Waskyra’s pivotal studies were presented this week during the annual meeting of the American Society of Hematology. The post FDA Flexibility Leads to First Approved Gene Therapy for Rare Blood & Immune System Disorder appeared first on MedCity News.

Gene Therapy Wins FDA Approval for Life-Threatening Immunodeficiency

(MedPage Today) -- The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and older with Wiskott-Aldrich syndrome, a rare primary immunodeficiency. The life-threatening genetic...

FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

FDA Approves Etuvetidigene Autotemcel, First Gene Therapy for Wiskott-Aldrich Syndrome

The FDA approved etuvetidigene autotemcel, the first gene therapy for Wiskott-Aldrich syndrome, transforming treatment options for affected patients.

In a first, FDA approves a gene therapy from a nonprofit

The FDA on Tuesday approved a gene therapy for a rare immune disease called Wiskott-Aldrich syndrome, for which patients have little options besides a bone marrow transplant. The gene therapy will be marketed as Waskyra ...