Gene Therapy Approval

FDA Approves Zolgensma, Landmark AAV-Delivered Gene Therapy to Treat Spinal Muscular Atrophy

May 24, 2019

Zolgensma (Novartis, AveXis), an AAV-delivered gene therapy used to treat spinal muscular atrophy (SMA) also known as AVXS-101, was approved for clinical use in the United States by the Food and Drug Administration today. 

Annual Meeting 2019   |   ASGCT News   |   Gene Therapy Approval
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Spark Therapeutics leadership Dr. Kathy High and Jeff Marrazo in the laboratory. Credit: Spark Therapeutics

ASGCT Celebrates FDA Approval of Voretigene Neparvovec (Luxturna) as Treatment for Inherited Retinal Disease

December 19, 2017

Groundbreaking gene therapy is the first AAV viral vector product to be approved for use in the United States.

Gene Therapy Approval   |   Luxturna
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First CAR T Cell Therapy for Adults Approved by FDA

ASGCT Staff | October 20, 2017

With Wednesday’s FDA approval of axicabtagene ciloleucel (Yescarta®, Kite Pharma, Inc/Gilead), the administration has put into clinicians’ hands the first CAR T cell therapy for adult patients with relapsed or refractory large B-cell lymphoma.

Gene Therapy Approval   |   Policy Updates   |   Yescarta
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Scanning electron micrograph of a human T lymphocyte (also called a T cell) from the immune system of a healthy donor. Credit: NIAID (CC BY 2.0)

FDA Approves First CAR T Cell Treatment for Acute Lymphoblastic Leukemia

August 30, 2017

“We are incredibly excited to see a CAR T-cell treatment come to patients in the United States,” Dr. Helen E. Heslop, ASGCT President and Director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital says. “This remarkable approval is the beginning of what we see as a chance to transform the way in which we treat cancer.”

Gene Therapy Approval   |   Kymriah   |   Policy Updates
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ASGCT 25th Annual Meeting

May 16-19, 2022

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