Cell & Gene Therapy Approval

FDA Approves Lenmeldy for Metachromatic Leukodystrophy

Jeffrey S. Chamberlain, PhD | March 18, 2024

Lenmeldy is the first and only treatment in the U.S. for early onset MLD.

Cell & Gene Therapy Approval
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FDA Approves First Gene Therapy to Treat Severe Hemophilia A

Jeffrey Chamberlain, PhD | June 29, 2023

Roctavian is the first FDA-approved gene therapy for adults with severe hemophilia A.

Cell & Gene Therapy Approval
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FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

Jeffrey Chamberlain, PhD | June 22, 2023

The approval of Elevidys marks the first time a gene therapy has been approved for any form of this severely debilitating disease.

Cell & Gene Therapy Approval
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FDA Approves First Gene Therapy for Dystrophic Epidermolysis Bullosa (DEB)

Hans-Peter Kiem, MD, PhD | May 24, 2023

Vyjuvek is the first FDA-approved gene therapy for dystrophic epidermolysis bullosa (DEB) and the first topical gene therapy approved in the U.S. It's indicated for use in patients 6 months of age or older.

Cell & Gene Therapy Approval
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FDA Approves First Gene Therapy for Bladder Cancer

Hans-Peter Kiem, MD, PhD | December 21, 2022

Adstiladrin is the first FDA-approved gene therapy for bladder cancer. It's indicated for people with high-risk superficial bladder cancer unresponsive to Bacillus Calmette-Guérin immunotherapy. 

Cell & Gene Therapy Approval
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FDA Approves First Gene Therapy for Hemophilia B

Hans-Peter Kiem, MD, PhD | November 22, 2022

The approval of Hemgenix is the first of its kind for hemophilia B patients, who typically control the disease with regular infusions of the clotting factor they lack.

Cell & Gene Therapy Approval
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FDA Approves SKYSONA, Second Lentiviral Vector Gene Therapy in the U.S.

Hans-Peter Kiem, MD, PhD | September 20, 2022

The approval of SKYSONA® comes one month after the first lentiviral vector gene therapy, Zynteglo, was approved for use in the U.S.

Cell & Gene Therapy Approval
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Beti-Cel Becomes the First Lentiviral Vector Gene Therapy in the U.S.

Hans-Peter Kiem, MD, PhD | August 17, 2022

The approval of beti-cel represents a huge step forward for beta thalassemia patients, giving them the option to pursue a one-and-done treatment approach.

Cell & Gene Therapy Approval
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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  |  Cell & 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.

Cell & 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.

Cell & 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.”

Cell & Gene Therapy Approval  |  Kymriah  |  Policy Updates
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2024

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September 23-24, 2024 | Washington, D.C.

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