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

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. 

ASGCT Co-Hosts Congressional Briefing on Progressing Gene Therapy for Sickle Cell Disease
June 20, 2019

On Tuesday, June 18, ASGCT co-hosted a congressional briefing on sickle cell disease with Sens. Tim Scott and Cory Booker addressing a standing-room only audience of nearly 100 congressional staff members and other stakeholders about progress in gene therapy and gene editing for sickle cell disease and the policy implications surrounding the emerging technologies.

ASGCT Heats Up in Summer 2019
June 14, 2019
Advocacy ASGCT News

Up-to-date clinical trials info and a new policy-centered event keep up the post-meeting momentum.

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

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. 

22nd Annual Meeting Recap
May 16, 2019
Annual Meeting 2019

Edith Pfister, Ph.D., associate editor of ASGCT's newsletter The Vector, not only attended the record-breaking ASGCT 22nd Annual Meeting but presented an abstract on the meeting's final day. Read about her experience and recap an incredible week of science.

The Case for Growth

Following a record-breaking ASGCT Annual Meeting, the Society is ready to branch out and ensure its growth is sustainable.

2019
ASGCT Policy Summit
November 4 – 6 | Washington D.C.
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