ASGCT News

Join Leading Experts for an FDA-ASGCT Workshop on AAV Gene Therapy Immunogenicity

Andrew Liermann | January 13, 2025

This Jan. 22-23 event brings together thought leaders and regulatory experts for two half-days of in-depth discussion on pressing challenges and emerging solutions in the field.

Policy & Advocacy

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Watch FDA's Patient Listening Session on Early Enrollment into Gene Therapy Trials for Rare Diseases

Ali Kujawski, MPH and Alexis Starosta | January 10, 2025

FDA CBER recently held the second patient listening session to hear from patients and caregivers about early enrollment in CGT clinical trials. Read this recap and watch the session on demand.

Outreach

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RNA Exon Editing with Ascidian Therapeutics' Drs. Robert Bell and Michael Ehlers

Listen to the latest Molecular Therapy Podcast! | January 07, 2025

This conversation explores Dr. Bell's recent MTNA publication "RNA Exon Editing: Splicing the Way to Treat Human Diseases."

Podcasts

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The Power of Patient Voices: A Preview of the Empowering Patients Summit

Alexis Starosta, Advocacy Programs Manager  | December 20, 2024

Patients, advocates, and caregivers bring essential perspectives to the table, shaping the future of treatments in ways others cannot. Their lived experiences, challenges, and hopes are the heart of the drug development process.

Outreach

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FDA Approves RNA-targeted Therapy for Familial Chylomicronemia Syndrome

Paula Cannon, PhD | December 19, 2024

Tryngolza is the first approved treatment in the U.S. for FCS, which prevents the body from breaking down fats. The treatment is an antisense oligonucleotide (ASO).

Cell & Gene Therapy Approval

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Congressional Briefing Highlights CGT Research for HIV

Paula Cannon, PhD | December 18, 2024

We heard from two researchers as well as Paul Edmonds, who shared his experience as one of the few people who have been cured of HIV.

Policy & Advocacy

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Advancing CGTs in 2024: ASGCT’s Policy and Advocacy Highlights

Caitlin McCombs, MBA | December 18, 2024

Read our recap on policy initiatives and significant milestones of 2024.

Policy & Advocacy

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Reaching Our Community of Stakeholders: ASGCT’s Outreach Highlights

Alexis Starosta | December 18, 2024

Review the highlights from our patient and global initiatives guided by members throughout the year. 

Outreach

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Cell Therapy Development for Solid Tumors with AffyImmune's Sonal Gupta, MD, PhD

Listen to this episode of The Issue now! | December 10, 2024

Sonal Gupta, MD, PhD talks with host Emily Walsh Martin, PhD, about the challenges of cell therapy clinical trials.

Podcasts

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Community Quick Takes: What Is Focused Ultrasound?

Explore focused ultrasound technology in CGT | December 06, 2024

We explore how non-invasive focused ultrasound technology can enhance the delivery of cell and gene therapies in treating various diseases. This content was developed in partnership with the Focused Ultrasound Foundation.

Outreach

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Navigating Challenges in AAV Gene Therapy: A Preview of the FDA-Partnered Workshop

Andrew Liermann | December 03, 2024

The upcoming Class Considerations on Immunogenicity for AAV Gene Therapy Products workshop on Jan. 22 and 23 will address some challenges facing the class of AAV gene therapies.

Policy & Advocacy

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28th Annual Meeting Registration Is Open

Secure your spot in New Orleans, May 13-17! | December 03, 2024

Join us in New Orleans May 13-17 for the largest gathering of CGT professionals at the 28th Annual Meeting! 

Annual Meeting 2025

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Single-chain Antibody-directed Herpes Simplex Viruses with Joseph Glorioso, PhD

Listen to The Molecular Therapy Podcast | December 03, 2024

In this episode, Dr. Timothy Cripe and Dr. Joseph Glorioso discuss an article published in Molecular Therapy Oncology by Dr. Glorioso and colleagues, Oncolytic Herpes Simplex Viruses Designed for Targeted Treatment of EGFR-bearing Tumors.

Podcasts

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Congratulations to our Honorific and Funded Award Winners

Learn more about the award recipients on our blog. | December 02, 2024

Meet the recipients of our honorific and funded ASGCT awards.

Awards

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ASGCT-AAAS Congressional Policy Fellow Reflects on First Weeks

Adriana Bankston, PhD | November 19, 2024

Learn what the first few weeks of the Congressional Policy Fellowship have been like for Dr. Adriana Bankston and apply for next year's fellowship through Feb. 24.

Policy & Advocacy

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Breakthroughs in Muscular Dystrophy Conference Networking Tips

Use these networking tips to connect with colleagues next week! | November 15, 2024

Here are some tips for making the most out of our upcoming muscular dystrophy conference.

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