ASGCT News

FDA Cell & Gene Therapy Roundtable: Putting Every Patient Within Reach of Innovation

Terry Flotte, MD, and Paula Cannon, PhD | June 11, 2025

Roughly 15 million Americans - about half of them children - live with rare genetic disorders, yet more than 100 clinical stage gene therapy programs have been shelved since early 2023.

Policy & Advocacy

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25 Years of Molecular Therapy with Sheila Mikhail and Dr. Mark Kay

Listen + subscribe to the ASGCT Podcast Network! | June 11, 2025

In this episode, Dr. Glorioso discusses two recent reviews published in Molecular Therapy with Sheila Mikhail and Dr. Mark Kay.

Molecular Therapy  |  Podcasts

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ASGCT Builds Bridges: Advancing Access Through Patient-Centered and Global Initiatives

Caitlin McCombs, MBA; Ali Kujawski, MPH; and Lexi Starosta | June 10, 2025

During #ASGCT2025, ASGCT reinforced its commitment to patient-centered approaches through multiple sessions designed to elevate patient voices and address access challenges for cell and gene therapies (CGTs). 

Annual Meeting 2025  |  Outreach  |  Policy & Advocacy

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ASGCT Congressional Briefing Highlights Transformative Potential of Gene Editing Technologies

Paula Cannon, PhD | June 10, 2025

Gene editing represents a paradigm shift in medicine, offering the possibility to address the root genetic causes of disease rather than just treating symptoms.

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From Trials to Triumph: Patient Advocates Share the Path from Research to Approval

Alison Kujawski, MPH | June 09, 2025

Check out our summary of a standout session during ASGCT's Empowering Patients Summit, The Journey from Early-stage Trials to Approval.

Outreach

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2025 Award Applications Open Through Aug. 1

ASGCT members can apply for nearly $2 million in awards! | June 06, 2025

This awards season, ASGCT will provide up to nearly $2 million in funded awards to members. If you're a researcher growing your career, we invite you to take advantage of these opportunities!

Awards

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Thank You for Attending the 28th Annual Meeting!

Registrants can watch sessions on demand through July 18. | May 13, 2025

Did you attend #ASGCT2025 in person or virtually? Let us know how we did!

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Critical Juncture in Hemophilia Treatment: Global Organizations Issue Urgent Call to Action

Read the full statement endorsed by ASGCT. | May 06, 2025

The ISTH, EAHAD, and WFH urge continued innovation and equitable access in hemophilia care.

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25 Years of Molecular Therapy with Drs. Terry Flotte and Carl June

Listen + subscribe to the Molecular Therapy Podcast! | May 01, 2025

In honor of our flagship journal's 25th anniversary, EIC Dr. Joseph Glorioso will cover two reviews of critical developments in the field with Drs. Terry Flotte and Carl June.

Molecular Therapy  |  Podcasts

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FDA Approves First Cell-Based Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

Paula Cannon, PhD | April 29, 2025

Zevaskyn, also known as pz-cel is the first autologous, cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB).

Cell & Gene Therapy Approval

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Now Available: 28th Annual Meeting Abstracts

Read + download the latest CGT reserach | April 28, 2025

Read the most innovative science in the field, with 2,200 pieces of original research—over 10% more than last year! 

Annual Meeting 2025  |  Publications

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Congratulations to Our New Board Members!

These incoming BOD members take office June 1. | April 18, 2025

The incoming members of the Board of Directors will assume their roles on June 1, 2025.

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Read the ASGCT-Citeline 2025 Q1 Landscape Analysis Field Report

Read + download the latest report | April 17, 2025

In Q1 2025, one new therapy was approved for each of the gene, cell, and RNA categories.

Publications

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The Importance of Long-term Data with Dolan Sondhi, PhD

Listen to The Issue on the ASGCT Podcast Network | April 14, 2025

Emily is joined by Dolan Sondhi, PhD, of Weill Cornell Medical College, to discuss her recent paper, “Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease.”

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Vote in ASGCT's 2025 Election

Members + Transitional Members can vote through April 11. | March 17, 2025

We need your help to select five new members of the ASGCT Board of Directors!

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FDA Approves Encapsulated Cell Therapy for Degenerative Eye Disease

Paula Cannon, PhD | March 17, 2025

Encelto is the first and only approved treatment for MacTel type 2.

Cell & Gene Therapy Approval

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