ASGCT News

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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The When, What, How, and Why of FDA Engagement with Niki Gallo

Listen to The Issue on the ASGCT Podcast Network! | March 11, 2025

Whether you’re from industry or academia, today’s conversation will help wrap all our heads around testing novel therapies for the first time in humans.

Podcasts

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Incorporating miRNAs into a taRNA vector platform with Drs. Tim Beissert and Ayşegül Yildiz

Listen to this episode of the Molecular Therapy Podcast! | March 04, 2025

Drs. Ayşegül Yildiz and Tim Beissert join Dr. Paloma Giangrande to discuss a recent article published in Molecular Therapy Nucleic Acids.

Molecular Therapy

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Exploring AAV Immune Responses in Gene Therapy: Insights from ASGCT's Community Quick Take

Alexis Starosta | March 03, 2025

ASGCT’s latest Community Quick Take broke down insights from a recent ASGCT workshop, Class Considerations on Immunogenicity for AAV Gene Therapy Products.

Outreach

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Uniting for Rare Disease Day: Advancing Research and Empowering Patients

Alexis Starosta | February 27, 2025

Rare Disease Day on Feb. 28 is more than just a day. It is a call to action for us to support research, push for policy changes, and empower patients in their journey toward treatments.  

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March 12 + 13: Join CGT Advocates, Caregivers at Empowering Patients Summit

Allison M Bradbury, MS, PhD | February 18, 2025

Explore valuable insights from foundation leaders, patient advocates, alongside investigators and clinicians who are mission-focused on CGT development. Register today!

Events  |  Outreach

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Submit a Late-breaking Abstract through March 14

We want to see your late-breaking research at #ASGCT2025. | February 17, 2025

We want to see your late-breaking research at the 28th Annual Meeting!

Annual Meeting 2025

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Trust, Innovation, and the Power of Patient Advocacy with Brendan Hayes

Listen to The Issue on the ASGCT Podcast Network! | February 11, 2025

On this episode of The Issue, Brendan and host Emily Walsh Martin dive into how patient communities shape the development of new therapies, the historical context that influences decision-making in clinical trials, and more.

Podcasts

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Ethical Issues in Pediatric Gene Therapy: Multistakeholder Approaches

Samantha Rose and Ayden Eilmus | January 28, 2025

Open dialogue among stakeholder groups is thus vital to the ethical development and use of these interventions in children, both in clinical trials and in the clinic.

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