This inaugural virtual event March 12 and 13, 2025 (9 a.m. - 12:30 p.m. ET on both days), will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCT’s , the goal is to empower advocates, patients, and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field, as well as patient advocacy group leaders. There will be opportunities to ask questions and deepen your knowledge of the community’s CGT journey.
In advance of the Summit, visit ASGCT’s resources on the Patient Education website and subscribe to The Patient Press, to stay up to date on latest events, resources, and news relevant to the patient community.
Agenda
March 12, 2025
Event Moderator: Allison Bradbury, PhD, Nationwide Children's Hospital
Partners in Progress: The Value of Patients and Advocates
9 – 9:15 a.m.
The opening address will spotlight the important role patients, caregivers, and advocates have in advancing CGTs for rare diseases and disorders.
- Paula Cannon, PhD, University of Southern California, ASGCT President
Intro to Gene Therapy Approaches
9:15 – 9:45 a.m.
This session will review the science behind some common treatment approaches (Gene addition, gene editing, CAR-T, RNA, ASO), along with the unique risks, challenges, and potential benefits of CGTs.
- Steven Gray, PhD, University of Texas Southwestern
Why Are Clinical Trials for Rare Diseases Different?
9:45 – 10:15 a.m.
There are important considerations for any person thinking of participating in an experimental CGT trial, especially for those with limited time and treatment options. This talk will introduce the clinical development pipeline, from pre-clinical studies all the way to FDA approval. Dive into learning how CGT trials differ from the traditional drug development framework.
- Amy Waldman, MD, Children's Hospital of Philadelphia
Complexity of Study Design in Cell and Gene Therapy
10:25 - 11:25 a.m.
The research team makes several complex decisions when designing a clinical trial including, but not limited to, determining eligibility, dosage, and the use of placebo or external controls. This session will feature a thoughtful discussion with clinical trial experts to illuminate how clinicians make those decisions and their impact on potential participants.
- Raymond Wang, MD, Children's Hospital Orange County
- Heather Lau, MD, Ultragenyx
- Moderator: Sharon King, Aldevron, Taylor's Tale
When Innovation Stumbles + Pathways Forward
11:30 a.m. - 12:25 p.m.
This session will explore the state of commercially pre-viable therapies (sometimes referred to as dropped or deprioritized trials). We will hear both a clinician’s take on why this happens and potential solutions, and from a patient advocate who has dealt with this challenge.
- Terry Flotte, MD, University of Massachusetts
- Cara O'Neill, MD, Cure Sanfilippo Foundation
Closing Remarks
March 13, 2025
Event Moderator: Kimberly Goodspeed, MD, Ultragenyx
Morning Coffee with Dr. Peter Marks
9:05 - 9:50 a.m.
This live sit-down with FDA leadership will share insights on Agency efforts for rare disease drug development. The discussion will touch on patient experience data for regulatory pathways, the new Rare Disease Innovation Hub, expedited pathways, and more.
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Peter Marks, MD, PhD, Food and Drug Administration – Center for Biologics Evaluation and Research (FDA CBER)
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Moderator: Kristen Van Goor, PhD, Takeda
CGT Manufacturing Basics
9:55 - 10:25 a.m.
This presentation will break down the complex and critical role of manufacturing for CGTs. The speaker will first cover the basics of Chemistry, Manufacturing, and Controls (CMC) in an accessible way. Discussion will also include how manufacturing is linked to clinical trials and why CGTs lend themselves to bespoke manufacturing, why CMC issues can be a major hurdle for early-stage therapies, and current efforts to address manufacturing bottlenecks.
- Amy Raymond, PhD, Worldwide Clinical Trials
The Journey from Early-stage Trials to Approval
10:40 - 11:45 a.m.
In this live panel discussion, we’ll hear from patient advocate leaders in varying disease areas who are at different points in the CGT journey. The discussion will span funding first research initiatives, through approaching a CGT approval. Panelists will share their experiences and insights on how to overcome potential obstacles along the road to treatment.
- Brendan Hayes, National Bleeding Disorder Foundation
- Amanda Moore, Angelman Syndrome Foundation
- Kelly Brazzo, CureLGMD2i Foundation
- Sarah Cortell Vandersypen, United MSD Foundation
Exploring Goals of Care and Personal Values when Considering CGT Trials
11:50 a.m. - 12:20 p.m.
It is important to manage participant and family expectations for any potential CGT clinical trial. Patients, caregivers, clinicians, and advocates often must engage in challenging discussions when considering an investigational treatment. This talk will outline some of those common dilemmas and offer advice to help navigate difficult conversations.
- Jennifer Siedman, Courageous Parents Network
- Megan Maack, Childhood Dementia Initiative
Closing Remarks