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
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.
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.
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.
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.
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 discontinued 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.
Closing Remarks
March 13, 2025
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.
Developing Gene Therapies as Platform Technologies
9:55 - 10:25 a.m.
This session will dive into the concept of platform technologies to streamline CGT development. Speakers will explain how platforms work in CGTs and how public-private partnerships are helping to lay the tracks for developers to use.
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.
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.
Closing Remarks