Gene Therapy Summit

About the Summit

ASTCT introduces the Gene Therapy Summit, an interactive meeting of representatives from academic and clinical centers, the biotechnology industry, ASTCT and additional collaborative professional societies focused on ex vivo gene therapy using hematopoietic stem cells.

The overarching goal of the Summit is to explore how the strengths of ASTCT centers and members can be leveraged to bring effective therapies safely and rapidly to patients while advancing the field of ex vivo gene therapy. Summit sessions will bring together experts from multiple disciplines to educate, discuss and exchange best practices, and to provide a platform for biotechnology industry representatives to interact with academic clinicians and researchers.

The 1.5 day summit will be held September 10-11, 2025, at the Joseph B. Martin Conference Center at Harvard Medical School in Boston, Massachusetts. The Summit is not accredited and will not offer any continuing education credit.

Who Should Attend

Clinicians
Advanced Practice Providers
Nurses
Pharmacists
Administrative Directors
Multidisciplinary stakeholders at centers involved or considering involvement in gene therapy

Key Information

The program will include an evening networking reception and keynote lecture, general sessions and concurrent sessions. 2025 topics include:

State of ex vivo HSC gene therapy
Pathway from research through commercialization
Therapeutic apheresis for different patient types and gene therapy platforms
Patient outreach, support and engagement
Long-term follow up
Society, biotechnology industry and academic collaborations

Agenda

Schedule is subject to change.

Day 1: Wednesday, September 10

Day 2: Thursday, September 11

Day 2 AGENDA
8:00 – 8:05 AM	Welcome and Summit Goals Speaker: Christine Duncan, MD; Dana-Farber/Boston Children's Cancer and Blood Disorders Center
8:05 AM – 9:35 AM	Concurrent 1: Mobilization and Apheresis for Gene Therapy: 201 Session Chair: Suzanne Thibodeaux, MD, PhD; Washington University Topics: Operational and Logistical Challenges of Gene Therapy for Sickle Cell Disease Strategies to Potentially Improve Mobilization and Collection with SCD Gene Therapy Experiences with Pediatric Mobilization Practices Speakers: Yvette Tanhehco, MD, PhD, MS; Columbia University Patricia Shi, MD; New York Blood Center Enterprises Cyril Jacquot, MD, PhD; Children's National Hospital	Concurrent 2: Administrative Focus Session Chair: Colleen Dansereau, MSN, RN, CPN; Boston Children's Hospital Topics: CMS CGT Model Hospital Perspective on Risk/Benefit of Onboarding New High-Cost Therapies Cost Recovery Speakers: Alyce Norcross, MPH; Boston Children's Hospital Donna Casey, SVP; Boston Children's Hospital Rayne Rouce, MD; Baylor College of Medicine
9:35 – 10:05 AM	Break
10:05 – 11:00 AM	The Parent's Journey Through Drug Development: How to Develop Cell and Gene Therapy for a Rare Disease Such as Angelman Syndrome Speaker: Allyson Berent, DVM, DACVIM; Foundation for Angelman Syndrome Therapeutics (FAST) Moderator: Matthew Porteus, MD, PhD; American Society of Gene and Cellular Therapy President-Elect, Stanford University
11:00 – 12:00 PM	Lunch
12:00 – 1:30 PM	Concurrent 3: Moving Beyond Busulfan Conditioning Session Chair: Sung-Yun Pai, MD; National Institutes of Health Topics: Melphalan Conditioning in Stem Cell Gene Therapy for Hemophilia A Briquilimab-Based Conditioning and Allogeneic Transplant for Fanconi Anemia Multiplex Editing Including c-Kit Epitope Editing in Nonhuman Primate Model of Sickle Cell Disease Speakers: David Wilcox, PhD; Medical College of Wisconsin Rajni Agarwal, MD; Stanford Medicine Selami Demirci, PhD; National Institutes of Health	Concurrent 4: Accreditation and Onboarding: Is an 80-20 Model Possible in Gene Therapy? Session Chair: Sarah Nikiforow, MD, PhD; Dana-Farber Cancer Institute Topics: Introduction and Overview of the 80:20 Model in CAR T How To Administer and Accredit a Gene Therapy Program Industry-Academic Tensions in Oversight Panel Discussion Speakers: Sarah Nikiforow, MD, PhD; Dana-Farber Cancer Institute Robert Richards, MSBI, MBA; Penn Medicine Vinod K. Prasad, MD, FRCP; bluebird bio Panelists: Robert Richards, MSBI, MBA; Penn Medicine Jose Cancelas, MD, PhD, CABP; Dana-Farber Cancer Institute and AABB Incoming President David Schmahl; FACT Chief Executive Officer Vinod K. Prasad, MD, FRCP; bluebird bio
1:30 – 2:00 PM	Break
2:00 – 2:45 PM	Concurrent Session Recaps Speakers: All Concurrent Session Chairs
2:45 – 3:30 PM	Managing Unexpected and Adverse Outcomes Roundtable Session Chairs: Hemalatha Rangarajan, MD; Nationwide Children’s Hospital; Rayne Rouce, MD; Baylor College of Medicine Panelists: Colleen Dansereau, MSN, RN, CPN; Boston Children's Hospital David Jacobsohn, MD, SCM, MBA; Children's National Mark Leick, MD; Massachusetts General Hospital Kathleen O'Sullivan-Fortin, Esq.; ALD Connect John Tisdale, MD; National Institutes of Health
3:30 – 3:40 PM	Closing Remarks

Housing Information

ASTCT does not have a formal room block but recommends the following nearby hotels:

Sponsorship

If you are interested in sponsoring this event, please contact Angie Dahl at adahl@astct.org.