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 Harvard Medical Center in Boston, Massachusetts. 

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.

  • 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

Registration

Register

Registration Rates
Type Price
Government Employee Rate $100
ASTCT Member Rate $250
AABB Member Rate $250
ASGCT Member Rate $250
Non-Member Rate $350
Industry Rate $850

 

Agenda

Day 1: Wednesday, September 10
Day 1 AGENDA
5:30 – 6:30 PM

Welcome Reception
6:30 – 7:00 PM

Keynote Speaker: To be announced
Day 2: Thursday, September 11
 
Day 2 AGENDA
9:00 – 9:05 AM Welcome and Summit Goals
9:05 – 10:05 AM

Exploring Abandoned Products in Rare Diseases

Moderators: Matthew Porteus, MD, PhD - American Society of Gene and Cellular Therapy Vice-President, Stanford University; David Barret, JD - American Society of Gene and Cellular Therapy CEO
10:05 – 10:30 AM Break
10:30 AM – 12:00 PM

Concurrent 1: Mobilization and Apheresis for Gene Therapy: 201

Moderator: Suzanne Thibodeaux, MD, PhD - Washington University; Jeff Wren, MBA - AABB

Topics:

  • Clinical Practices in Mobilization and Apheresis
  • Special Considerations for Pediatric Patients
  • Quality Oversight in Gene Therapy Processes
  • Operational Challenges in Mobilization and Apheresis

Concurrent 2: Administrative Focus

Moderator: Colleen Dansereau, MSN, RN, CPN - Boston Children's Hospital

Topics:

  • CMS CGT Model
  • Expanded Access Model
  • Cost Recovery
12:00 – 1:00 PM Lunch
1:00 – 2:30 PM

Concurrent 3: Moving Beyond Busulfan Conditioning

Moderators: Sung-Yun Pai, MD - National Institutes of Health

Topics:

  • Melphalan Conditioning in Stem Cell Gene Therapy for Hemophilia A
  • Anti-c-Kit Antibody Conditioning in Fanconi Anemia
  • Multiplex Editing Including c-Kit Epitope Editing in Nonhuman Primate Model of Sickle Cell Disease

Concurrent 4: Accreditation and Onboarding: Is an 80-20 Model Possible in Gene Therapy?


Moderator: Sarah Nikiforow, MD - 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
2:30 – 3:00 PM Break
3:00 – 3:45 PM

Concurrent Session Recaps
3:45 – 4:30 PM Managing Unexpected and Adverse Outcomes Roundtable


Moderators: Hemalatha Rangarajan, MD - Nationwide Children’s Hospital; Rayne Rouce, MD - Baylor College of Medicine
4:30 – 4:45 PM Closing Remarks

Housing Information

Sponsorship

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