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

Registration

Register

Registration will close on Tuesday, September 9 at 5 p.m. CDT.

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

Registration will close on Tuesday, September 9 at 5 p.m. CDT.

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 Cancer Institute 
8:05 AM – 9:35 AM

Concurrent 1: Mobilization and Apheresis for Gene Therapy: 201

Session Chair: Suzanne Thibodeaux, MD, PhD; Washington University

Topics:

  • An Operational Overview, Pain Points and Solutions in Mobilization
  • Technical Challenges in Managing Poor Mobilizers
  • 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 Schmal; 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

Sponsorship

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