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; 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; 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

Call for Abstracts

The call for abstracts closed on Sunday, August 10. Thank you to everyone who submitted! Decisions will be sent later this month.

Call for Abstracts Timeline

  • Friday, July 18: Call for abstracts opens
  • Sunday, August 10: Call for abstracts closes
  • Mid-August: Decisions for abstracts sent

Abstract Submission Criteria

Abstracts will be scored based on the following criteria; however, our Organizing Committee is also open to submissions that focus on practical tips, education, implementation strategies, and quality projects in the field of gene therapy:

  • Importance and Relevance: Is a clear rationale for the work provided? Does the study address an important question? Do its findings have the potential to advance the field of gene therapy?
  • Originality and Innovation: Is the work novel? Does it present new findings and/or address a problem using an original or improved approach?
  • Quality of Methods and Writing: Are methods well described and valid to answer the question? Is the abstract clear and well written? Is the abstract within the 300-word limit?

Please note that the abstract submission form does not have a tracker for word count. We recommend that you prepare your abstract submission in a Word document to confirm that you stay within the 300-word limit.

All accepted abstracts will be required to attend and present their posters at the Summit during the reception on Wednesday, September 10.

Responsibility of Abstract Authors

The Presenting Author is responsible for the following:

  • Ensuring that all authors have read the abstract and agreed to be co-authors.
  • Notifying all co-authors of any additions, deletions, and changes to the program, as may be communicated by the 2025 ASTCT Gene Therapy Summit.
  • Obtaining all of the conflict-of-interest disclosure information from co-authors.
  • Making certain that abstracts are complete (tables included and labeled) and written in clear, concise and grammatically correct English, so that reviewers are able to focus solely on the scientific merits of the submission. For authors with any questions regarding the clarity, grammar or spelling of their abstract, we encourage identifying and using an independent reviewer to edit the abstract prior to submission.

Please contact Haedyn Smith (hsmith@astct.org) with any questions regarding your submission. 

When you begin your submission, you will be asked to "Name this Entry", please use this section to include the high-level topic/area/theme that your submission will address.

Housing Information

Sponsorship

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