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 November 5 – 6 at the Harvard Medical Conference Center in Boston, Massachusetts. The event will kick off the evening of the 5th with a networking reception with a presentation on cutting edge gene therapy science followed by the conference on November 6th.

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 $50
ASTCT Member Rate $250
AABB Member Rate $250
ASGCT Member Rate $250
Non-Member Rate $350
Industry Registration $850

 

Agenda

Day 1: Tuesday, November 5
Day 1: Tuesday, November 5
5:30 – 7:30 PM

Welcome Reception

Take a break from the reception to attend the 2024 keynote.
6:30 – 7:00 PM

Keynote Speaker: The Promise of Hematopoietic Gene Therapy

George Daley, MD, PhD; Dean of the Faculty of Medicine; Harvard University
Day 2: Wednesday, November 6
 
Day 2: Wednesday, November 6
9:00 – 9:10 AM Welcome and Summit Goals

Christy Duncan, MD; Dana-Farber Cancer Institute
9:10 – 10:10 AM Not-So-Secret Societies: Understanding Gene Therapy and the ASTCT, ASGCT and AABB
Moderator: Corey Cutler, MD, MPH; American Society of Transplantation and Cellular Therapy President; Dana-Farber Cancer Institute


Speakers:

  • Corey Cutler, MD, MPH; American Society of Transplantation and Cellular Therapy President; Dana-Farber Cancer Institute
  • Matthew Porteus, MD, PhD; American Society of Gene and Cellular Therapy Vice-President; Stanford University
  • Jose Cancelas, MD, PhD; Association for the Advancement of Blood & Biotherapies President-Elect, Dana-Farber Cancer Institute, Harvard Medical School
10:10 – 10:30 AM Break
10:30 – 12:00 PM

Concurrent 1: The ABCs of Transfusion Medicine in Gene Therapy: The Critical Role of Apheresis, Blood Banking and Cellular Therapy Laboratories

Moderator: Suzanne Thibodeaux, MD, PhD; Washington University

Speakers:

  • John Manis, MD, PhD: Boston Children's Hospital
  • Stella Chou, MD; University of Pennsylvania
  • Jose Cancelas, MD, PhD; Dana-Farber Cancer Institute, Harvard Medical School

Topics:

  • Apheresis in Ex Vivo Hematopoietic Gene Therapy: Complex Patients and Processes
  • Transfusion Support of the Complex Sickle Cell Disease Patient
  • Cellular Therapy Laboratory Considerations for Hematopoietic Gene Therapies

Concurrent 2: Equity + Engagement = Access in Gene Therapy

Moderators: Jeffery Auletta, MD; Senior Vice President, Health Equity, NMDP; Chief Scientific Director, CIBMTR NMDP; David Barrett, JD; American Society of Gene and Cellular Therapy; Rayne Rouce, MD; Baylor College of Medicine

Speakers:

  • Amar Kelkar, MD; Dana-Farber Cancer Institute
  • Rayne Rouce, MD; Baylor College of Medicine
  • Sarah Rayel, MPP; PhRMA
  • Adrienne Shapiro, Axis Advocacy 

Topics:

  • Complexities of Access Related to Gene Therapy
  • Patient Engagement: How To Make It Happen
  • Practical Considerations for Emerging Access Models for Medicaid/Medicare Recipients: CMMI CGT Access Model
12:00 – 1:00 PM Lunch
1:00 – 2:30 PM

Concurrent 3: Long-Term Follow Up

Moderators: Sung-Yun Pai, MD; National Institute of Health; Hemalatha Rangarajan, MD; Nationwide Children’s Hospital

Speakers:

  • Sonali Chaudhury, MD; Ann & Robert H. Lurie Children's Hospital of Chicago
  • Patricia Steinert, PhD, MBA; Executive Scientific Director, Policy and Governance, CIBMTR; Associate Professor, Department of Medicine, Medical College of Wisconsin
  • Kevin Flanigan, MD; Nationwide Children's Hospital

Topics:

  • Strategies for Long-Term Follow-Up: Registry Interaction With Industry
  • Data Repository Experience
  • Sample Sharing and Shared Research
  • Adverse Event Monitoring

Concurrent 4: Transitioning From Investigational to Commercial Product in Ex Vivo Gene Therapy


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

Speakers:

  • Colleen Dansereau, MSN, RN, CPN; Boston Children's Hospital 
  • Keren Shani, MPH; Executive Director, Cell & Gene Therapy Center of Excellence, Trinity Life Sciences
  • Lisa Eisenbud, Senior Director, Patient Financial Services; Boston Children's Hospital 

Topics:

  • Becoming a Qualified Treatment Center: The Center Perspective
  • The Transition From Study to Commercial Therapy: The Industry Perspective 
  • Securing Financial Approval for Commercial Gene Therapy
2:30 – 2:45 PM Break
2:45 – 3:45 PM

Industry/Academic Interactive Roundtable: Critical Issues in Commercial Gene Therapy: Manufacturing, Handling Post-Marketing Insertional Oncogenesis, Access and Future Directions

Moderator: Akshay Sharma, MD; St. Jude Children’s Research Hospital

Speakers: 

  • Julie Kanter, MD; Professor, University of Alabama Birmingham; President, National Alliance of Sickle Cell Centers
  • Esther Shamir, MPH; President, Shamir Consulting
  • Matthew Porteus, MD, PhD; American Society of Gene and Cellular Therapy Vice-President; Stanford University
  • Melissa Bonner, PhD; Chief Scientific Officer, Nvelop Therapeutics
3:45 – 4:30 PM Concurrent Session Reports

Housing Information

Sponsorship

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