Pre-Meeting Workshops

ASGCT's pre-meeting workshops run concurrently on May 15, the day before the 21st Annual Meeting begins. Attendees who register for any of the three meetings at the same time as the full annual meeting receive a $25 discount on total registration. Online registration for the pre-meeting workshops is included in the online registration form for the 21st Annual Meeting.

PDF registration forms are available for people who have already registered for the 21st Annual Meeting and would like to add a workshop to their existing registration:

Gene Editing (8:30 a.m. – 5 p.m.)

The Gene Editing Workshop has SOLD OUT. Please contact the ASGCT office to be added to the Gene Editing Workshop waitlist.

Syllabus

Full Schedule

Commercialization (8:30 a.m. – 5 p.m.)

Registration Form

Syllabus

Full Schedule

CAR T Cell Therapies (1 – 5 p.m.)

Registration Form

Syllabus

Full Schedule

Gene Editing Workshop Program

Introduction: 8:30 – 9 a.m.

Speaker:

• J. Keith Joung, MD, PhD

Pre-Clinical Development: 9 – 10:30 a.m.

Chair: Matt Porteus, MD, PhD

Speakers:

• James Wilson, MD, PhD
• Kris Saha, PhD
• Paula Cannon, PhD
• Daniel Dever, PhD

Coffee Break: 10:30 – 11 a.m.

Clinical Gene Editing Programs: 11 a.m. – 12 p.m.

Chair: Toni Cathomen, PhD

Speakers:

• Justin Eyquem, PhD
• Beverly L. Davidson, PhD
• Leonela Amoasii, PhD
• Toni Cathomen, PhD

Lunch: 12 – 1 p.m.

Non-Cononical Gene Editing: 1 – 2:30 p.m.

Chair: Charlie Gersbach, PhD

Speaker:

• Charlie Gersbach, PhD
• Omar Abudayyeh
• Nicole Gaudelli, PhD

Junior Investigator: 2:30 – 3 p.m.

Chair: Paula Cannon, PhD

Speakers:

• Vikram Pattanayak, MD, PhD
• Ayal Hendel, PhD

Coffee Break: 3 – 3:30 p.m.

Junior Investigator: 3:30 – 4 p.m.

Speakers:

• Suk See De Ravin, MD, PhD
• Alexis Komor, PhD

Corporate Review : 4 – 5 p.m.

Chair: J. Keith Joung, MD, PhD

Speakers:

• Mike Holmes, PhD
• Mark Behlke, MD, PhD
• Tirtha Chakraborty, PhD
• Tom Barnes, PhD
• Charlie Albright, PhD
• Julianne Smith, PhD

Commercialization Workshop Program:

Keynote Address: 8:30 – 9:30 a.m.

Speakers:

• John Furey, Spark

Value Frameworks for Gene and Cell Therapy: 9:30 – 10:30 a.m.

The panel will review recent value framework efforts and discuss ways to define value for therapies with uncertain durability and how recent assessments of gene and cell therapies have impacted payer decision making.

Chair: Michael Werner, JD, Alliance for Regenerative Medicine 

Speakers:

• Sarah K Emond, MPP, Institute for Clinical and Economic Review
• Phil Reilly, MD, JD, Third Rock
• Colleen Rye, PhD, FasterCures
• Marianne Hamilton Lopez, PhD, MPA, Duke-Robert J. Margolis, MD, Center for Health Policy

Coffee Break: 10:30 – 11 a.m.

Regulatory Advances: 11 a.m. – 12 p.m.

Chair: Anne-Virginie Eggimann, MS, bluebird bio

Speakers:

• Peter Marks, MD, PhD, FDA Center for Biologics Evaluation and Research
• Snehal Naik, PhD, Janssen Research & Development, LLC
• Robert Pietrusko, Pharm.D. Voyager Therapeutics Inc.
• Lynne McGrath, MPH, PhD, REGENXBIO

Lunch: 12 – 1 p.m.

Patient Voice: 1 – 2 p.m.

Gene and cell therapies offer transformative benefits for rare diseases, patient advocates will discuss their perceptions of these new therapies, how they could impact current treatment paradigms and any challenges they believe might impact patient access

Chair: Jennifer Bernstein, Horizon Government Affairs

Speakers:

• Stephen Rose, PhD, Foundation Fighting Blindness
• Laurice Levine, MA, CCLS, Children's Los Angeles, Thalassemia Support Foundation
• Melissa Hogan, JD Project Alive
• Jennifer Farmer, MS, CGC, Friedreich's Ataxia Research Alliance

Real World Market Access: 2 – 3 p.m.

Industry, payer and coalition leaders will discuss lessons learned from the real world launch of recent gene and cell therapies

Chair: Kristin Viswanathan Wolff, MPH, bluebird bio

Speakers:

• Sarah Pitluck, Spark Therapeutics
• Mark Trusheim, MIT NEWDIGS, FoCUS Project
• Rocio Manghani, Kite Pharma
• Bill Martin, Accredo Health

Coffee Break: 3 – 3:30 p.m.

CMC Considerations Panel: 3:30 – 4:15 p.m.

Development of a regenerative medicine product from promising discovery to viable clinical therapy requires significant investment in preparation and planning for CMC considerations. This panel of manufacturing experts from cell and gene therapy companies will discuss key CMC challenges and provide perspective on how these challenges are being addressed as companies are approaching commercialization.

Chair: Stewart E. Abbot, PhD, Fate Therapeutics

Speakers:

• Zhenhong Li, PhD, REGENXBIO
• Jerry Keybl, MilliporeSigma
• Bitao Liang, PhD, Celgene
• Peter Olagunju, bluebird bio
• Xin Swanson, PhD, MBA, Lonza

Industry Perspectives Panel: 4:15 – 5 p.m.

In recent years, the role of large cap Biotech and Pharma organizations in cell and gene therapy has grown to include investment, R&D and clinical partnerships, and licensing opportunities as well as mergers and acquisition. This panel of Biotech and Pharma executives will provide insight on the current state of the industry and how their organizations are strategically positioning themselves to support these new therapeutic modalities.

Chair: Morrie Ruffin, Alliance for Regenerative Medicine 

Speaker: 

• Jonanthon Garen, uniQure
• Barrie J. Carter, PhD, BioMarin Pharmaceutical
• Donna Armentano, PhD, Pfizer Inc.
• Amy DuRoss, MBA, Vineti

CAR T Cell Therapies Program:

The CAR T Cell Therapies Workshop, presented in collaboration with the Foundation for the Accreditation of Cellular Therapy (FACT) and the American Society for Blood and Marrow Transplantation (ASBMT), features discussions of the current state of CAR T therapeutic approaches in light of recent FDA approvals including the implementation of treatment protocols and challenges surrounding the implementation of commercial products.

Welcome and Introductory Remarks: 1 – 1:10 p.m.

• Helen Heslop, MD
• Krishna Komanduri, MD

Setting up an Immune Effector Program: 1:10 – 2:20 p.m.

In this session there will be talks describing different models for setting up immune effector programs either as stand-alone programs or as programs integrated with a hemopoietic stem  cell transplant program. The resources required for financial co-ordination, care co-ordination, clinical care and data management will be discussed in different models covering both adult and pediatric patients.

Chairs: Helen Heslop, MD and Sergio Giralt, MD

Speakers:

• Helen Heslop, MD
• Sergio Giralt, MD
• EJ Shpall, MD
• Samer Khaled, MD

Accreditation: 2:20 – 3 p.m.

In this session Drs. Nikiforow and Warkentin will describe the development of standards to promote quality practice in immune effector cell administration. They will also discuss the voluntary, peer-led FACT accreditation program.

Chair: Sarah Nikiforow, MD, PhD

Speakers:

• Phyllis Warkentin, MD
• Sarah Nikiforow, MD, PhD

Coffee Break: 3 – 3:30 p.m.

Implementing Commercial Products: 3:30 – 5 p.m.

In this session we will discuss the challenges of implementing commercial adoptive immunotherapy including practical considerations and our current understanding of the financial systems and potential barriers faced by patients and centers. 

Chair: Krishna Komanduri, MD

Speakers:

• Stephanie Farnia, MPH
• Krishna Komanduri, MD  
• Fred LeMaistre, MD
• Fred Locke, MD