Symposium Proceedings

View Video* of the Conference

Concurrent Sessions

Day 1 Keynote and Plenary Session

Day 2 Plenary Sessions

*where presenter permission was granted

View Pictures of the Conference

Photographs by Ed Cunicelli

Thursday, May 6, 2021

Agenda with Presentations

Time
Session
8:00 am EDT

Log-in to Virtual Conference - Visit Virtual Exhibit Hall and Posters

9:00 am EDT

Concurrent Sessions (Pre-Recorded) Begin and will be available Thursday May 6 and Friday May 7

There are a total of 8 tracks. Scroll left and right to view all tracks.

Track 1 Nursing
CNE-Accredited

Track 2 Pediatric Care

Track 3 Pre-Clinical / Manufacturing

Track 4 Next-Gen Clinical Research

Track 5 Regulatory and Commercial Development

Track 6 Solid Tumor CARs

Track 7 Financial Implications

Track 8 From Local to Global: Development of Engineered Cell & Gene Therapies

9:00 am EDT

Track 1 Nursing
CNE-Accredited
9:00 am EDT

Introductions by:

Madeline Bell
Children’s Hospital of Philadelphia
Kevin B. Mahoney, DBA | Penn Medicine

Joint Adult and Pediatric Sessions - Nursing and Patient Care

State of the Union: Nursing in the Era of Cell and Gene Therapies

Regina Cunningham, PhD, RN, NEA-BC, FAAN | Penn Medicine
Ellen Tracy, RN, MSN, NEA-BC | Children’s Hospital of Philadelphia
 

Keynote Session: The Use of CRISPR Technology in the Treatment of Malignant and Non-Malignant Diseases

Making CAR T Cells "CRISPeR"

Edward A. Stadtmauer, MD | Penn Medicine

Adult Q&A Moderator: Patricia Mangan, MSN, CRNP | Penn Medicine

Nursing Considerations / Implications for Patients with SCD in the Era of Gene Tx

Claire M. White, MSN, RN | Children’s Hospital of Philadelphia

Elizabeth A. Worster, MSN, CRNP | Children’s Hospital of Philadelphia (Pediatric Q&A Moderator)
PRESENTATION

Pediatric Nursing Session

Moderator: Laura Smith, RN, MSN, CRNP | Children’s Hospital of Philadelphia  

Immunotherapy in Pediatrics: AML/CART 33: Nursing considerations during COVID pandemic

Diane Baniewicz, RN, MSN, CRNP | Children’s Hospital of Philadelphia
PRESENTATION

Immunotherapy in Pediatrics: Nursing Considerations for U CART in Pediatrics

Colleen Callahan, RN, MSN, CRNP | Children’s Hospital of Philadelphia
PRESENTATION

Adult Nursing Session

Moderator: Patricia Mangan, MSN, CRNP | Penn Medicine

Preparing for an Autologous Stem Cell Transplant

Kelly Salam, RN, BSN | Penn Medicine
Keri McDevitt, RN, MSN, OCN | Penn Medicine
PRESENTATION

Preparing for an Allogenic Transplant

Rachel Golding, RN, BSN | Penn Medicine
Lila Quinn, BSN, RN, OCN | Penn Medicine
PRESENTATION

COVID Management of Liquid Oncology Patients

Abbey Walsh, MSN, RN, OCN | Penn Medicine
Angela M. Rubin, RN, BSN, OCN | Penn Medicine
PRESENTATION

Updates on Immunotherapies in Myeloma

Patricia Mangan, MSN, CRNP | Penn Medicine
PRESENTATION

Late Effects and Survivorship Care Following Donor Stem Cell Transplant: What to expect after the 1st year?

Linda M. Perry, MS, PA-C | Penn Medicine
PRESENTATION

9:00 am EDT

Track 2 Pediatric Care 9:00 am EDT

Introductions by:

Stephan A. Grupp, MD, PhD | Children’s Hospital of Philadelphia

Future of Gene Therapy in Pediatrics Session

Moderator: Timothy S. Olson, MD, PhD | Children’s Hospital of Philadelphia

Clinical Overview/Impact

Timothy S. Olson, MD, PhD | Children’s Hospital of Philadelphia
PRESENTATION

Gene Therapy Techniques

Matthew Porteus, MD, PhD | Stanford University
PRESENTATION

Implication of Gene Therapy Treatment in Pediatrics Session

Moderator: Timothy S. Olson, MD, PhD | Children’s Hospital of Philadelphia

Hemoglobinopathies

Janet L. Kwiatkowski, MD, MSCE | Children’s Hospital of Philadelphia

Immune Deficiencies

Sung-Yun Pai, MD | National Institute of Health
(Consent to Post Not Granted)

Leukodystrophies

Amy T. Waldman, MD, MSCE | Children’s Hospital of Philadelphia
PRESENTATION

9:00 am EDT

Track 3 Pre-Clinical / Manufacturing 9:00 am EDT

Introductions by: 

Bruce L. Levine, PhD | Penn Medicine

Target Discovery and Immunogenicity Session

Moderator: Robert H. Vonderheide, MD, DPhil | Abramson Cancer Center

Antibody Discovery for New CAR Constructs

Donald L. Siegel, MD, PhD | University of Pennsylvania
PRESENTATION

Immune Responsiveness or Immune Ignorance?

Lisa Butterfield, PhD | Parker Institute for Cancer Immunotherapy
PRESENTATION

Immune Health

Robert H. Vonderheide, MD, DPhil | Abramson Cancer Center
PRESENTATION

Viral Vector Free Delivery Session

Moderator: Bruce L. Levine, PhD | Penn Medicine

Overview/Survey of Tech

Bruce L. Levine, PhD | Penn Medicine
PRESENTATION

Electro-Mechanical Transfection

Cullen R. Buie, PhD | Massachusetts Institute of Technology
(Consent to Post Not Granted)

Transient Cell Volume Exchange

Todd Sulchek, PhD | Georgia Institute of Technology
PRESENTATION

Enabling the Next Wave of Non-Viral Cell Therapies

Michael Maguire, PhD, CEO | Avectas
PRESENTATION

Gene Delivery and Editing for Better CARs Session

Moderator: Fyodor D. Urnov, PhD | University of California at Berkley

CRISPR and Beyond

Alexander Marson, MD, PhD | Gladstone-UCSF Institute of Genomic Immunology

PiggyBac and High-Fidelity Gene Editing for Fully Allogeneic CAR-T

Eric Ostertag, MD, PhD | Poseida
(Consent to Post Not Granted)

Sleeping Beauty in Trials

Laurence J.N. Cooper, MD, PhD | Ziopharm Oncology

9:00 am EDT

Track 4 Next-Gen Clinical Research 9:00 am EDT

Introductions by:

David L. Porter, MD | Penn Medicine

Newest Model CARs Session

Moderator: Joseph A. Fraietta, PhD | University of Pennsylvania

Engineering Remote-Controllable CAR T Cells for Cancer Immunotherapy

Peter Yingxiao Wang, PhD | University of California San Diego
PRESENTATION

T Cell Antigen Couplers

Jonathan Bramson, PhD | McMaster University, Canada
(Consent to Post Not Granted)

in vivo CAR's

Christian Buchholz, PhD | Paul-Ehrlich-Institut, Germany
(Consent to Post Not Granted)

CAR's on the Unpaved Road Session

Moderator: James L. Riley, PhD | University of Pennsylvania

CAR's in Fibrosis

Jonathan A. Epstein, MD | University of Pennsylvania
PRESENTATION

CAARs for Antibody-mediated Autoimmune Disease

Michael C. Milone, MD, PhD | University of Pennsylvania
(Consent to Post Not Granted)

CAR's in HIV

James L. Riley, PhD | University of Pennsylvania
PRESENTATION

Allogeneic and Donor Derived CAR T Cells, NKs, iPSC's Session

Moderator: Melody Smith, MD, MS | Memorial Sloan Kettering Cancer Center

Allo CARs Intro

Melody Smith, MD, MS | Memorial Sloan Kettering Cancer Center
(Consent to Post Not Granted)

Making Allogeneic CAR T Therapy a Reality for Patients: A pathway to controlling rejection

David Chang MD, PhD | Allogene Therapeutics
(Consent to Post Not Granted)

FT536 – An iPSC-Derived, Off-the-Shelf Multiplexed Engineered CAR MICA/B NK Cell Product Targeting Solid Tumors

Bob Valamehr, PhD, MBA | Fate Therapeutics
(Consent to Post Not Granted)

Donor-Derived Gene-Edited Allogeneic CAR-T 

Samarth Kulkarni, PhD | CRISPR Therapeutics
(Consent to Post Not Granted)

CAR-T Vaccine Booster Session

Moderator: Michael C. Milone, MD, PhD | University of Pennsylvania

RNA Vaccine Boosting CAR T Cells

Katharina Reinhard, PhD | Immunoreceptor Therapy
(Consent to Post Not Granted)

CD19 CAR T “Booster” T-APCs

Colleen Elizabeth Annesley, MD | Seattle Children’s Hospital
PRESENTATION

CMV Vaccine

Stephen J. Forman, MD | City of Hope

9:00 am EDT

Track 5 Regulatory and Commercial Development 9:00 am EDT

Introductions by: 

Bruce L. Levine, PhD | Penn Medicine

Managing Toxicities Session

Moderator: Noelle V. Frey, MD, MSCE | Penn Medicine

Consistency in Grading and Reporting

Stephan A. Grupp, MD, PhD | Children’s Hospital of Philadelphia
PRESENTATION

Pathophysiology of Neurotoxicity

Saad J. Kenderian, MB, ChB | Mayo Clinic

CAR T-Cells: Subacute and late toxicities beyond CRS

Nirali N. Shah, MD, MHSc | National Institute of Health
PRESENTATION

Gene Therapy INDs and Regulatory Considerations for Accelerated Development Session

Moderator: Anne Chew, PhD | Penn Medicine

Multiple Versions of Gene Therapy Products

Wilson W. Bryan, MD | U.S. Food and Drug Administration
PRESENTATION 

Expediting Clinical Development for Cell and Gene Therapies - Applying Master Protocol Concept in Early-Phase Trials

Ke Liu, MD, PhD | Sana Biotechnology
PRESENTATION

Regulatory Considerations to Accelerate Gene Therapy Development

Jiwen Zhang, PhD | Passage Bio
PRESENTATION 

Parent-Child Development Strategy:  A novel clinical and regulatory framework to evaluate multiple generations of NY-ESO1 targeted cell therapies in rapid sequence

Aiman Shalabi, PharmD, MBA | GlaxoSmithKline
(Consent to Post Not Granted)

Updates on Approved CAR T Cell Therapies Session

Moderator: David L. Porter, MD | Penn Medicine

Mechanisms of Treatment Resistance and Toxicity Related Markers in Context of Axicabtagene Ciloleucel for Lymphoma

Adrian Bot, MD, PhD | Kite
PRESENTATION

International Supply Chain Expansion and Approvals During COVID

Amir Hefni, PhD, MBA | Novartis
PRESENTATION

Lisocabtagene Maraleucel Combination Strategies in Lymphoma

Joseph Grosso, PhD | Bristol Myers Squibb

9:00 am EDT

Track 6 Solid Tumor CARs 9:00 am EDT

Introductions by:

David L. Porter, MD | Penn Medicine

Solid Tumor CARs – ONE Session

Moderator: Partow Kebriaei, MD | MD Anderson

Driving CARs in Solid Tumors: The promise and challenges

Partow Kebriaei, MD | The University of Texas MD Anderson Cancer Center
PRESENTATION

GBM Trials

Donald O’Rourke, MD | Penn Medicine
PRESENTATION

Prostate Cancer Trials

Naomi B. Haas, MD | Penn Medicine
PRESENTATION

Solid Tumor CARs – “TOO” Session

Moderator: Mark H. O'Hara, MD | Penn Medicine

Muc1 Trials

Mark H O'Hara, MD | Penn Medicine
PRESENTATION

MESO/Pancreatic

Janos L. Tanyi, MD, PhD | Penn Medicine
PRESENTATION

What’s “Neo” in Solid Tumors Session

Moderator: Gerald P. Linette, MD, PhD | Penn Medicine

Chromosomal Instability and Immune Evasion in Cancer Evolution

Charles Swanton, FRCP PhD FMedSci, FAACR, FRS | Cancer Research UK
(Consent to Post Not Granted)

Immunotherapy for Clonal Driver Neoantigens

Gerald P. Linette, MD, PhD | Penn Medicine
(Consent to Post Not Granted)

Toward Commercializing Tumor Infiltrating Lymphocyte Cell Therapy for Treatment of Solid Tumors

Maria Fardis, PhD, MBA | Iovance Biotherapeutics
PRESENTATION

 
9:00 am EDT

Track 7 Financial Implications 9:00 am EDT

Introduction by:

David L. Porter, MD | Penn Medicine

Financial Implications Session

Moderator: Kevin B. Mahoney, DBA | Penn Medicine

Pricing Tolerance and the Evolution of Cell Therapy Costs of Care

Richard T. Maziarz, MD | Oregon Health & Science University
PRESENTATION

How do We Pay for the Cell and Gene Therapy Revolution?

Kevin B. Mahoney, DBA | Penn Medicine
PRESENTATION

Why do We Need to Keep Costs Down for Cell Therapies? A few ideas for how to do it

Ezekiel J. Emanuel, MD, PhD | University of Pennsylvania
PRESENTATION

9:00 am EDT

Track 8 From Local to Global: Development of Engineered Cell & Gene Therapies 9:00 am EDT

Introductions by:

Stephan A. Grupp, MD, PhD | Children’s Hospital of Philadelphia

Lessons Learned for Stimulating CAR-T Research and Commercialization:  USA, UK, Canada and Australia Session

Moderator:  Bruce L. Levine, PhD | Penn Medicine

USA: A View from Cellicon Valley

Bruce L. Levine, PhD | US: Cellicon Valley

Australia Cell and Gene Therapy Research and Commercialization: Lessons learned   

Simon Harrison, MBBS, MRCP (UK), PhD, FRCPath (UK), FRACP | Australia: Peter MacCallum Cancer Centre
PRESENTATION

Tackling Challenges: A look at lessons learned from the UK

Matthew Durdy, MA, MBA | Cell and Gene Therapy Catapult
PRESENTATION

Canada’s Experience in Commercializing Regenerative, Cell and Gene Therapies

Michael May, PhD | Centre for Commercialization of Regenerative Medicine
PRESENTATION

Stimulating Cross-Border Collaborative Research in CGT Session

Moderator:  Anthony Ting, PhD | Bone Therapeutics

Cross-Border Partnerships to Accelerate Cell Manufacturing Technologies: Creating a globally-experienced workforce and developing unique supply chain solutions

Krishnendu Roy, PhD | Georgia Institute of Technology

Exploiting the CAR Technology to Quell Inflammatory Tissue Damage

Alberto Sanchez Fueyo, MD | Kings College London

Perspectives from ISCT

Anthony Ting, PhD | Bone Therapeutics
PRESENTATION

Dissemination of CAR T Clinical Program Expertise Session

Moderator: Robert E. Richards, MSBI, MBA| Penn Medicine

CARs Cruising Down the Highway: From academic to community medicine

Robert E. Richards, MSBI, MBA | Penn Medicine
PRESENTATION

Sarah Cannon’s Approach to Offering CAR T Therapy in the Community

Rocky Billups, MS | Sarah Cannon
PRESENTATION

Dissemination of CAR T Trials-Platforms and Partnerships

Rimas Orentas, PhD | Seattle Children’s Hospital
PRESENTATION

1:00 pm EDT

Welcome

Amy Gutmann, PhD | University of Pennsylvania

1:15 pm EDT

Keynote Address

Engineering Smarter T Cells

Stanley R. Riddell, MD | Fred Hutchinson Cancer Research Center
PRESENTATION

2:15 pm EDT

Cell and Gene Startups and Innovation in Cellicon Valley

Moderator: John S. Swartley, MBA, PhD | University of Pennsylvania

Aimee S. Payne, MD, PhD | Founder, Cabaletta Bio

Debora Barton, MD | CMO, Carisma Therapeutics

Usman “Oz” Azam, MD | CEO, Tmunity Therapeutics

Jeffrey D, Marrazzo, MBA, MPA | CEO, Spark Therapeutics

Audrey Greenberg, MBA, CPA | Co-Founder and Executive Managing Director, The Discovery Labs

3:00 pm EDT

Live Q&A For Concurrent Sessions
Featuring Presenters and Moderators from Pre-recorded concurrent sessions

There are a total of 8 tracks. Scroll left and right to view all tracks.

Track 1 Nursing
CNE-Accredited

Track 2 Pediatric Care

Track 3 Pre-Clinical / Manufacturing

Track 4 Next-Gen Clinical Research

Track 5 Regulatory and Commercial Development

Track 6 Solid Tumor CARs

Track 7 Financial Implications

Track 8 From Local to Global: Development of Engineered Cell & Gene Therapies

3:00 pm EDT

Track 1 Nursing
CNE-Accredited
3:00 pm EDT

Q&A
Joint Adult and Pediatric Sessions - Nursing and Patient Care

The Use of CRISPR Technology in the Treatment of Malignant and Non-Malignant Diseases:

Making CAR T Cells “CRISPeR”

3:15 pm EDT

Track 1 Nursing
CNE-Accredited
3:15 pm EDT

Q&A
Joint Adult and Pediatric Sessions - Nursing and Patient Care

The Use of CRISPR Technology in the Treatment of Malignant and Non-Malignant Diseases:

Nursing Considerations / Implications for Patients with SCD in the Era of Gene Tx with panel

3:30 pm EDT

Track 1 Nursing
CNE-Accredited
3:30 pm EDT

Q&A
Pediatric Nursing Session

Immunotherapy in Pediatrics

AML/CART 33: Nursing Considerations during COVID Pandemic

Nursing Considerations for U CART in Pediatrics

Q&A
Adult Nursing Session

Preparing for an Autologous Stem Cell Transplant
Preparing for an Allogenic Transplant
COVID Management of Liquid Oncology Patients
Updates on Immunotherapies in Myeloma
Late Effects and Survivorship Care Following Donor Stem Cell Transplant: What to Expect After the 1st Year?
3:00 pm EDT

Track 2 Pediatric Care 3:00 pm EDT

Q&A
Future of Gene Therapy in Pediatrics Session

Clinical Overview/Impact
Gene Therapy Techniques
3:15 pm EDT

Track 2 Pediatric Care 3:15 pm EDT

Q&A
Implication of Gene Therapy Treatment in Pediatrics Session

Hemoglobinopathies
Immune deficiencies
Leukodystrophies
3:00 pm EDT

Track 3 Pre-Clinical / Manufacturing 3:00 pm EDT

Q&A
Target Discovery and Immunogenicity Session

Antibody Discovery for New CAR Constructs
Immune Responsiveness or Immune Ignorance?
Immune Health
3:15 pm EDT

Track 3 Pre-Clinical / Manufacturing 3:15 pm EDT

Q&A
Viral Vector Free Delivery Session

Overview/Survey of Tech
Electro-mechanical Transfection
Transient Cell Volume Exchange
Enabling the Next Wave of Non-Viral Cell Therapies
3:30 pm EDT

Track 3 Pre-Clinical / Manufacturing 3:30 pm EDT

Q&A
Gene Delivery and Editing for Better CARs Session

CRISPR and Beyond
PiggyBac and High-Fidelity Gene Editing for Fully Allogeneic CAR-T
Sleeping Beauty in Trials
3:00 pm EDT

Track 4 Next-Gen Clinical Research 3:00 pm EDT

Q&A
Newest Model CARs Session

Engineering Remote-Controllable CAR T Cells for Cancer Immunotherapy
T Cell Antigen Couplers
in vivo CAR's
3:15 pm EDT

Track 4 Next-Gen Clinical Research 3:15 pm EDT

Q&A
CAR's on the Unpaved Road Session

CAR's in Fibrosis
CAAR's PV by Cabaletta
CAR's in HIV
3:30 pm EDT

Track 4 Next-Gen Clinical Research 3:30 pm EDT

Q&A
Allogeneic and Donor Derived CAR T Cells, NKs, iPSC's Session

Allo CARs Intro
Making Allogeneic CAR T Therapy a Reality for Patients: A pathway to controlling rejection
FT536 – An iPSC-Derived, Off-the-Shelf Multiplexed Engineered CAR MICA/B NK Cell Product Targeting Solid Tumors
Donor-Derived Gene-Edited Allogeneic CAR-T
3:45 pm EDT

Track 4 Next-Gen Clinical Research 3:45 pm EDT

Q&A
CAR-T Vaccine Booster Session

RNA Vaccine Boosting CAR T Cells
CD19 CAR T “Booster” T-APCs
CMV Vaccine
3:00 pm EDT

Track 5 Regulatory and Commercial Development 3:00 pm EDT

Q&A
Managing Toxicities Session

Consistency in Grading and Reporting
Pathophysiology of Neurotoxicity
CAR T-Cells: Subacute and late toxicities beyond CRS
3:15 pm EDT

Track 5 Regulatory and Commercial Development 3:15 pm EDT

Q&A
Gene Therapy INDs and Regulatory Considerations for Accelerated Development Session

Multiple Versions of Gene Therapy Products
Expediting Clinical Development for Cell and Gene Therapies - Applying Master Protocol Concept in Early-Phase Trials
Regulatory Considerations to Accelerate Gene Therapy Development
Parent-Child Development Strategy:  A novel clinical and regulatory framework to evaluate multiple generations of NY-ESO1 targeted cell therapies in rapid sequence
3:30 pm EDT

Track 5 Regulatory and Commercial Development 3:30 pm EDT

Q&A
Updates on Approved CAR T Cell Therapies Session

Mechanisms of Treatment Resistance and Toxicity Related Markers in Context of Axicabtagene Ciloleucel for Lymphoma
International Supply Chain Expansion and Approvals During COVID
Define CD4 CDA Doses in CAR T Cell Therapy
3:00 pm EDT

Track 6 Solid Tumor CARs 3:00 pm EDT

Q&A
Solid Tumor CARs - ONE Session

Driving CARs in Solid Tumors: The promise and challenges
GBM Trials
Prostate Cancer Trials
3:15 pm EDT

Track 6 Solid Tumor CARs 3:15 pm EDT

Q&A
Solid Tumor CARs - "TOO" Session

Muc1 Trials
MESO/Pancreatic
 
3:30 pm EDT

Track 6 Solid Tumor CARs 3:30 pm EDT

Q&A
What’s “Neo” in Solid Tumors Session

Chromosomal Instability and Immune Evasion in Cancer Evolution
Immunotherapy for Clonal Driver Neoantigens
Toward Commercializing Tumor Infiltrating Lymphocyte Cell Therapy for Treatment of Solid Tumors
3:00 pm EDT

Track 7 Financial Implications 3:00 pm EDT

Q&A
Financial Implications Session

Pricing Tolerance and the Evolution of Cell Therapy Costs of Care
How do We Pay for the Cell and Gene Therapy Revolution?
Why do We Need to Keep Costs Down for Cell Therapies? A few ideas for how to do it

 

3:00 pm EDT

Track 8 From Local to Global: Development of Engineered Cell & Gene Therapies 3:00 pm EDT

Q&A
Lessons Learned for Stimulating CAR-T Research and Commercialization: USA, UK, Canadam and Australia Session

USA: A View from Cellicon Valley
Australia Cell and Gene Therapy Research and Commercialization: Lessons learned   
Tackling Challenges: A look at lessons learned from the UK
Canada’s Experience in Commercializing Regenerative, Cell and Gene Therapies
3:15 pm EDT

Track 8 From Local to Global: Development of Engineered Cell & Gene Therapies 3:15 pm EDT

Q&A
Stimulating Cross-Border Collaborative Research in CGT Session

Cross-Border Partnerships to Accelerate Cell Manufacturing Technologies: Creating a globally-experienced workforce and developing unique supply chain solutions
Exploiting the CAR Technology to Quell Inflammatory Tissue Damage
Perspectives from ISCT

 

3:30 pm EDT

Track 8 From Local to Global: Development of Engineered Cell & Gene Therapies 3:30 pm EDT

Q&A
Dissemination of CAR T Clinical Program Expertise

CARs Cruising Down the Highway: From academic to community medicine
Sarah Cannon’s Approach to Offering CAR T Therapy in the Community
Dissemination of CAR T Trials-Platforms and Partnerships