Full Program

PRESIDENTIAL PLENARY

Pioneering Gene Editing and Gene Modification for Patient Benefit: Trailblazing New Paths in Designing Immunity

Chair: Bruce Levine, PhD, University of Pennsylvania, United States
ESP Co-Chair: Nisha Durand, PhD, Mayo Clinic Florida, United States
Speakers:

• • Fyodor Urnov, PhD, University of California Berkeley, United States
  • Kristen Hege, MD, Bristol-Myers Squibb, United States
  • Carl June, MD, University of Pennsylvania, United States

When there was no beaten path, these pioneers created the path from discovery and technology development, to clinical trial translation, regulatory approval, and commercialization for gene modified immune cell therapies.  Now, we look at what will be needed to take our ability to design immunity from generation 1.0 to 2.0.

• Address the advantages, disadvantages, and unknowns of various gene editing approaches
• Bring new technologies for gene modification and immune cell engineering to the clinic
• Move from concept to scalable product that can achieve regulatory approval and benefit patients

MSC Plasticity and Effect of In-Vivo Environments on MSC Actions: What actually happens to the MSCs after administration?

Chair: Yufang Shi, PhD, Soochow University, China
Speakers: 

• • Tracey Heng, PhD, Monash University, Australia
  • Sara Enes, PhD, Lund University, Sweden

Prospects and Challenges for iPSC-based Cell Therapy

Chair: Shin Kawamata, MD, PhD, Foundation for Biomedical Research and Innovation, Japan
Speakers:

• • Guojun Bu, PhD, Mayo Clinic, United States
  • Shin Kaneko, MD, PhD, Kyoto University, Japan
  • Maxime Feyeux, PhD, TreeFrog Therapeutics, France

CORPORATE SESSION
MIRUS BIO, A Gamma Biosciences Company

Innovative and Scalable Transient Transfection Processes for GMP Manufacture of AAV and LV

Speaker: Leisha Kopp, Field Applications Scientist, Mirus Bio, a Gamma Biosciences Company, United States

Recombinant adeno-associated virus (AAV) and lentivirus (LV) are essential components of many gene and cell therapies designed to treat a vast array of acquired and heritable diseases. Thus, the need for large-scale manufacture of safe and effective viral vectors for the development of biotherapeutics has never been greater. In this talk, we will detail efficient, scalable and cost-effective systems for AAV and LV generation via transient transfection of HEK 293 cells. We will discuss how the TransIT-VirusGEN® Transfection Reagent, an innovative blend of lipids and polymers, facilitates significantly higher titers than PEI-derived polymers or liposomes alone. We will also highlight how TransIT-VirusGEN® GMP Reagent and VirusGEN® GMP AAV and LV Enhancers support gene and cell therapy researchers from R&D through commercial manufacturing. Key learning objectives include:

• Optimization and scaling considerations for AAV and LV generation in small- and large-volume platforms
• The utility of LV and AAV enhancers to further increase physical and functional virus titers over previously optimized conditions
• How VirusGEN® GMP Reagent and Kits offer workflow flexibility, cost savings, and expand manufacturing capabilities for gene and cell therapies

Non-Malignant Applications of CAR T

Chair: Ken Mickelthwaite, MBBS, PhD, FRACP, FRCPA, The University of Sydney, Westmead Hospital, Australia
Speakers:

• • Qizhi Tang, PhD, University of California, San Francisco, United States
  • Boro Dropulic, PhD, MBA, Caring Cross, United States
  • Michael Milone, MD, PhD, University of Pennsylvania, United States

State of the Art in EV (Exosome) Research: Rigor and Function

Chair: Bernd Giebel, PhD, University of Duisburg-Essen, Germany
Speaker:

• • Joshua Welsh, PhD, National Institutes of Health, United States

In-Vivo Gene Editing

Chair: Paula Rio, PhD, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Spain 
Speakers:

• • Sean Burns, MD, Intellia Therapeutics, United States
  • Nikoletta Psatha, PhD, Vlaams Instituut voor Biotechnologie, Belgium
  • John Sheridan, PhD, Cystic Fibrosis Foundation, United States

GLOBAL SHOWCASE PRESENTATIONS

SONY Theater 1
Terumo Blood and Cell Technologies (09:45 - 10:00)
Connecting the Future of Apheresis: Part 1  - Data is king
Speaker: Sara Lewandowski, MS, Global Data Analytics Engineer | Veda Solutions, Terumo Blood and Cell Technologies, United States Residence, Global Coverage

Terumo Blood and Cell Technologies (10:00 - 10:15)
Connecting the Future of Apheresis: Part 2  - Data is centralized
Speaker: Amar P S Chahal, MD, MBA, CEO and Founder | Stafa Cellular Therapy, Terumo Blood and Cell Technologies
United States Residence, Global Coverage

SONY Theater 2
WellSky (09:45 - 10:00)
Elevate innovative therapies with WellSky’s purpose-built software solution.
Speaker: Jonathan Wofford, Senior Director of Solution Architecture, WellSky, United States

Celonic (10:00 - 10:15)
Cutting edge cell and gene therapy manufacturing in Switzerland
Speaker: Theodoros Zografou, Business Development Director, Celonic AG, Switzerland

SCIENCE AND INNOVATION PLENARY

Today's Science Fiction/Tomorrow's Science
Promising Innovations in CGT Therapeutic Developments

Chair: Daniel J. Weiss, MD, PhD, University of Vermont, United States
Speakers:

• • Edward Botchwey, PhD, Georgia Institute of Technology, United States
  • Maroun Khoury, PhD, IMPACT Center, Chile
  • Shin Kawamata, MD, PHD, FBRI at Kobe, Japan

This session will discuss the application of computational modeling and other engineering approaches to cell and gene therapies and to tissue engineering.  Key goals and objectives are to:

• Understand advances in utilizing in silico and computational modeling to cell and gene therapies 
• Illustrate advances in tissue engineering approaches to CGT
• Understand immune challenges to CGT tissue engineering

CORPORATE SESSION
TERUMO

Where Cells Thrive – Scalability and Flexibility in Bioreactor Technology

Chair: Kathie Schneider, Director Cell Therapy Technologies, Terumo Blood and Cell Technologies, USA

Speakers:

• Amanda Conerty, Sr. Director of CMC, Artiva Biotherapeutics, USA
• Katy Spink, PhD, COO & Managing Partner, Darkhorse Consulting, USA
• Augustus Kilgore, PhD, Global Product Manager, Terumo Blood and Cell Technologies, USA

When advancing from process development to clinical phase to commercial, you risk automating too late or with a platform that does not meet later phase needs. Having multiple therapy products at different stages of development can add to this risk, especially if platforms are not flexible enough to handle a range of cell culture needs. Join us as we discuss how automating with a single platform technology beginning at process development can de-risk and accelerate your commercialization; what decisions are critical to carry your therapy into late-stage success; and what aspects to focus on, from core technology to software and more, as you decide the future of your cell expansion.

CORPORATE SESSION
THERMO FISHER SCIENTIFIC

Strategic Partnering to Enable Cell Therapy Commercialization

Moderator: Betty Woo, PhD, Vice President and General Manager, Cell Therapy, Thermo Fisher Scientific

Speakers:

• • Rupa Pike, PhD, Director of Enterprise Science and Innovation Partnerships, Thermo Fisher Scientific
  • Xavier De Mollerat Du Jeu, PhD, Senior Director of Research and Development, Thermo Fisher Scientific
  • David Shaw, PhD, Principal Scientist , Head of Cell Therapy Process Development, Genentech
  • Max Sidorov, Senior PD Engineer, Caribou Biosciences

As emerging cell therapies move from early discovery to commercialization, focus has pivoted to establishing scalable and reproducible manufacturing processes and innovations. Our panel of industry professionals come together to discuss how strategic partnering between biotech and pharma can help facilitate the challenging transition of moving therapies through the commercialization pipeline.

Objectives of the Symposium:

• Innovation in “fit for purpose” closed cell therapy manufacturing
• Process optimization through collaboration
• Bridging the gap between early discovery and late-phase development

TRANSLATION TO CLINIC PLENARY

Pushing the Envelope, Managing the Risk

Chair: Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Baylor College of Medicine, United States
Speakers:

• • Kenneth Micklethwaite, MBBS, PhD, The University of Sydney, Westmead Hospital, Australia
  • Nina Kotsopoulou, PhD, Orchard Therapeutics, United States
  • Rodney Rietze, PhD, iVexSol, United States

The transition from pre-clinical to clinical studies is a well-trodden, yet unique journey that is impacted by your drug product, the facility/resources you have available, and the complexity of your manufacturing process. 

• What are the risks of new gene modification systems and gene therapy products and how can we ameliorate those risks?
• How to develop potency assays for an early phase product to ensure a smooth transition not only into later phase studies but also into the market
• Navigating Technology Transfer to ensure feasibility of successful products and avoiding costly delays due to supply chain, raw materials, and the like. etc.

GLOBAL SHOWCASE PRESENTATIONS

SONY Theater 1
GCON (14:30 - 14:45)
Innovative Facility Options in an Era of Urgent Capacity Needs
Speaker: Peter Makowenskyj, Director of Design Consulting, G-CON Manufacturing, United States

CELLARES (14:45 - 15:00)
Manufacturing across geographies- balancing standardization and flexibility during a product’s development lifecycle
Speaker: John Tomtishen, VP of Operations, Cellares, United States

SONY Theater 2
CORNING (14:30 - 14:45)
Scaling Stem Cell and AAV Production to Manufacturing Scales with a Novel Intensified Adherent Cell Culture System
Speaker: Dr. Austin Mogen, Corning Incorporated, Life Sciences Division, United States

CORNING (14:45 - 15:00)
Improve cell processing and advance cell therapy with the Corning® X-SERIES® Cell Separation Platform
Speaker: Tom Bongiorno, PhD, Field Application Scientist, Corning Incorporated, United States

Enough Bait Cutting, Time to Fish- Rethinking first in Human MSC Studies

Chair: Jacques Galipeau, MD, University of Wisconsin-Madison, United States
Speakers: 

• • Dominique Farge, MD, PhD, Paris University and McGill, France
  • Karin Tarte, PhD, Rennes University, France
  • Fermín Sánchez‐Guijo, MD, PhD, IBSAL-Hospital Universitario De Salamanca, Spain

Objectives:

• Explore early phase clinical trial design and outcome to maximize clinical and mechanistic insights for future use
• Deep dive omics of MSC as informative companion for deciphering MOA and predictive biomarkers
• Discuss value of large case series to gain insights on value of MSCs as cell pharmaceutical for autoimmune disease

Weaponized Immune Cells

Chair: Chrystal Louis, MD, CRISPR, United States
Speakers:

• • Catherine Bollard, MD, MBChB, Children's National Research Institute, United States
  • Kevin Tosh, PhD, Carisma Therapeutics Inc., United States
  • Carlos Ramos, MD, Baylor College of Medicine, United States

Objectives:

• Address expanding therapy for cancer into new cell types
• Understand how to safely incorporate off the shelf CT products for early clinical investigations

Ethical Considerations and Consent in Novel Therapies

Chair: Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Baylor College of Medicine, United States
Speakers:

• • Akshay Sharma, MBBS, St. Jude Children's Research Hospital, United States
  • Patricia Zettler, JD, The Ohio State University Moritz College of Law, United States

Objectives:

• Describe the social, legal, and ethical challenges related to consent for clinical trials of novel high risk high reward interventions.
• Describe limitations to enrollment of vulnerable populations in clinical trials of such interventions.
• Describe ethical and regulatory issues associated with the expanded access and “right to try” pathways for non-trial preapproval access to such interventions
• Discuss methods to promote diversity, equity, and justice in relation to access to investigational interventions.

GLOBAL SHOWCASE PRESENTATIONS

SONY Theater 1
MILTENYI (16:00 - 16:15)
CliniMACS Prodigy® Platform – Master the Complexity in Cell Manufacturing
Speaker: Jessica Oenarto, PhD, Miltenyi Biotec, Germany

SCALEREADY (16:15 - 16:30)
Simplifying the path to rapid, reliable translation of cell manufacturing through platform, process, and product continuity beginning with the G-Rex Bioreactor
Speaker: Josh Ludwig Global Director, Commercial Operations ScaleReady

SONY Theater 1
EPPENDORF (16:00 - 16:15)
Fueling automated hiPSC production in stirred-tank bioreactors by exploiting multi process parameter monitoring and feedback-based process regulation
Speaker: Dr. Robert Zweigerdt, Hannover Medical School (MHH), Germany

CURATE BIO (16:15 - 16:30)
Curate Cell Processing: Using Deterministic Cell Separation to dramatically improve cell therapy manufacturing starting material quality and quantity
Speaker: Tony Ward, Chief Technology Officer, Curate Biosciences, USA

First in Human Study Design

Chair: Catherine Bollard, MBChB, MD, Children's National, United States
Speakers:

• • Kristin Baird, MD, IQVIA, United States
  • Joycelynne Palmer, PhD, City of Hope, United States

Will CMC Trump Your Data?

Chair and speaker: Qasim Rafiq, PhD, University College London, United Kingdom
Speaker:

• • Anthony Davies, PhD, Dark Horse Consulting, United States

CORPORATE SESSION
CYTIVA

Cell therapy 2.0 – When versatility meets efficiency

Chair: Bill Shingleton, Director, Applications and Biology, UK

Speakers: 

• • Sandeep Kumar, Cell Therapy Advanced Specialist, NHS Blood and Transplant, UK
  • Vicki Remley, IRTA Post-bacc, NIH, US
  • Rohin Iyer, Global Program Manager Market Support, Cytiva, Canada

As one of the major suppliers for cell and gene therapy manufacturers, Cytiva continuously strives to advance and accelerate new therapeutics by bringing innovative solutions to labs around the world. In this session, we will introduce some of our latest developments in a series of short presentations. From process optimization to new products, the solutions presented will span the entire manufacturing process. Presentations will be followed by several breakout sessions that will dive deeper into each topic, allowing you to ask questions to learn from other cell therapy manufacturers. This session will leave you with a better understanding of how you can achieve an optimized and GMP-compliant cell therapy manufacturing process, enabling you to unlock the potential to reach successful commercialization.

Regulatory T Cells: Clinical Applications

Chair: Alexey Bersenev, MD, PhD, Yale University, United States
Speakers: 

• • Maria Grazia Roncarolo, MD, Stanford, United States
  • Megan Levings, PhD, University of British Columbia, Canada

Hematopoietic Stem Cell Engineering for Gene Therapy

Chair: Sandeep Soni, MD, CRISPR Therapeutics, United States
Speakers:

• • Paula Rio, PhD, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Spain
  • Mort Cowan, MD, University of California San Francisco, United States

MSC Trials in COVID-19

Chair: Jan Nolta, PhD, UC Davis, United States
Speakers:

• • Michael Matthay, MD, University of California San Francisco, United States
  • Camillo Ricordi, MD, University of Miami, United States

CAR T Cell Manufacturing 2.0 - Accelerating the Time to Patient Treatment

Chair: Bruce Levine, PhD, University of Pennsylvania, United States
Speakers:

• • Jen Brogdon, PhD, Novartis, United States
  • Charles Calderaro, MBA, Kite Pharma, United States

Pitfalls in Late Stage CMC: What's the Solution

Chair: David DiGiusto, PhD, Resilience, United States
Speaker:

• • Peter Marks, MD, PhD, FDA, United States

FROM FIRST IN HUMAN TO CLINICAL ACCLERATION PLENARY

Allogeneic Cell Therapies: From Proof of Concept to Patient Application

Chair: Michael Gustafson, PhD, Mayo Clinic, United States
Speakers:

• • Wayne Chu, MD, Fate Therapeutics, United States
  • Rafael Amado, MD, Allogene, United States
  • Howard Foyt, MD, PhD, FACP, ViaCyte, United States

This session will highlight the human applications of allogeneic cell-gene therapies, provide clinical updates and discuss some of the challenges in the field. 

• Describe various applications of allogeneic CGT
• Update on clinical trials and patient outcomes
• Identify current challenges in the field

GLOBAL SHOWCASE PRESENTATIONS

SONY Theater 1
ORGENESIS (12:15- 12:30)
Roadmap to enabling CAR-T therapy to be affordable, accessible, available through our decentralized POCareTM Platform.
Speaker: Vered Caplan, CEO Orgenesis

AVENTACELL (12:30 - 12:45)
Human Platelet Lysate Product Innovations to Support Cell and EV Production from Clinical to Commercial Scale
Speaker: Bill Milligan, VP Business Development, AventaCell BioMedical Corp., Co-Chair ISCT APAC Industry Committee, Canada

BIO-TECHNE (12:45- 13:00)
TcBuster Transposon System for Cell Therapy Applications
Speaker: David Hermanson, PhD., Senior Manger of R&D Applications, Bio-Techne, USA

RESILIENCE (13:00 - 13:15)
Partnering To Solve Cell Therapy Funding and Manufacturing Gaps – What Does The Future Hold?
Speaker: Eytan Abraham Ph.D, Business Head Cell Therapy Franchise, Resilience, USA

FUJIFILM IRVINE SCIENTIFIC (13:15 - 13:30)
How to achieve optimal cell immunotherapy bioprocessing results through the scientifically-driven selection of chemically-defined and GMP-grade media and reagents
Speaker: Robert Newman, Chief Scientific Officer FUJIFILM Irvine Scientific

CORPORATE SESSION
MAXCYTE

Scalable Electroporation of Adult Keratinocytes with Multiplexed CRISPR Ribonucleoproteins for the Development of Novel Cell Therapeutics

Chair: Chair: Lesley Eschinger, MaxCyte, USA

Speakers:

• • Celine Mahieu, UCSF, USA
  • Andrew Mancini, MaxCyte, USA

In the past, engineered keratinocyte-based cell therapies have been limited by the lack of efficient transfection methods for adult keratinocytes. Here we aimed to develop a GMP-compliant, scalable cell engineering process using the MaxCyte® cell electroporation platform to transfect neonatal and adult primary keratinocytes from four distinct anatomical locations. Delivery of multiple CRISPR RNPs in a single electroporation achieved highly efficient, multiplexed gene editing in a simple, adaptable process. These improvements in transfection efficiency and cell viability reduced keratinocyte engineering times by up to 4 weeks, with a significantly higher success rate than a standard chemical transfection method. Lastly, we demonstrate the scalability of the MaxCyte electroporation process, enabling the engineering of millions of primary keratinocytes without any loss of efficacy.

LAUNCH AND PATIENT ACCESS PLENARY

You Made It, Now What? Delivering a World-Class, Holistic CGT Launch

Chair: Sven Kili, MBChB, MRCS (Eng), Antion Biosciences SA, United Kingdom
Speakers:

• • Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute, United States
  • Greg Russotti, PhD, Century Therapeutics, United States
  • Warner Biddle, Kite, United States
  • Michaela Gabaldo, MSC, Fondazione Telethon, Italy
  • Luca Pani, MD, University of Miami, United States

This session will discuss the challenges and pitfalls of preparing for and executing a flawless CGT product launch. It will include input from all the major stakeholders, including clinicians, patients, payers, developers and manufacturers.

• What are the critical success factors to be considered in planning a CGT launch?
• In thinking about a successful commercial CGT product – what are the key value drivers for each group of stakeholders?
• What lessons have been learned by the speakers in planning and carrying out a launch?

Make it, don’t break it!  Assuring global commercial success through a strong CGT manufacturing strategy

Chair: Phil Vanek, PhD, GammaBio, United States
Speakers: 

• • Jacqueline Barry, PhD, CGT Catapult, United Kingdom
  • Nicholas Ostrout, PhD, Charles River, United States
  • Derrell Porter, MD, Cellevolve Bio, United States

Shining a Light on Unlicensed CGTs: Where is the Line Between the Right to Care, Ethics, and Patient Safety?

Chair:  Michela Gabaldo, MSc, Fondazione Telethon, Italy
Speakers: 

• • Ilona Reischl, PhD, Austrian Agency for Health and Food Safety, Austria
  • Laertis Ikonomou, PhD, University at Buffalo, United States
  • Simone Boselli, EURORDIS, Belgium
  • Michael Lehmicke, MSc, Alliance for Regenerative Medicine, United States

Are Cell & Gene Therapies a First World only Luxury?

Chair: Sven Kili, MBChB, MRCS (Eng), Antion Biosciences, United Kingdom
Speakers: 

• • Boro Dropulić, PhD, MBA, Caring Cross, United States
  • Maroun Khoury, PhD, Universidad de los Andes, Chile
  • Michael Pepper, MBChB, MD, PhD, Institute for Cellular and Molecular Medicine, South Africa
  • Tay Salimullah, BSc Mgmt, Novartis, United States

Do Accelerated Pathways Live up to Their Promise?

Chair: Sharon Rooney-Mayer, MBA, Novartis, United States
Speakers: 

• • Colin Lee Novick, BA, Innovacell K.K., Japan
  • Wendy L. Corbett, PhD, MBA, Bristol Myers Squibb, United States

Investor Perspectives – Raiders of the Lost Art: Finding the Next CGT Gem

Chair: Reni Benjamin, PhD, JMP Securities, United States
Speaker: 

• • Konstantina Katcheves, MSc, Bristol Myers Squibb, United States
  • Bin Lu, Temasek, Singapore