Translational Pathway Program

ABOUT THE PROGRAM

The ISCT 2024 Vancouver Translational Pathway Program will feature an extraordinary lineup of five plenaries and 20 concurrent sessions bringing together leading global experts to present preclinical research with high-impact potential, and promising results across phases of clinical trials.

Join us in Vancouver to dive deep into cutting-edge data, uncover groundbreaking innovations and hear from experts on the status of the field and their predictions on its future direction.

La présidentielle showcases the focus and vision of leaders from sister scholarly societies with a focused interest in discovery, development and clinical deployment of Cell & Gene Therapy cures.

Anna Sureda, MD, PhD – President of EBMT – will speak to the unique opportunities and challenges of CGT deployment in the European context. A mirrored American perspective will be provided by Corey Cutler, MD, MPH – President of ASTCT. Amander Clark, PhD – President of ISSCR – will speak to the emerging use of iPSCs as tools and therapies. All will also provide these views in the context of their own research programs.

SCIENCE & INNOVATION

Addresses scientific advances, emerging cell and cell-based therapeutic platforms, as well as technological developments in pre-clinical CGT research with clinical implications.

 PLENARY 

The basic and translational science underlying cell and gene therapies continues to evolve at a breathtaking pace.  Traditional avenues of investigation are rapidly expanding to encompass cutting edge areas including tissue engineering, artificial intelligence and machine-based learning, and advances in imaging and tracking technologies.

Join us as we explore these and other novel technologies and see how these will lead to both better mechanistic knowledge and to future clinical successes.

 CONCURRENT SESSIONS 

Extracellular Vesicles: Potential Tools for Targeted Gene Editing In Vivo

CHAIR: Bernd Giebel, PhD, University of Duisburg-Essen, Germany

SPEAKERS: 

  • Samir El-Andaloussi, PhD, Karolinska Institutet, Sweden
  • Olivier G. de Jong, PhD, Utrecht University, Netherlands
  • Houjian Cai, PhD, University of Georgia, United States




Beyond the Dead (MSCs)

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

SPEAKERS: 

  • James Ankrum, PhD, University of Iowa, United States
  • Tracy Heng, PhD, Monash University, Australia
  • Francesco Dazzi, MD, PhD, AstraZeneca, United Kingdom




MSC Route of Delivery

CHAIR: Katarina Le Blanc, MD, PhD, Karolinska Institutet, Sweden

SPEAKERS: 

  • Mark Lowdell, PhD, FRCPath, FRSB, University College London, United Kingdom
  • Sara McCoy, MD, PhD, University of Wisconsin, United States
  • Sowmya Viswanathan, PhD, University Health Network, Canada




Emerging Technologies in Cell Therapy

CHAIR: Alice Bertaina, MD, PhD, Stanford Medicine, United States

SPEAKERS: 

  • Robert Cockrell, PhD, University of Vermont, United States
  • Jamie Freeman, PhD, Asimov Inc., United States




Advanced Techniques for Regenerative Medicine

CHAIR: TBD

SPEAKERS: 

  • Finn Hawkins, M.B. B.Ch., Boston University, United States
  • Nika Shakiba, PhD, University of British Columbia, Canada
  • Katja Gabriele Weinacht, MD, PhD, Stanford Medicine, United States



TRANSLATION TO CLINIC

This segment addresses topics arising in moving from pre-clinical research to First in Human studies, including results from promising Phase I clinical trials, process development, clinical trial development with an eye to eventual market-authorization and product launch, early-stage CMC, regulatory requirements, and sustainable business models.

 PLENARY 

Cell and gene therapies have evolved from an academic concept to a full-fledged reality, reshaping the landscape of intractable disease treatment. This plenary will delve into the transformative potential of these revolutionary treatments for diseases such as Parkison's, blood cancers, and autoimmune diseases. 

Explore current manufacturing and regulatory challenges and how they are being tackled. Keep up with recent advances and challenges in the development of cellular nanoparticles for therapeutic applications. Join us in unravelling the potential and complexities of these groundbreaking treatments that are at the forefront of medical innovation.

 CONCURRENT SESSIONS 

The State of iPSC-Derived Cell Therapies and Accelerating Clinical POC

CHAIR: Xiaokui Zhang, PhD, Aspen Neuroscience, United States

SPEAKERS: 

  • Adrienne Farid, PhD, Century Therapeutics, United States
  • Xiaokui Zhang, PhD, Aspen Neuroscience, United States
  • Shigeto Shimmura, MD, PhD, Fujita Health University, Japan





Entering the MSC 2.0 Era: MSCs as Cellular Building Blocks for Next Generation Therapies

CHAIR: Jon Rowley, PhD, RoosterBio, United States

SPEAKERS: 

  • Gi-Hoon Nam, MD, PhD, SHIFTBIO Inc., Republic of Korea
  • Sarindr "Ik" Bhumiratana, PhD, EpiBone, United States
  • Remo Moomiaie-Qajar, MD, Cytonus Therapeutics, United States





Accelerating Your Product Timeline to Market by Keeping Up with Process Development Advancements

CHAIR: Lise Munsie, PhD, CCRM, Canada

SPEAKERS: 

  • Elizabeth Wood, PhD, JURA Bio, Inc., United States
  • Nicole Verdun, MD, FDA, United States
  • Nooshafarin Sanaie, PhD, Poseida Therapeutics, Inc., United States





Overlooked and Underappreciated Non-MSC Exosomes

CHAIR: Sai Kiang Lim, PhD, A*STAR Institute of Molecular and Cell Biology, Singapore

SPEAKERS: 

  • Yong Song Gho, PhD, Pohang University of Science and Technology, South Korea
  • Naohiro Seo, PhD, The University of Tokyo, Japan




Purposeful Cellular and Microbiome Engineering—Not Just CAR-Ts

CHAIR: Sowmya Viswanathan, PhD, University Health Network, Canada

SPEAKERS: 

  • Sascha Abramson, PhD, Carisma Therapeutics, United States
  • Carolina Tropini, PhD, PLEng, University of British Columbia, Canada
  • Megan Levings, PhD, University of British Columbia, Canada



FROM FIRST IN HUMAN TO CLINICAL ACCELERATION

Addresses topics arising in moving from Phase I of clinical trials through to marketing authorization, including results of promising Phase II/III clinical trials, discussions of regulatory requirements and barriers to approval, COGs, business models and operational structures to scale up, partnering and investment.

 PLENARY 

Witness the application of CRISPR-edited cells to patients, revolutionizing the very fabric of genetic medicine. Delve into the promising realm of in vivo applications, envisioning a future where gene editing occurs directly within the human body.

Explore the transformative potential of antisense technology in tackling ultra-rare genetic diseases, offering hope to individuals long overlooked by traditional medical approaches. Join us for an impactful journey into the exciting cell and gene therapy frontier where science fiction becomes medical reality.

 CONCURRENT SESSIONS 

Accelerated Approvals: The Viability of Surrogate Endpoints

CHAIR: Mo Heidaran, PhD, GC Therapeutics, United States

SPEAKERS: 

  • Wilson Bryan, MD, Greenleaf Health, United States
  • Kemi Olugemo, MD, FAAN, Ultragenynx, United States
  • David Lebwohl, MD, Intellia Therapeutics, Inc., United States




The Cutting-Edge of Stem Cell Genetic Innovation

CHAIR: Bruce Levine, PhD, University of Pennsylvania, United States

SPEAKERS: 

  • Alice Bertaina, MD, PhD, Stanford Medicine, United States
  • Susan Prockop, MD, Boston Children's Hospital, United States

PANELIST:

  • Jaap Jan Boelens, MD, PhD, Memorial Sloan Kettering Cancer Center, United States




Unlocking the Value of Your Clinical Study

CHAIR: Sven Kili, MBChB, MRCS (Eng), Sven Kili Consulting, United Kingdom

SPEAKERS: 

  • Patrick Rivers, MBA, Aquilo Capital Management, United States
  • Daniella Kranjac, ChEng, MBA, Dynamk Capital, United States
  • Sven Kili, MBChB, MRCS (Eng), Sven Kili Consulting, United Kingdom




From Attackers to Allies: Engineering Cells to Quench the Autoimmune Inferno

CHAIR: Anthony Ting, PhD, Kiji Therapeutics, United States

SPEAKERS: 

  • Michael Aigner, PhD, University Hospital of Erlangen, Germany
  • Tracey Lodie, PhD, Quell Therapeutics, United States
  • Peter Maag, PhD, Kyverna Therapeutics, United States




Enabling Gene Editing Platforms for Novel In Vivo and Ex Vivo Therapeutics

CHAIR: Uma Lakshmipathy, PhD, Thermo Fisher, United States

SPEAKERS: 

  • Bruce Levine, PhD, University of Pennsylvania, United States
  • Fyodor Urnov, PhD, University of California, Berkeley, United States
  • Laura Sepp-Lorenzino, PhD, Intellia Therapeutics, United States



LAUNCH & PATIENT ACCESS

Addresses topics arising in bringing about the clinical adoption of market-authorized CGT products for patients globally, for example, discussions on pricing and reimbursement, business models, scaling up and scaling out, global launch strategies, and real-world evidence.

 PLENARY 

Building upon the experience of late-stage and commercial stakeholders, the session progresses operational excellence and broader patient access to CGTs by serving as a collaborative platform to build the roadmap for maturing our field.

As a community, we recognize the clinical and commercial milestones, and celebrate the transformational impact for patients, but we remain committed to addressing the challenges. This Plenary session will feature experiences and considerations towards operational execution, challenges and concerns about cell/gene/tissue-based therapies for society, and insights into these therapies as viable and sustainable models. Bring your thinking caps to learn, and your hard hats to get to work so that we can continue to pave the future of cell and gene therapies.

 CONCURRENT SESSIONS 

Industrializing Manufacturing & Distribution

CHAIR: Madhusudan V. Peshwa, PhD, Tessera Therapeutics, United States

SPEAKER: 

  • Martin Westberg, MSc, Cytiva, Sweden

PANELISTS: 

  • Nirupama Pike, MDS, PhD, Catalent Pharma Solutions, United States
  • Julie Allickson, PhD, Mayo Clinic, United States





Value and Reimbursement

CHAIR: Tania Bubela, BSc (Hons), PhD, JD, FCAHS, FRSC, Simon Fraser University, Canada

PANELISTS:

  • Doug Danison, MBA, Bayer, United States
  • Dean Regier, PhD, University of British Columbia, Canada
  • Brad Groves, PhD, MAP Patient Access Limited, United Kingdom




Reimagining Patient Access

CHAIR: Mark Flower, BSc, Cryoport, United States

PANELISTS: 

  • Peter Olagunju, MBA, March Biosciences, United States
  • Antinea Chair, BSc, Cellares, United States
  • Julie Kanter, MD, University of Alabama at Birmingham, United States
  • Matthew L. Plaud, MSM, Cryoport, United States




Are In Vivo Cell Therapies the Future?

CHAIR: Madhusudan V. Peshwa, PhD, Tessera Therapeutics, United States

SPEAKERS: 

  • Christopher Wegener, MS, BME, Fresenius Kabi, United States
  • Madhusudan V. Peshwa, PhD, Tessera Therapeutics, United States

PANELISTS: 

  • Dalip Sethi, PhD, MBA, Terumo BCT, United States
  • David Perritt, PhD, Lupagen, United States




Rare Diseases and Patient Access - The Mission and The Challenges

CHAIR: Susan B. Nichols, BSc, Propel Bio LLC, United States

SPEAKERS: 

  • Donald Kohn, MD, University of California, Los Angeles, United States
  • Susan B. Nichols, BSc, Propel Bio LLC, United States