Translational Pathway Program

Cell and gene therapies represent a significant achievement, not only for the biotech and life sciences industry but also, and importantly, for patients who have witnessed the lifesaving potential of these new biotherapeutics.

The progress for patients could not have been realized without the development of new technologies and this session brings together global experts to discuss the innovations that will shape the future of the field.

1. The Rise of Mitochondrial Transfer Technology in Regenerative Medicine

• Chair: Rachele Ciccocioppo, MD, AOUI Policlinico GB Rossi & University of Verona
• Speakers: 
  • Giuseppe Orlando, MD, PhD, Marie Curie Fellow, Wake Forest University School of Medicine
  • Marvin Edeas, MD, PhD, Université de Paris, Institut Cochin, INSERM U1016
    

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2. Promise and Obstacles of Cell-Derived Nanovesicular Therapies

• Chair: Eva Rohde, MD, Paracelsus Medical University Salzburg
• Speakers:
  • Pieter Vader, PhD, UMC Utrecht University
  • Ewa Zuba-Surma, PhD, Jagiellonian University in Krakow
  • Mario Gimona, PhD, Paracelsus Medical University Salzburg

3. Novel Genome Editors and their Application in Human Disease

• Chair: Alice Bertaina, MD, PhD, Stanford School of Medicine
• Speakers:
  • Benjamin Kleinstiver, PhD, Mass General Hospital & Harvard Medical School
  • Shengdar Tsai, PhD, St. Jude Children's Research Hospital

4.Scaffolding and Extracellular Matrix

• Chair: Giuseppe Orlando, MD, PhD, Marie Curie Fellow, Wake Forest School of Medicine
• Speakers:
  • Graziella Pellegrini, PhD, Università degli Studi di Modena e Reggio Emilia
  • Ivan Martin, PhD, University of Basel
  • Riccardo Levato, PhD, Utrecht University

5. Organs-on-Chips: Advanced Culture Systems to Shape Future Therapies

• Chair: Ivan Martin, PhD, University of Basel

• Speakers:
  • Paola Occhetta, PhD, Politecnico di Milan
  • Jose Luis Garcia Cordero, PhD, Roche Institute for Translational Bioengineering

As the demand for advanced therapies continues to grow, improvements in CGT manufacturing workflow are becoming increasingly necessary. Despite recent advances, the translation of cell and gene therapies to the clinic is still hampered by obstacles that prevent these innovative technologies from becoming standards of care.

This session brings together eminent speakers that will provide insights into how to navigate the road to the clinic successfully.

1. Progress and Challenges with Gene Editing

• Chair: Patrick Hanley, PhD, Children's National Hospital
• Speakers:
  • Mick Fellows, PhD, AstraZeneca
  • Paula Rio, PhD, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
  • Shin Kaneko, MD, PhD, Kyoto University

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2. Advancing iPSCs for Clinical Use

• Chair: Jacqueline Barry, PhD, Cell and Gene Therapy Catapult
• Speakers: 
  • Tenneille Ludwig, PhD, WiCell Stem Cell Bank
  • Massimiliano Gnecchi, MD, PhD, FESC, University of Pavia & Fondazione IRCCS Policlinico San Matteo
  • Kapil Bharti, PhD, National Institutes of Health

3. Extracellular Vesicles are Getting Hotter than Ever

• Chair: Bernd Giebel,  PhD, University of Duisburg-Essen
• Speakers: 
  • Antonio Salgado, PhD, University of Minho
  • Amanda Silva, PhD, Université Paris Cité
  • Eva Rohde, MD, Paracelsus Medical University Salzburg

4. New Frontiers in CGT for Cardiovascular Diseases

• Co-Chairs: 
  • Massimiliano Gnecchi, MD, PhD, FESC, University of Pavia, & Fondazione IRCCS Policlinico San Matteo
  • Giulio Pompilio, MD, PhD, CARE working group of the European Society of Cardiology
• Speakers:
  • Doris Taylor, PhD, FACC, FAHA, Organamet Bio Inc.
    
  • Kikuo Yasui, MSc, Heartseed Inc.
  • Silviu Itescu, PhD, MBA, Mesoblast Inc. 
    
    

5. Next-Generation MSC Therapies

• Chair: Dominique Farge, MD, PhD, Hôpital St-Louis

• Speakers: 

• • Khalid Shah, PhD, Harvard Medical School
  • Sam James, MD, PhD, University College London
  • Massimo Dominici, MD, PhD, Università degli studi di Modena e Reggio Emilia
    
    

Biotherapeutics will change the way healthcare is delivered in the future. When organ restoration and tissue engineering become mainstream, we may be able to improve the way organs function. While academic healthcare continues to lead innovation, progress in commercialization offers potential to capitalize on the science.

This session will focus on improving healthcare for patients globally by integrating post-approval considerations during clinical trial phases of development.

1. Tackling Challenges in Cell Therapy for Solid Tumors

• Chair: 

  • Bruce Levine, PhD, University of Pennsylvania
    
    

• Speakers:

  • Christina Coughlin, MD, PhD, CytoImmune Therapeutics, Inc.
    

  • Adrian Hayday, PhD, King's College London
    

  • Khalid Shah, MS, PhD, Harvard Medical School

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2. Quality, Process Development, Process Optimization

• Chair: Christopher Bravery, PhD, Advbiols

• Speakers:

  • Kowid Ho, PhD, F. Hoffmann-La Roche

  • Christiane Niederlaender, PhD, Parexel International
  • Florence Salmon, PhD, Ridgeline Discovery

3. Allogeneic vs Autologous Debate: Developing Cell Therapies with an Eye to Patient Access 

• Co-Chairs: 
  • Julie Allickson, PhD, Mayo Clinic's Center for Regenerative Biotherapeutics 
  • Bruce Levine, PhD, University of Pennsylvania
• Speakers:
  
  • David Sourdive, PhD, Cellectis
  • Catherine Bollard, MBChB, MD, Children's National Hospital
  • Matthew Hewitt, PhD, Charles River Laboratories
    
  • Saad Kendarian, MB, ChB, Mayo Clinic

4. Implementing the First Mile of Big Data Early to Deliver Smarter, Scalable Manufacturing

• Chair: Dominic Clarke, PhD, Discovery Life Sciences
• Speakers: 
  • Kevin Gordon, MSIS, Ori Biotech
  • Mark Lowdell, PhD, FRCPath, FRSB, University College London
  • Najib Rehman, ATMPS

5. Investor Accelerator, What’s of Interest During Clinical Acceleration

• Chair: Bryan Poltilove, MBA, BroadOak Capital Partners
• Speakers: 
  • Julian Hanak, MSc, Purespring Therapeutics
  • Geoffroy De Ribains, DEA, AdBIo Partners

Taking a retrospective approach, this session explores what needs to happen in the coming years to realize the potential of advanced therapies including cell, gene, and tissue engineering. 

The session will integrates important perspectives that will shape post-launch success focusing on investment, scaling manufacturing and global distribution, driving patient access, integrating into clinical practice, and navigating regulatory oversight to achieve market success and greater global availability.