Organized by the ISCT Business Models & Investment Commercialization Sub-Committee, the inaugural Investigators to Investors (I to I) Pre-Conference Workshop will bring together cell and gene therapy investors for a half-day educational event providing unparalleled access to cell and gene therapy key opinion leaders.
Target Audience & Access
Access is reserved for Investors and Analysts interested in learning about the latest advancements in cell and gene therapy translation, and accessing leading experts from across the commercialization pipeline.
Access to this Pre-Conference Workshop is included in ISCT 2021 registration.
Session 1 – Ask the Experts
90-minutes: 10:00-11:30 ET
(What Time Is This For Me?)
Rapid fire panel discussion on the latest advancements in CGT – Ask your critical questions to leaders in cell and gene therapy clinical manufacturing, clinical trial design, therapeutic delivery and product commercialization. The track will commence with a state of the industry overview and panel discussion covering topics crucial to investor due diligence.
Jaap Jan Bolens, MD, PhD
Chief of Transplant & Cellular Therapy
Memorial Sloan Kettering
Founder and Chief Scientific Officer, EVP of Research and Development
Sven Kili, MD
United Kingdom / Switzerland
Bruce Levine, PhD
Professor of Cell and Gene Therapy
University of Pennsylvania
President & Chief Commercial Officer
State of the Industry Address Provided by:
Managing Director, Senior Biotech Analyst
Patrick Rivers, MBA
Co-Chair, ISCT BM&I Sub-Committee
Director of Research
Aquilo Capital Management
Session 2 – Meet the Experts
120-minutes: 12:00-14:00 ET
(What Time Is This For Me?)
Opportunity to engage in four small group 30-minute roundtable breakouts with Cell and Gene Therapy experts. Breakouts will consists of 4-6 investors per KOL; appointments available on a first come first served basis.
Additional Experts To Be Announced
Clinical Development Expert Roundtable Leaders:
Catherine Bollard, MD, MBChB.
Director of the Center for Cancer and Immunology Research at the Children’s National Research Institute
Catherine Bollard, MD, MBChB, is the Bosworth Chair for Cancer Biology, Director of the Center for Cancer and Immunology Research at Children’s National Hospital and Professor of Pediatrics and Immunology and Associate Center Director for Translational Research and Innovation at the GW Cancer Center at The George Washington University in Washington, DC. Her training includes She received medical degrees from the University of Otago in Dunedin, New Zealand and is board certified in pediatrics and hematology. Dr. Bollard has held numerous national and international positions including current president of the Foundation for the Accreditation of Cellular Therapy (FACT) and past president of the International Society for Cellular Therapy (ISCT). Dr. Bollard She served on the Cellular, Tissues and Gene Therapies Advisory Committee for the Food and Drug Administration (FDA) from 2015-2019. An associate editor of the journal Blood since 2014, she will serve as editor-in-chief of Blood Advances beginning in September 2021. Dr. Bollard has more than 200 peer-reviewed publications and has been continuously and independently NIH-funded for over a decade. Her bench and translational research focuses on improving outcomes for patients after bone marrow and cord blood transplantation and on the development of novel cell therapies for viral diseases and cancer.
Massimo Dominici, MD
Full Professor, Medical Oncology and Director, Division of Medical Oncology Residency School of Medical Oncology Program in Cellular Therapy and Immuno-oncology Laboratory of Cellular Therapy University Hospital of Modena
MD degree at the University of Pavia in 1996. Post-graduate internship at the Vienna University (Austria). Residency in Hematology at the University of Ferrara. Post-Doctoral Associate at St. Jude Children’s Hospital in Memphis (USA, 2000-2003). Currently, associate professor, director of the Residency School of Medical Oncology, of the Division of Medical Oncology and of the Program of Cellular Therapy and Immuno-oncology at the University Hospital of Modena and Reggio Emilia (Italy). Since 2005 he is heading the Laboratory of Cellular Therapies. More than 140 papers with more than 21000 citations, 12 patents, five book chapters published, three books published. 22 grants as principal investigator with 11 on competitive calls, 10 grants as co-investigator all on competitive calls, 11 grants from biotech industries for sub-contracting investigations with a total of 14,6 Mio € since 2004. Scientific founder of the university start-up Rigenerand raising 12,5 Mio € since 2009. Co-founder and coordinator of the Mirandola Science & Technology Park. 170 invited lectures as plenary speaker between national, international meetings and for University lectures, 14 peer reviewed oral communication and 31 posters presented in international meetings. Berlucchi Award for Research in Oncology (2012); “Fanfullino della Riconoscenza Premio Città Di Lodi” (2017); International Award Pico della Mirandola (2017). Referee and advisor for 16 research founding Bodies. He has been co-founder of the Forum of Italian Researcher on MSC (FIRST), board member of JACIE, WBMT and scientific advisor for the Italian Minister of Health. He has been member of ISCT, ASH, ESCGT, IFATS, IPLASS. He has been President of ISCT 2014-2016, now member Emeritus, Member of the Strategic Advisory Council and Chair of the ISCT Presidential task-force on unproven cell and gene therapies.
Richard T. Maziarz, MD
Medical Director, BMT & Cellular Therapy Program, Oregon Health and Science University
Program at the Oregon Health and Science University Knight Cancer Institute in Portland, OR. He received his medical degree from Harvard Medical School, his hematology/medical oncology training at the Dana-Farber/ Brigham and Women’s Cancer Center and has been in practice for more than 35 years. He specializes in blood and marrow transplant, immunology, leukemia, lymphoma, myeloma, and graft-versus-host disease. He also has been recently involved with the expansion of immune effector cell therapy applications for patients with hematologic disorders.
Manufacturing and Scale Up Expert Roundtable Leaders:
Mark Lowdell, PhD FRCPath FRSB
Professor of Cell & Tissue Therapy University College London, Director of the Centre for Cell, Gene & Tissue Therapeutics, Royal Free Hospital, London
Professor Lowdell is a qualified diagnostic immunopathologist as well as holds MHRA Qualified Person status for the release of ATMPs for clinical trials. He is a past Vice President (Europe) of ISCT and has over 25 years’ experience in cell and gene therapy. Much of his career has focused on adoptive immunotherapy of cancer and has targeted the role of the innate immune responses; especially the role of natural killer (NK) cells, although his experience includes a variety of adoptive T cell therapies (cancer and viral infection) as well as dendritic cell vaccines and CAR-T cells.
He established the first academic GMP facility in the UK for the production of human cells as medicines in 2003. This GMP facility now produces somatic cell medicines, gene therapies and tissue engineered products and is particularly adept at developing processes for scale-up of ATMP production.
Professor Lowdell is a consultant to or founder of many of the key companies involved in ATMP development in the EU and USA including Achilles Therapeutics Ltd, Autolomous Ltd, Autolus Ltd, NWBio Therapeutics and INmuneBio Inc, which he helped take through Nasdaq listing and for which he acts as CSO.
Micheal Scott, PhD
Senior Vice President of Product Development, BlueRock Therapeutics
Dr. Scott and the BlueRock teams develop cellular medicines that integrate therapeutic cells, clinical formulations and medical device technology to create products targeted to treat otherwise intractable and unmet clinical needs. He leverages 10 years of product development of cell therapies, medical devices and combination products on product and manufacturing process development. Prior to BlueRock Dr. Scott was Chief Development Officer of ViaCyte, a clinical-stage cell therapy company focused on treatment of insulin-requiring diabetes. Prior to that he spent almost two decades development cardiac medical devices to treat valvular heart disease and heart failure.
Regulatory Expert Roundtable Leaders:
Jacqueline Barry, PhD
Chief Clinical Officer, Cell and Gene Therapy Catapult
Jacqueline Barry is the Chief Clinical Officer, responsible for Clinical Translation and Delivery activities and an Executive Director of the Cell and Gene Therapy Catapult. She has extensive experience in the development of advanced therapy medicinal products and leads a multi-disciplinary team of Nonclinical, Regulatory, Clinical Operations and Programme Management specialists. She also leads the coordination of the UK Advanced Therapy Treatment Centre Network and is a Director of the Global Alliance for iPSC Therapies.
She feels passionately about making advanced therapies available for patients and works closely with the MHRA and the NHS on the development of the ecosystems to support the adoption of these therapies.
Prior her time at Cell and Gene Therapy Catapult, Jacqueline worked at the Scottish National Blood Transfusion Service where amongst other activities she designed the regulatory strategy for the Cellular Therapies for the Blood Transfusion Service and acted as Qualified Person for their release. She has a PhD from the University of Aberdeen and did post-doctoral research in neurophysiology at the University of Edinburgh.
Paula Salmikangas, PhD
NDA Advisory Board
Paula Salmikangas is a Director of ATMPs and member of the Advisory Board at NDA. She is a clinical biochemist by training and holds a Ph.D. in molecular and cell biology. Dr. Salmikangas has background as a research scientist and she is an Adjunct Professor of Biochemistry for the University of Helsinki.
Before joining NDA Dr. Salmikangas worked as a Research Professor at the Finnish Medicines Agency (2003-2017), acting as the Finnish member and Vice-Chair of the Committee for Advanced Therapies (CAT) at the European Medicines Agency from 2009 to 2014 and as the Chair of the CAT 2014-2017. She has also been the Chair of the Cell Products Working Party (CPWP) and a member of the Biologics Working Party (BWP). Her main areas of expertise are biological medicinal products, especially ATMPs, including CMC, regulatory and immunogenicity aspects of biopharmaceuticals.
Commercialization Expert Roundtable Leaders:
Head Alliance Management & Regulatory Affairs, Fondazione Telethon, SR-TIGET San Raffaele Telethon Institute for gene therapy
Michela Gabaldo is Head, Alliance Management & Regulatory Affairs at Fondazione Telethon in Milan (Italy). She is an talented leader and expert in regulatory affairs with more than 20 years experience in drug development . She’s currently supporting the 2 internal Scientific Institutes in Italy, SR-TIGET (San Raffaele-Telethon Institute for gene therapy in Milan) and TIGEM (in Naples) in providing global strategic direction in regulatory affairs for the progression of gene therapy (ex-vivo and in-vivo) and gene editing projects tackling rare genetic disease at different stages of development spanning from research and pre-clinical up to patient access. These programs are developed both internally and in partnership with relevant pharmaceutical industries or through start-ups. Within this role she’s been actively involved in the development and access of the 1st ex-vivo gene therapy registered worldwide – Strimvelis – in 2016 and in the Libmeldy program who has reached recently the EU approval.
Before joining Telethon in 2011 she has been working in the pharma industry for GSK in Clinical Operations and CMC Regulatory Affairs areas for New Chemical Entities (NCE) development.
After graduation in Pharmaceutical Chemistry and Technology, she granted a Master Degree in Regulatory Affairs and Market Access for NCEs and Biopharmaceuticals.
Winner of 2017 edition of TopRA Award in the Future category, Michela offers up to date knowledge of the most recent regulations in ATMPs and Orphans.
She’s currently a member of the IRDIRC (International Rare Disease Reasearch Consortium) Therapies Scientific Committee where as core member she has actively contributing to the development of the IRDIRC orphan drug development guide (ODDG).
Michael May, PhD
President & CEO, Centre for Commercialization of Regenerative Medicine (CCRM)
Michael May is President and CEO of the Centre for Commercialization of Regenerative Medicine (CCRM), a Canadian, not-for-profit that develops technologies, launches new companies and catalyzes investment in the field of regenerative medicine, including cell and gene therapy. Prior to CCRM, Michael was the President, and co-founder, of Rimon Therapeutics Ltd., a Toronto-based tissue engineering company developing novel medical polymers that possess drug-like activity.
Michael sits on a number of boards and advisory committees including: the ARM Foundation; AgeX Corporation; panCELLa Inc.; CCRM Enterprises; the International Advisory Board of the Swedish Centre for Advanced Medical Products (CAMP); the Poietis SAB; the Entrepreneurship Leadership Council at the University of Toronto; the TO Health! Human & Science Steering Committee; the Cell and Gene Therapy Insights, Editorial Advisory Board; the Commercialization Committee of the International Society for Cell and Gene Therapy; He is the Chairman of ExCellThera Ltd.
Emanuele Ostuni, PhD, MSc
Head of Europe, Cell and Gene Therapy, Novartis Oncology
Emanuele is Head of Europe for Cell and Gene Therapies at Novartis Oncology, where he oversees all commercialization aspects of this portfolio in Europe, including Kymriah. With his team, he focuses on expanding access for this one-time therapy across Europe – they have innovated business and reimbursement models and executed them since September 2017.
Emanuele is with Novartis since 2012. He was with Sandoz in the Global Strategy Team, then the Head of Specialty and Hospital Franchises in Central/Eastern Europe, and later as Business Unit Head, Rx in Romania.
Prior to joining Novartis in Europe he was based in the USA where he was Vice President of Business Development at Nano Terra, Engagement Manager at McKinsey & Co and a Senior Research Investigator at Surface Logix. Emanuele was also the co-founder of Enumeral Biomedical and served on its BOD through its public listing in 2014. He currently serves on the Scientific Advisory Board of Anixa Biosciences.
Emanuele holds a PhD in Physical Chemistry from Harvard University and BS and MS degrees in chemistry from Georgetown University.
2021 I to I Pre-Conference Workshop Organizing Committee:
ISCT Business Models & Investment Commercialization Sub-Committee
Sven Kili, MD
Co-Chair, ISCT BM&I Sub-Committee
United Kingdom/ Switzerland
Patrick Rivers, MBA
Co-Chair, ISCT BM&I Sub-Committee