08:30Registration Opens for Workshops A and B
Choose Workshop A or B
9:00WORKSHOP A: T-Cell based treatment of inflammation: antigen-specific Treg cells, TCR and CAR engineered Treg cells in transplantation, autoimmunity and chronic inflammatory diseases
An Emerging Field with Potential for industrialisation
Regulatory T cells "Treg" naturally control inflammation and induce immune tolerance. As such, these cells can be used as efficient pharmaceutical agents through adoptive cell therapy protocol to treat patients with inflammatory diseases, including autoimmune diseases, chronic inflammation and transplantation. Several subtypes of Treg cells and multiple approaches are being currently tested, not only in models on disease in animals but also in patients in early stage clinical trials. Those approaches include the use of polyclonal Tregs, TCR engineered Treg and CAR-Tregs. For therapeutic purposes, Treg cells can be either isolated directly from peripheral blood using a cell sorting device or differentiated in vitro with specific differentiation cocktails. Taking into account the extremely low frequency/numbers of Treg cells obtained with the two methods, an ex-vivo expansion is considered mandatory for obtaining doses allowing therapeutic intervention. Being able to produce therapeutic Tregs on an industrial scale will be a first step towards meeting the healthcare community’s transplantation needs and its need for treatments for autoimmune and chronic inflammatory diseases. This is especially the case for patients who are refractory to available medications. With such a market potential, a future Treg treatment could very well represent the next breakthrough in the medical landscape,
Arnaud Foussat, Chief Scientific Officer, TXCELL SA

- Zelig Eshhar - Weizmann Institute of Science, Rehovot, Israel
- Kate Mc Donald - University of British Columbia, Vancouver, Canada
- Qizhi Tang - University of California, San Francisco
- James Riley - University of Pennsylvania, Philadelphia
- Ignacio Anegon - Inserm UMR1064, Center for Research in Transplantation and Immunology, France
- Arnaud Foussat - TxCell SA, Valbonne Sophia-Antipolis France
WORKSHOP B: Second Generation CAR-T and TCR Approaches for Enhancing Efficacy and Durability Adoptive cell therapies have captured the imagination of investors and clinicians, having demonstrated potentially transformative patient responses in early clinical trials. However, these therapies can also generate serious, often fatal, toxicities. While many of the side effects of these treatments can be mitigated by the use of steroids and anti-inflammatories, experience with the most effective CAR-T therapies shows that a significant percentage of patients develop life-threatening symptoms that have resulted in a number of patient deaths.

Furthermore, the therapeutic potential of CAR-T therapy has, to date, been primarily limited to hematologic malignancies: against solid tumors, current CAR-Ts have yet to demonstrate meaningful response rates. TCR therapies have yielded promising results in some solid tumor indications, but generally have not been able to show durability.

Cell therapies confound traditional paradigms of dosing. Unlike small molecules and biologics, “therapeutic dosing” in cell therapies is not determined by the number of cells administered. Cells may proliferate out of control after administration, or fail to proliferate or survive at all. In order to control the therapeutic dose with CARs and TCRs, a clinician must be able to regulate the activity of the cells within in the patient. To create truly transformative adoptive cell therapies, we must develop “second generation” constructs that address these problems:

· CARs that exhibit enhanced potency, persistence and proliferation to overcome the challenges of solid tumor indications, but also manage the risks associated with therapyinduced toxicity
· TCRs with enhanced co-stimulatory signaling that solve the issue of tumor-mediated MHC downregulation, preventing TCR-based therapies from detecting cancer-associated antigens
· Cell therapies that can be modulated within the patient, allowing a clinician to prevent potential toxicity or drive efficacy against difficult tumor types

Bellicum Pharmaceuticals has developed platform technologies that have the potential to address these challenges. Chemical Induction of Dimerization (“CID”) utilizes safe, reliable and rapid proteinprotein interactions that can allow a clinician to manage the dose within a patient, modulating the effects of adoptive cell therapies. In addition, the Company’s proprietary MyD88/CD40 (“MC”) dual co-stimulatory domain enables the creation of potentially more efficacious CAR-T and TCR constructs. We will discuss these and other proposed approaches to creating “second generation” adoptive cell therapy products that will deliver the enhanced safety, efficacy and control required to advance cell therapies and overcome the limitations of current approaches.
Peter Hoang, SVP, Business Development and Strategy, BELLICUM
12:00Networking Lunch for All Workshop Attendees
12:30Registration Opens for Workshops C and D
Choose Workshop C or D
13:00WORKSHOP C: CAR T-Cell Workshop Personalized or off-the shelf CAR T-Cells: Will there be a winner or is there room for both?

In this workshop we will compare and discuss personalized and off-the shelf approaches for gene modified cell therapy approaches across a range of product development stages.
· Brief overview of gene modified cell therapies (Including CAR T-Cells)
· Compare (pro/con): Discovery, Preclinical, and Manufacturing/Supply Chain
· Break
· Compare (pro/con): Regulatory, Clinical and Commercial
In the end participants will walk away with a completed “work sheet” detailing whether Personalized treatments or Off-The-Shelf solutions are better in particular categories.. A pros & cons sheet will also be generated for each topic for attendees to take notes on. Topics include Discovery, Preclinical, Manufacturing, Supply
Chain, Regulatory, Clinical and Commercial.
Sicco Popma, Scientific Director and leader of Gene-Modified
Cell Therapies at JBIO (JANSSEN R&D)
WORKSHOP D: Approaches to Re-Imbursement of Adoptive Cell Therapy – with Professor Robert Hawkins, Chief Executive Officer and Co-founder of Cellular Therapeutics Ltd, CRUK Chair of Medical Oncology at The University of Manchester & Consultant in Medical Oncology at The Christie NHS Foundation Trust

Robert’s research interests are in gene and immunotherapy. In addition to clinical training at the Royal Marsden Hospital and Addenbrookes Hospital in Cambridge he was an MRC Research Fellow with Dr Greg Winter and Dr Cesar Milstein at the MRC laboratory of Molecular Biology in Cambridge. His work there was important for the development of commercially successful antibodies. His PhD was in antibody engineering and as a Cancer Research UK Senior Clinical Fellow he developed translational research interests in antibody based gene therapy. He was first appointed as a consultant in Medical Oncology at Addenbrooke’s Hospital in Cambridge in 1995 and then became Professor of Oncology at the University of Bristol in 1996. In 1998 he moved to the Christie Hospital to become Professor and Director of Medical Oncology. Clinically, Professor Hawkins focuses on treating renal cancer and on developing immunotherapy, particularly cellular immunotherapy. He heads a clinical research group undertaking trials and also a range of early phase clinical trials of biological agents including vaccines, antibody based molecules and cell therapies. He has successfully led the clinical development of biological agents from Phase I to Phase III studies. He has received grant funding from Cancer Research UK, the Kay Kendall Leukaemia Fund and the European Union (he is Coordinator of the ATTACK project and was coordinator of the ATTRACT integrated training network) and has published widely in scientific and clinical journals as well as an inventor/co-inventor on several antibody related patents.3

Professor Hawkins led the development of the GMP authorized
facility which has lead to a commercial venture - Cellular
Therapeutics Ltd (CTL).