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Workshop Day

24th February 2020

 Translation

Manufacturing

Development & Commercialisation

A

Learning from Success with Solid Tumours
• TIL therapy and checkpoint inhibitors demonstrate that, unlike CAR-T therapy, natural T-cells can eradicate solid tumours
• Dissection of these successes shows that dominant T-cell clonotypes do not target neoantigens but instead target novel shared epitopes and antigens
• Knowing what works rekindles hope for therapeutic cancer vaccines
Andrew Sewell, Research Director of the Institute of Infection and Immunity, Cardiff University

B

Manufacturing for an Allogeneic Future
• Overcome variation in starting materials with uniform cells sourced from healthy donors
• Control and predict safety and efficacy with standardisedcell banks
• Creating reproducible and consistent products, managing quality specifications
• Explore methods to carry out large scale expansion whilst preserving efficacy and potency
• Lessons learned to translate reproducible, safe and virus free constructs

C

Building Flexibility in Clinical Trial Structure to Meet Unique Trial Needs
• Operational experience executing clinical trials across Europe
• Managing patient population change or new manufacturing aspect without changing clinical trial structure through master protocols
• Understand the agreements in place in trial design protocol to carry out sub studies in a shorter time frame
Reuben Benjamin, Consultant Haematologist, Kings
College Hospital, NHS

D

Redefining Preconditioning Standards for Malignancies Beyond CD19
• Review potential solutions for preconditioning toxicity
• Strategies to reduce the dose of preconditioning to allow quick supply and safe treatment, securing the value in patient safety
• Case study: T4 immunotherapy of head and neck cancer (Phase I trial) – the case for lymphodepletion
• Preclinical evaluation of pan ErbB targeted T4 immunotherapy
• Dose escalation of intratumoural T4 immunotherapy in SCCHN
• Plans and experience to date with lymphodepletion prior to intratumoural T4 immunotherapy
John Maher, Immunology Consultant, King’s College London / CSO, Leucid Bioy

E

Process Development to Improve Scalability and Reduce Cost of Production
Review how manufacturing processes can affect the sensitivity of living drug products
• Outline technology to scale manufacture whilst overcoming negative effects on potency, quality and durability
• Discuss methods to improve process development based on cell biology
Lothar Germeroth, SVP, MD, Juno Therapeutics

F

Getting a TCR-T cell Trial Approved in Europe - Focus on Germany
• Main approval streams in a gene modified cell therapy trial
• Interplay of the different submissions: pitfalls and solutions
• Timeline considerations to achieve a successful start
Kai Pinkernell, CMO & CDO, Medigene & Klaus Tressl, VP,
Quality Assurance and Regulatory Affairs, Medigene

G

CAR T Cells for Solid Tumours, To Do List (TEO):
• Traffic (how to get to the tumours, relevant to silent, non chemoattractant or chemorepulsive tumours)
• Engage (how to penetrate and engage with tumour cells, relevant to immune excluded tumours)
• Overcome (how to overcome immune suppression/compensatory immune suppression, relevant to inflamed tumours)
Francesco Marincola, Chief Scientific Officer, Biotech Refuge

H

Driving Analytical Development to Reduce Time and Cost
• Innovations in analytical testing with automation in batch records
• Discuss the need to speed up the process for QP to release product
• Overcome the burden of release through novel quality, safety and efficacy testing
Therese Choquette, Analytical Project Leader & Senior
Fellow, Novartis

I

Early Evidence Needs to Improve Access and Commercial Potential of CAR-TCR products
• Review value frameworks used by global HTA agencies for marketed CAR-T case studies and impact on
reimbursement status in global markets
• Explore ramifications of payer value frameworks on evidence needs for CAR and TCR therapies and requirements to support value-based pricing of autologous and allogeneic products
• Explore requirements to displace 1st generation cell therapies with 2nd gen products
Aura Mackenzie, Senior Director, AVES, Market Access,
bluebird bio

Translation

Workshop A

Learning from Success with Solid Tumours
• TIL therapy and checkpoint inhibitors demonstrate that, unlike CAR-T therapy, natural T-cells can eradicate solid tumours
• Dissection of these successes shows that dominant T-cell clonotypes do not target neoantigens but instead target novel shared epitopes and antigens
• Knowing what works rekindles hope for therapeutic cancer vaccines
Andrew Sewell, Research Director of the Institute of Infection and Immunity, Cardiff University

Workshop D

Redefining Preconditioning Standards for Malignancies Beyond CD19
• Review potential solutions for preconditioning toxicity
• Strategies to reduce the dose of preconditioning to allow quick supply and safe treatment, securing the value in patient safety
• Case study: T4 immunotherapy of head and neck cancer (Phase I trial) – the case for lymphodepletion
• Preclinical evaluation of pan ErbB targeted T4 immunotherapy
• Dose escalation of intratumoural T4 immunotherapy in SCCHN
• Plans and experience to date with lymphodepletion prior to intratumoural T4 immunotherapy
John Maher, Immunology Consultant, King’s College London / CSO, Leucid Bioy

Workshop G

CAR T Cells for Solid Tumours, To Do List (TEO):
• Traffic (how to get to the tumours, relevant to silent, non chemoattractant or chemorepulsive tumours)
• Engage (how to penetrate and engage with tumour cells, relevant to immune excluded tumours)
• Overcome (how to overcome immune suppression/compensatory immune suppression, relevant to inflamed tumours)
Francesco Marincola, Chief Scientific Officer, Biotech Refuge

Manufacturing

Workshop B

Manufacturing for an Allogeneic Future
• Overcome variation in starting materials with uniform cells sourced from healthy donors
• Control and predict safety and efficacy with standardisedcell banks
• Creating reproducible and consistent products, managing quality specifications
• Explore methods to carry out large scale expansion whilst preserving efficacy and potency
• Lessons learned to translate reproducible, safe and virus free constructs
Helen-Tayton Martin, CBO, Adaptimmune

Workshop E

Process Development to Improve Scalability and Reduce Cost of Production
Review how manufacturing processes can affect the sensitivity of living drug products
• Outline technology to scale manufacture whilst overcoming negative effects on potency, quality and durability
• Discuss methods to improve process development based on cell biology
Lothar Germeroth, SVP, MD, Juno Therapeutics

Workshop H

Driving Analytical Development to Reduce Time and Cost
• Innovations in analytical testing with automation in batch records
• Discuss the need to speed up the process for QP to release product
• Overcome the burden of release through novel quality, safety and efficacy testing
Therese Choquette, Analytical Project Leader & Senior
Fellow, Novartis

Development & Commercialisation

Workshop C

Building Flexibility in Clinical Trial Structure to Meet Unique Trial Needs
• Operational experience executing clinical trials across Europe
• Managing patient population change or new manufacturing aspect without changing clinical trial structure through master protocols
• Understand the agreements in place in trial design protocol to carry out sub studies in a shorter time frame
Reuben Benjamin, Consultant Haematologist, Kings College Hospital, NHS

Workshop F

Getting a TCR-T cell Trial Approved in Europe - Focus on Germany
• Main approval streams in a gene modified cell therapy trial
• Interplay of the different submissions: pitfalls and solutions
• Timeline considerations to achieve a successful start
Kai Pinkernell, CMO & CDO, Medigene & Klaus Tressl, VP,
Quality Assurance and Regulatory Affairs, Medigene

Workshop I

Early Evidence Needs to Improve Access and Commercial Potential of CAR-TCR products
• Review value frameworks used by global HTA agencies for marketed CAR-T case studies and impact on
reimbursement status in global markets
• Explore ramifications of payer value frameworks on evidence needs for CAR and TCR therapies and requirements to support value-based pricing of autologous and allogeneic products
• Explore requirements to displace 1st generation cell therapies with 2nd gen products
Aura Mackenzie, Senior Director, AVES, Market Access, bluebird bio