Advanced Therapies Day One


Advanced Therapies and Regenerative Medicine Day 1 - Wednesday 16th May 2018

08:00 Registration opens

Plenary - The current trends and future implications of ATMP in medicine: How can we benefit patients?


Welcome from Terrapinn


Chair’s opening remarks

Dr Margo R. Roberts

Combination therapy: an update on clinical trial design and translation

Keith Thompson

Keynote presentation: Delivering Advanced Therapies at scale: from manufacturing to the clinic

  • Current technologies will get the therapies into the clinic but how are they going to become mainstream products?
  • What’s needed in manufacturing and supply chain?
  • What’s needed to ensure routine clinical adoption?
Robert Shaw

Industry on the edge: The speed of clinical success and the challenge of commercialization

  • Clinical successes expected in 2017/2018 have provided great momentum and anticipation
  • Major gaps in process industrialization still exist and present challenges to commercial economics
  • Best practices for strategy and implementation provide options for risk mitigation and opportunities to accelerate industrial success
Robert Shaw, Vice President and General Manager of Cell Culture and Cell Therapy, Thermo Fisher Scientific Inc

Networking refreshment break

round tables

Plenary Round Table Discussion Session: Process Development & Manufacturing Focus

Table 1. Regulatory and scientific challenges for global development
Paula Salmikangas

Paula Salmikangas, Director for Biopharmaceuticals & ATMPs, N.D.A. Regulatory Advisory Board

Table 2. Raw material supply for the commercial manufacturing of cell and gene therapies
Frank Hecht

Frank Hecht, Vice President Marketing and Sales, CellGenix GmbH

Table 3. Gene therapy and recombinant viral vectors manufacturing: A variation of the traditional biopharmaceutical production process or a challenge for novel and customized needs?
Brian Bory

Brian Bory, global poduct manager, Sartorius Stedim North America

Table 4. The future of cell therapy manufacturing technologies: All-in-one platforms or unit operation-specific devices?
David Smith

David Smith, Head of Innovation and Engineering, Hitachi Chemicals Advanced Therapeutics Solutions, LLC

Table 5. Strategies for autologous therapy infrastructure
Benoit Champluvier

Benoit Champluvier, Chief Technology And Manufacturing Officer, Bone Therapeutics

Table 6. Implementation and supply chain for next-generation therapies
Table 7. Automation and process development

Speed Networking

A fun, exciting and effective way to make a lot of initial connections (in a very different environment from the standard business networking meetings).
Panel discussion

Regulation, Reimbursement & Commercialisation of Advanced Therapies around the Globe

Developments in clinical trials and translations of CAR-T

New paradigms in manufacture: gene therapy and viral vectors

Automation of manufacture next generation therapeutics

Chaired by: TBC

Chaired by: TBC

Automation of manufacture next generation therapeutics


New paradigms in manufacture: gene therapy and viral vectors

Case study: Meeting the demand of lentiviral vector production - clinical and commercial

  • The route to the world first commercial process for lentiviral vectors
  • Production strategies to meet increasing demands
Automation of manufacture next generation therapeutics

Towards novel solutions for automated stem cell production - challenges and potentials for advanced therapy manufacturing

  • Considerations for achieving end-to-end process automation with adaptive cell processing
  • Examples for automated platform concepts for closed and fully automated production of cells
  • Potential of industry 4.0 for cell manufacture and a glance into the future

Panel discussion: Pricing, reimbursement, and market access for cell & gene therapies: challenges and potential solutions

  • Pricing and reimbursement to-date of pioneering cell and gene therapies in the EU and US
  • Challenges and approaches to defining and demonstrating therapy value, including perspectives on NICE & ICER reviews
  • The range of reimbursement models likely to emerge (multi-year, outcomes-based, other value-based arrangements, etc.)
  • How partners and intermediaries might fit into and shape future value chains

Panel Discussion Continued

New paradigms in manufacture: gene therapy and viral vectors

Bringing the cost of vector manufacture down to tackle the bottleneck of commercial scale autologous manufacture

  • What progresses have been made in vector manufacture?
  • Bringing the cost of vector manufacturing down to improve autologous cell manufacturing
  • What steps need to be taken to make T-Cell processing fully commercially viable

Panel Discussion Continued


Exploiting NK receptors for CAR T cell therapy of cancer

  • The NK cell receptor NKG2D can be effectively engineered as a CAR to provide T cells with ‘NK’ specificity
  • Current clinical testing of the NKG2D CAR (CYAD-01) involves targeting solid and haematological malignancy to examine the potency of the approach
New paradigms in manufacture: gene therapy and viral vectors

New paradigms in manufacturing: gene therapy and viral vectors

  •     Lentiviral vectors are a clinically proven to safely and efficiently generate gene-modified cells, such as CAR-T cells. 
  •     Large-scale Lentiviral vector manufacturing using a chemically defined, serum free suspension (CD-SFS) process has been successfully developed at Lentigen
  •     Tailored process development and manufacturing of cell products using the CliniMACS Prodigy® Technology within a robust and cost-effective workflow

Panel Discussion Continued


Panel Discussion Continued


Off-The-Shelf CAR-Ts: How do we overcome CAR T immunotherapy limitations

  • Gene-editing is a potent technology for the creation off-the-shelf CAR-Ts
  • Potential ease to re-dose a patient of this novel cell therapeutic strategy
  • Universal CAR-Ts to disrupt the treatment paradigm of cancer
New paradigms in manufacture: gene therapy and viral vectors

The production of a novel scFv against coeliac disease – from gene to final product

  • Introduction to coeliac disease and a novel scFV for treatment
  • Overview of the scFV inclusion body process and challenges therewith
  • Integrated Bioprocessing – how does one Unit Operation influence the other in the scFV IB process?
  • Is the final product useful and competitive to available treatment?
Automation of manufacture next generation therapeutics

Implementing Process Optimization to Accelerate Cell Therapy Commercial Readiness

  • Two cases studies will be presented on optimizing approaches for manufacturing at scales necessary for cell-based therapies, (1) human mesenchymal stem cells (hMSCs) on microcarriers, and (2) induced pluripotent stem cells (hiPSCs) as aggregates.
  • Optimized conditions leverage a fed-batch protocol that is easily translated for scale, increases cell yield, and reduces production costs.
  • hMSC and hiPSC characterization for phenotype and function via immunocytochemistry, flow cytometry and cell differentiation to ensure cell-based product quality.
Panel discussion

Investment & funding strategies


Infrastructure and investment to drive pivotal late-stage clinical trials and clinical delivery of cell and gene therapies

  • How do Innovate UK and its family of networks and innovation centres address a range of issues including logistics, clinical delivery and patient tracking?
  • Collecting information that can be used to support pricing and reimbursement.
  • Supporting both pivotal late-stage clinical trials and clinical delivery of cell and gene therapies? 

Clinical development of regulatory T cells (Tregs) to assist solid organ transplantation

  • What we know from clinical trials involving liver and renal transplantation
  • Tackling the challenges of autologous TRegs expansion
  • Steps needed to get to phase II and new work using CARs with T-Regs
New paradigms in manufacture: gene therapy and viral vectors

Viral DSP development, stem cell and cell therapy purification and concentration

  • GMP and coping with the complexity of purifying new format products
  • Tackling the challenges of variability and maintaining high standards of quality during purification
  • Ensuring high purity and lack of contamination when preparing product release
Automation of manufacture next generation therapeutics

Suspension Vero cell line for production of viral vaccines and viral therapeutics

As the acceptances of viral vectors as a delivery system for therapeutics grows, biomanufacturers are looking for an alternative to the classical adherent cell production models

Panel: A new landscape for investment and innovation in the UK

  • How do Innovate UK and its family of networks and innovation centres support innovative advanced therapy businesses?
  • How does government investment support the development of new partnerships and supply chains?
  • How will the UK’s Industrial Strategy drive growth in the sector?

Alex Pemberton, Lead Regenerative Medicine Programme Manager, Medical Research Council
New paradigms in manufacture: gene therapy and viral vectors

Future regulatory considerations for regenerative therapy

  • Current and Future Regulatory Landscape
  • GDP Compliance Standards and Quality Management in the Logistics Space
  • Application of Quality by Design to Address Future GDP Compliance Standards

Panel Discussion Continued


Intra tumour application of CAR-T therapy to treat solid head and neck cancer

  •          Robust manufacture of T4 immunotherapy can be achieved from lymphopenic patients with advanced head and neck cancer using a blood draw

  •          Intratumoural T4 immunotherapy of head and neck cancer has proven safe to date, at doses of up to 100 million CAR T-cells

  •          Cautious dose escalation will proceed in order to define the maximum tolerated dose of this immunotherapy


Panel Discussion Continued

Stephen Badylak

Keynote: The transformation and clinical translation of regenerative medicine

  • Lessons learned from failed stem cell therapy and tissue engineering
  • The role of the immune system in tissue/organ development, homeostasis, response to injury and regeneration
  • The role of nanovesicles in functional tissue reconstruction

last published: 16/Mar/18 11:35 GMT




World Advanced Therapies & Regenerative Medicine Congress


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