Advanced Therapies Day Two


Advanced Therapies and Regenerative Medicine Day 2 - Thursday 17th May 2018


Keynote session: immuno-oncology and strategies to improve patient success

Alain Vertes

Chair: Alain Vertès, Managing Director, NxR Biotechnologies

Dr Jon Wigginton

Combination approaches for cancer immunotherapy, triggered by experience to date with Anti-CTLA-4 and Anti-PD-1 antibodies

  • There are several challenges to be considered in developing new combination immunotherapy regimens
  • Bispecific antibodies offer a novel approach for targeting multiple immunoregulatory pathways, including PD-1, LAG-3 and CTLA-4 among others.
Fernanda Masri

Process development for T-cell expansion in the ambr15 stirred-tank bioreactor system

  • Cellular immunotherapy bioprocessing
  • Stirred-tank bioreactors and T-cell expansion
  • Process development for T-cell expansion
Dr Frédéric Triebel

LAG-3: the next immune checkpoint after CTLA-4 and PD-1/PDL-1?

  • LAG-3/ MHC class II interactions and their modulation in both cancer and auto-immune diseases
  • Combination therapy with anti-LAG-3 and anti-PD-1 mAb in oncology: where do we stand?
  • Combination therapy with eftilagimod alpha (LAG-3Ig) and chemotherapy or anti-PD-1 mAb
Dr Robert Preti

Are we asking the right questions to achieve deliverable advanced therapies? Lessons from the past and the challenges of the future

  •     An overview of almost forty years of cell therapeutics development
  •     Establishing the potential of cell-based therapies, enabling technologies and organizations
  •     Developing an applicable regulatory framework and guidance and a better appreciation of drug development principles
  •     Themes of the present and future as we advance deliverability of cell-based therapies

Networking refreshment break

Regulation, pricing, reimbursement & market access of advanced therapies around the globe

Moving from phase II to phase III

Cell line development and gene editing for the production therapeutics

Benefits and bottlenecks in continuous and automated manufacture

Chair: Angela Osborne, Co-founder & Director, AMC

Angela Osborne, Managing Director of Strategy and Bioprocess and Phd Biochemical Engineering, eXmoor pharma concepts ltd

Panel discussion: Assessment of health technology and market access globally, challenges and pricing

  • How are the logistical challenges for cell and gene therapies going to work in the years ahead?
  • Affordability and sustainability. How are health authorities going to pay?
  • Process of health technology assessment
  • ROI considerations: what makes this product profitable?

Clinical development of the world’s first iPSC-derived MSC therapeutic product

Experience gained from conducting the world’s first clinical trial of an allogeneic, iPSC-derived cell therapy product
New, second-generation approach to manufacturing therapeutic MSCs that provides a robust, consistent product without reliance upon multiple donors
Progress toward delivering a successful product for graft-versus-host disease and potentially other targets.

Continuous integrated manufacturing of therapeutic proteins

  • New concepts for the up and downstream processing of biomaterials
  • Peculiarities of continuous processing
  • Advances on perfusion bioreactors

Panel Discussion Continued


Cellular medicines for intractable serious and life-threatening diseases

  •     Disruptive cellular technology platforms
  •     Commercial translation capabilities
  •     Advanced pipeline of cellular medicines targeting serious or life-threatening conditions with unmet needs 

Developing cell therapies with iPSC Technologies

  • CDI has four internal cell therapy programs on Ocular, Cardiac, Oncology and Parkinson's disease. Open to strategic partnerships.
  • CDI can offer cGMP iPSC lines from common HLA haplotypes as a platform for cell therapy, with reprogramming license out and contract custom MCB/WCB. Our partners and customers can get a jump start.
  • CDI can provide third parties with contract development and manufacturing services (CDMO) to speed development of their cell therapy project by putting together each party’s unparalleled know-how and expertise

Scalable bioprocessing for manufacturing of functional autologous skeletal implants

  • Comparison across bioreactor systems -  visualization tools for bioprocess efficiency
  • Evaluation of functionality of expanded cell populations in small animal models – setting the target quality profile
  • Development of real-time decision-making tools for optimising bioprocess management for high-risk autologous production
  • Adding a third dimension - controlling complexity in tissue engineered
Ioannis Papantoniou, ATMP Bioprocessing Coordinator, KU Leuven

The French Initiative: the role played by a patient association, the AFM, in the field of the rare diseases in the development of advanced therapies at an international level

  • The Challenge for a Patient Association to play a role in the Development of Medicinal Products
  • Creation of Integrated R&D Structures for Gene and cell Therapies in Orphan Diseases

Cell death for tissue regeneration: the paradox of mesenchymal stem cells

  •     Cell death is a key mechanism in mesenchymal stem cell immunomodulation and tissue repair
  •     Sensitivity to cell death characterise the product
  •     Recipient ability to kill MSC predicts clinical responses to MSC
  •     Recipient determines MSC clinical efficacy 

Maintaining quality with a robust supply chain; a thought to media decisions

  • Robust data set around cGMP media for sustainable production of cell therapy products
  • Streamlined development pathway with Development-by-Design (DbD) through a robust line of key raw material qualifications
  • Case study that maximizes mesenchymal stem cell (MSC) yield with a directly scalable culture method

Chormatogram fingerprinting to follow integrated bioprocesses

  • A toolbox comprising HPLC and chemometric tools allows monitoring the impurity pattern along an entire bioprocess
  • Only minutes after sampling a result is presented and actions can be taken
  • This tool facilitates continuous manufacturing

Developing later stage translation for novel therapeutic approaches

​Defining a new route for gene therapy, challenges and advantages
The correct pathway for industrialising gene therapy and regenerative medicine is still under development, how can we move forward?
What can we expect from gene therapy in 2018?

Process engineering for physiologic stem cell cultivation

  • Isolation of human mesenchymal stem cells in a three-dimensional environment
  • Approaches for upscaling the expansion of stem cells
  • Differentiation under dynamic conditions

High cell density perfusion process of human cells for the production of biopharmaceutical

  •     Human cell-based systems might bring important advantages compared to CHO cells, the workhorses of the biopharmaceutical field
  •     High cell density perfusion process at density up to 100 x 10^6 HEK 293 cells/mL was developed for EPO production to evaluate the potential of these cells for biopharmaceutical production
  •     A novel scale-down perfusion bioreactor of 200 mL working volume was developed

Networking Lunch

Regulatory updates

Clinical development of cell therapy

Manufacture and logistics of next generation therapeutics ​Chaired by: Harald Bradl, Director Cell Culture Development, Boehringer Ingelheim

PAT, process modelling, monitoring and control


Videolink presentation: A Chemistry Manufacturing and Controls (CMC) Perspective on Late Stage Development for a CAR T Cell Product

  • Familiar with the overall lifecycle of a CAR T cell therapy product development pathway
  • Familiar with the basic US FDA regulatory expectations for a CAR T cell product BLA with emphasis on chemistry, manufacturing (CMC) and controls perspective
  • Learn practical “points to consider” for a well-defined CAR T cell manufacturing process in preparation for late stage clinical trials and a biologics license application (BLA)

BrainStorm's NurOwn® treatment for neurodegenerative diseases

  •          Innovative adult Stem Cell therapy

  •          In clinical development for ALS (MND - Motorneuron Disease): 3 completed clinical trials

  •          Mechanism of action and next steps for clinical translation


Performance of single-use bags in cryopreservation conditions

  • Compare mechanical properties of single-use film polymers in cryogenic conditions
  • Understand glass transition temperature and how it relates to single-use bag performance
  • Discover how high purity fluoropolymers are being successfully implemented for cold temperature storage

The regulation of ATMPs in the UK

  • What support is available for companies from early in the development process?
  • Designing clinical trials in a way that comes up with the evidence regulators need to accelerate ATMPs
  • Ensuring safety while reducing cost of development: working together with regulators to make the approval process more efficient
  • Impact of Brexit and other initiatives

Engineering cell therapeutics for controlled drug delivery - clinical case studies

  • Distinguish the pharmacology of cell therapy by classifying therapeutic compartment of action
  • Identify the most appropriate administration route and engineered drug delivery system for a given condition
  • Explore examples of clinically applied cell therapies that have engineered delivery and compare outcomes

Moving from translational early cellular therapeutics manufacturing to larger scale manufacture to support pivotal clinical trials

  • Automation in advanced therapeutics manufacturing
  • Mesenchymal and T-Cell banking
  • Incremental improvement of processes already in use and clinical trial in order to streamline manufacturing and achieve increases in scale

A holistic approach to analytical support, for the biotech industry

  • Growing demand for new analytical tools;
  • Increasing infrastructure costs;
  • Subcontracting of analytical work

Placental derived cell for allogeneic treatment of solid cancer

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  •     Modifying cell secretion for cancer treatment.
  •     Route of cell administration and efficacy.
  •     In Vivo results: Triple Negative Breast Cancer (TNBC) treatment.

Advancing non-viral T-Cell engineering using therapeutically relevant strategies

  •     Non-viral methods of engineering CAR T-cells and delivering gene editing tools have advanced to clinic, but how will they progress from here?  
  •     We describe a key non-viral, enabling technology and the path to the clinic for the treatment of various cancers; and
  •     Discuss strategies for augmenting your current CART programs or developing your next-generation therapy using non-viral cell engineering
Caoimhe Nic An tSaoir, EU/London Sales Manager, MaxCyte

Applications of process analytics in continuous downstream processing

  • Conti flow manufacturing can have an enormous impact on manufacturing in terms of quality, quality and cost
  • Moreover, it is possible to design conti flow systems that are inherently more controllable than batch processes
  • To take advantage of these control possibilities requires us to develop robust and reliable real-time process measurements

Panel Discussion Continued


New data on a novel stem cell combination product for diabetes - development and clinical update

  •     What we know now: experiences from early clinical trials
  •     Developing a Stem Cell product pipeline
  •     Addressing combination product challenges 

Improvements of AAV vector production using the baculovirus system

  • Intensification of rAAV manufacturing
  • Improvement of baculovirus constructs
  • Improvement of AAV vector titers and quality

Practical interpretation of process validation guidelines for the validation of an Antibody Drug Conjugate (ADC)

  • Interpretation of PV guidelines to develop a practical approach for stage I activities (examples of activities and approaches)
  • Executing validation activities and maintaining consistent approaches for multiple processes of an ADC, across different CMOs
  • Coordinating activities across multiple cross-functional teams

Networking refreshment break: Poster Viewing and Presentation Session

Regulatory updates

Advances in chromatography and novel purification methods

Biotech Showcases Chair: Alain Vertès, Managing Director, NxR Biotechnologies

Manufacture and logistics of next generation therapeutics


Industry perspective: overcoming regulatory issues in cell therapy

Nigel Scott, Global Head of Regulatory Affairs, Celixir

Making blood cells in vitro for clinical use: when basic science needs to find the pathway to translational studies

  • Designing basic experiments bearing in mind the long term requirement of affordability and manufacturing process costs
  • Identifying the bottle necks and using genome editing to answer these challenges
  • In vivo environment: technologies that can reproduce it in the dish

Use of 3D printing for the fabrication of custom made anion exchange monoliths

  •     3D printing as fabrication method for chromatographic monoliths
  •     How 3D printing could revolutionise conventional chromatography
  •     Current challenges for the application of 3D printing in separation processes
Ursula Simon, pHD Student, University of Edinburgh

Panel: The regulatory landscape for cell based therapies has changed dramatically in the last 12 months

  • What impact will these changes really have?
  • How is the FDA policing point of care stem cell clinics?
  • Do new regulations really improve patient access to ‘legitimate’ treatments?
  • What needs to be done to improve safe access to cell-based therapies?
  • is the risk benefit ratio really in tune with patient needs?
Nigel Scott, Global Head of Regulatory Affairs, Celixir

Human extracellular matrix as the key tool for liver tissue engineering

  • Basic concept of tissue engineering
  • Decellularization-recellularization technology
  • Hepatic regenerative medicine

Strategies for upscaling the manufacturing of liver-derived mesenchymal stem cells

  • Maximizing the expansion potential using working cell banks
  • Advantages of chemically defined media
  • Implementing single-use bioreactors
last published: 14/May/18 16:05 GMT







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