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.
Maya Furstenau Sharp

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

Envisioning the advanced therapies superior delivery platform

  • End-to-end service solutions for delivery
  • Global platforms for delivery
  • Advancing therapies to commercialization and enabling commercial delivery

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





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?

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


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?

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

Converging technologies driving innovation in Biomanufacturing

  • Further integration of upstream, downstream, formulation development and analytics
  • Increasing the understanding on how the process defines the product
  • Process intensification will be key

GMP for ATMP: an update

  • Bracing for the impact of evolution to GMP guidelines specific to ATMPS
  • How to ready yourself for what’s new and what’s coming in the realm of GMP compliance for ATMPS in Europe?
  • Creative approaches to overcoming Europe’s GMP capacity shortfall: how diverse public and private sector organizations are employing innovative partnering models and initiatives to meet ATMP manufacturing needs at commercial scale
Hardy Tadahisa S Kagimoto, President and Chief Executive Officer, Healios K. K.

Development of a composite RPE cell bioengineered implant to the treatment of geographic atrophy

  • Critical preclinical activities to enable transition to of a stem cell-based product to clinical trials
  • Critical manufacturing considerations for a stem-cell based product
  • Critical design features for a phase 1/2a clinical trial

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

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 

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

Optimising continuous and automated manufacture and bioprocessing

  • Speaking opportunity available
    Please contact Erica Baeta (+44 (0)207 092 1152, for further details

Networking Lunch

Featured during the break: Workshop/panel discussion by Thermo Fisher Scientific

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)

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.

Defining a robust cell culture process control strategy for biologics launch processes – the application and limitations of small-scale and large-scale data

  • Process Control strategy prerequisites – CQA determination and Process Risk assessment (FMEA)
  • SSM Qualification
  • Process Characterization Studies: from De-risking to PAR studies – PAR calculation for critical parameters, NOR definition

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.

Clinical applications of MaxCyte’s cell engineering technology for gene editing

  • Robust non-viral method for transient expression of gene editing tools
  • Minimized risk of “on target, off tumor” toxicity
  • High-performance delivery platform is rapid, automated, and cGMP-compliant
Jessica Pellegrini, Marketing Specialist, 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

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


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

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?

Liver tissue engineering: from extracorporeal liver device to implantable tissues

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

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

Composite ceramic monolithic columns for chromatografic separations

  • Flow properties
  • The challenge of functionalization
  • Impact on biomolecule downstream process

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

Light distilleries of cells and proteins

Proteins and cells are bound on the adsorbent in dark and are recovered by applying light at a specific wavelength
This is a (hopefully) very innovative and safe manner to purify therapeutics that are so biochemically delicate that current purification strategies fail. We call this technology "Light distilleries of cells and proteins
last published: 16/Mar/18 11:35 GMT




World Advanced Therapies & Regenerative Medicine Congress


Erica Baeta
+44 (0)207 092 1152


Jessica Robinson
t/ +44 (0)207 092 1150


Issa Mauthoor
+44 (0)207 092 1257