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Informa
08:55 - 09:00 5 mins
Main agenda
Chairperson's Opening Remarks
  • Anthony Davies - President, Dark Horse Consulting, USA
more
09:00 - 09:20 20 mins
Main agenda
Regulatory Challenges in ATMP development
  • Renske ten Ham - Division of Pharmaco-epidemiology and Clinical Pharmacology, Utrecht University, The Netherlands
more

Since 2007 Advanced Therapy Medicinal Products (ATMPs) are specifically regulated in the EU. So far, a limited number of ATMPs have been approved in the European Union despite considerable development activity in this field. As part of the Escher platform for regulatory innovation, Lygature, together with EFPIA, EBE and Utrecht University has carried out a project researching factors associated with success and failure in the development and commercialization of ATMP products in Europe.

  • Challenges encountered by commercial developers in ATMP development within Europe?
  • Barriers and drivers in successful navigation of the EU Regulatory landscape
09:20 - 09:40 20 mins
Main agenda
Opportunities & Challenges Developing Joint CMC Regulatory Strategies for Gene & Cell Therapies – FDA/EMA/PMDA
  • Joann Parker - Senior Director, Regulatory CMC, Pfizer, USA
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  • Overview of the regulatory landscape for FDA/EMA/PMDA
  • Focus areas where different expectations can lead to challenges and divergent strategies
  • Approaches to developing aligned strategy
09:40 - 10:00 20 mins
Main agenda
GMP inspections at ATMPs manufacturing sites: recurrent deficiencies
  • Simona Russo - GMP Inspector, Italian Medicines Agency, Italy
more
  • Good Manufacturing Practice (GMP): regulations and guidelines relevant to ATMPs manufacturers
  • The experience of the Italian GMP inspectorate in the oversight of both academia and industry ATMPs manufacturers
  • Challenges and major outcomes of GMP inspections
  • Examples of recurrent deficiencies raised during GMP inspections
10:00 - 10:20 20 mins
Main agenda
Impact of CMC on safety and efficacy of ATMPs
  • Paula Salmikangas - Director of Biopharmaceuticals and ATMP, NDA Advisory Services Ltd., Finland
more

The approval of first Advanced Therapy Medicinal Products (ATMPs) both in EU and US have raised high expectations towards novel cell- and gene-based medicinal products, especially for indications with high unmet medical needs. On the other hand, clinical trials of first CAR-T cell products have shown the difficulties related to the substantial genetic modification of T-cells in the form of severe adverse reactions, cytokine release syndrome (CRS) and neurotoxicity. It is well known that for cell-based products the product composition, quality and control of product variability are critical for safety and efficacy.  For CAR-Ts certain signaling domains seem to be related to better cell persistence, whereas impact of the T-cell composition / characteristics on the product safety has not yet been clarified. For many cell-based products long in vitro culture has been associated with higher safety risks (e.g. tumourigenicity) and lower efficacy e.g. due to dedifferentiation or apoptosis of cells, leading to restrictions in cell expansion and lower cell doses. This presentation will discuss importance of early product design and how the balance between quality, safety and efficacy could be achieved.

10:20 - 11:05 45 mins
Main agenda
Morning Coffee and Networking
11:02 - 11:05 3 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Chairperson's Opening Remarks
  • Reinout Hesselink - Cell Therapy Consultant, Exmoor Pharma Concepts Ltd., The Netherlands
more
11:02 - 11:05 3 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Chairperson's Opening Remarks
  • Hanna Lesch - Research & Development Director, FinVector Vision Therapies
more
11:02 - 11:05 3 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Chairperson's Opening Remarks
  • Boro Dropulic - Chief Science Officer and General Manager, Lentigen Technology Inc., A Miltenyi Biotec Company
more
11:02 - 11:05 3 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Chairperson's Opening Remarks
  • Stefano Baila - Director Operations & Business Development, Holding F.I.S., Belgium
more
11:05 - 11:40 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Meeting the challenge of delivering autologous therapies in the commercial sector
  • Owen Bain - Head of ATMP Quality Control, Centre of Cell Gene and Tissue Therapeutics, University College London, UK
more
  • Case study
  • What is the future for autologous therapies?
  • Are they commercially viable
  • What are the strategies for ensuring that they are?
11:05 - 11:40 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Quality aspects when developing cell based products, assessor’s reflections from scientific advice, CTA and MAA
  • Axel Ståhlbom - Non-Clinical Assessor, Medical Products Agency, Sweden
more
  • Starting material & Raw material
  • Process design
  • Control strategy
  • Process validation
11:05 - 11:40 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Opportunities and Challenges of translating Cell and Gene Therapies in Academia
  • Pamela Tranter - Head Translational Research Group, Translational Research Office, UCL, UK
more
  • Review of the exciting and diverse pipeline of cell and gene therapy projects within UCL with example case studies

  • Opportunities for project funding: internal, public funds, industry partners and university spin outs

  • Tackling the challenges of GMP manufacturing capability and capacity



11:05 - 12:15 70 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Clinical regulatory breakfast surgery
  • Diego Ardigò - Project Leader Advanced Therapies, Corporate Drug Development, Chiesi Farmaceutici S.p.A., Italy
  • Simona Russo - GMP Inspector, Italian Medicines Agency, Italy
more

Informa is looking for 5-6 CAT members and industry representatives to join a panel to address various concerns for those working in the clinical space for both cell and gene therapies – attendees will be able to submit questions in advance.

Points to be discussed include:

  • Hospital exemptions (non-centralised procedures, national market authorisation, dis-harmonisation between member states)
  • Regulations surrounding Phase I and II studies – Europe specific
  • Regulatory CMC (release testing, criteria for release testing, comparability)
11:40 - 12:15 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Dual Dialogue: The next wave of cell therapy product – Allogeneic manufacturing solutions
  • Jan Spanholtz - CSO, Glycostem Therapeutics, The Netherlands
  • Sandra Van Wetering - COO, DC Prime, The Netherlands
more
  • Strategies for manufacturing an ‘off the shelf’ product
  • Regulatory perspectives on this
  • How can you ensure large batch sizes
  •  Scaling out solutions
11:40 - 12:15 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Control strategies and setting specifications
  • Marta Germano - Principal Scientist, Janssen, The Netherlands (to be confirmed)
more
11:40 - 12:15 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Novel Vectors for the treatment of cancer
  • Wenliang Dong - COO, ORCA Therapeutics BV, The Netherlands
more
  • Viral vectors for the treatment of cancer
  • Challenges in production of viral vectors
  • What are the potential combination therapies that will make a difference in treating cancers?


12:15 - 12:45 30 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Introduction to Yposkesi
  • Alan Lamproye - CEO, YposKesi, France
more
12:15 - 12:30 15 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Manfacturability of Viral Therapeutics
  • Rolf Werner - Professor, University of Tuebingen, Germany
more
  • Selection of viral vectors or oncolytic viruses for therapeutic approach
  • Selection of host cell and optimization of virus titer
  • Media optimization for cell growth, transfection and virus production
  • Adherent or suspension cell culture process with perfusion system for high cell density
  • USP Equipment and process alternatives
  • Harvest process equipment alternatives
  • DSP Process selection and process alternatives
  • Stable formulations for viral therapeutics
  • Analytical methodes
  • GMP facility design and operation
  • LMK Center of Excellence for process development and GMP manufacturing of viral therapeutics
12:15 - 12:45 30 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Automated workflows to generate autologous gene-modified cellular products
  • Boro Dropulic - Chief Science Officer and General Manager, Lentigen Technology Inc., A Miltenyi Biotec Company
more
  • How Lentiviral vectors are a proven robust technology to genetically modify cells
  • The Development of a Large-scale Lentiviral vector Manufacturing Process using a Chemically Defined, Serum Free Suspension Bioreactors
  • How automation using the CliniMACS Prodigy is a robust and cost effective method to generate patient specific CAR-T cells
  • The design and testing of CAR constructs - factors that influence in vivo efficacy
  • How automation provides options for the manufacture of CAR-T cell products: Centralized vs Decentralized models
12:15 - 12:35 20 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Optimized PEI-mediated production of clinical grade viral vectors, PEIpro® and PEIpro®-HQ
  • Valerie Kedinger - in vivo and Bioproduction Application Specialist, Polyplus Transfection, France
more

Gene- and cell therapy-based medicines are experiencing resurgence due to the advances made in developing new viral vector systems guided by safety, specificity and potency considerations. Adeno Associated Virus (AAV) and Lentivirus are very commonly used in therapeutics and often produced using PEI-mediated transient transfection in HEK-293 or HEK-293T cells. The critical raw materials needed for cGMP vector production must be sourced from approved suppliers and should have gone through a rigorous testing program to reduce the risk of introducing adventitious agents into the production process. PEIpro® and PEIpro®-HQ, provided and manufactured by Polyplus-transfection®, are the unique PEIs developed for transfection and suitable for virus production from process development up to use in GMP processes, respectively.

12:30 - 12:50 20 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Quality by Design approach for development and optimization of T cell-based processes
  • Alexandre Lennaertz - Development Manager, MaSTherCell, Belgium
more
12:45 - 14:00 75 mins
Main agenda
Lunch and Networking
13:58 - 14:00 2 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Chairperson's Opening Remarks
  • Jakub Cierny - Senior Quality Compliance Manager, Sotio AS, Czech Republic
more
14:00 - 14:35 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Ex vivo gene therapies - the need for process scale up and production
  • Lisa Fox-Craig - Vice President, Cell Therapy Operations, Orchard Therapeutics, USA
more
  • Case study
  • what production facilities can be used for the production of vectors and viruses?
14:00 - 14:35 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Combination of analytical methods for characterization of viral vectors
  • David Dobnik - Scientific Associate, National Institute of Biology, Slovenia
more
  • New combinations of analytical methods used in development and production of AAV vectors for gene therapy
  • Performance of AAV quantification with molecular methods
  •  Detection and quantification of full and empty viral capsids with electron microscopy
14:00 - 14:35 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Dual Dialogue: T cell and TCR therapies and the treatment of cancer
  • Owain Millington - Head, New Product Development, TC Biopharm, UK
more
  • What is the future for CAR-T and TCR therapies?
  • How scalable are these approaches?
  • What are the commercialisation challenges?
14:00 - 14:35 35 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Please move to another stream
14:35 - 15:10 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
CDMO Expert led panel: How do you choose your development partner?
  • David Morrow - Programme Manager, EATRIS,The Netherlands
  • Lisa Fox-Craig - Vice President, Cell Therapy Operations, Orchard Therapeutics, USA
  • Luca Alberici - Chief Business Officer, MolMed, Italy
  • Reinout Hesselink - Cell Therapy Consultant, Exmoor Pharma Concepts Ltd., The Netherlands
more

Informa is looking for 5 speakers to join this panel discussion looking at the below topics:

  • How do you choose your CDMO
  • What capabilities should they have?
  • Centralised manufacturing
  • Regional manufacturing
  • Local manufacturing
  • Considerations for autologous, allogeneic and gene therapy products
  • Tech transfer of processes to new facilities
14:35 - 15:10 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Challenges in analytical control of a cell-based immunotherapy
  • Jan Feijen - COO, Kiadis Pharma, The Netherlands
more
14:35 - 15:10 35 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Peptide technology to Deliver Proteins and CRISPR Ribonucleoprotein Complexes in Therapeutic Cells
  • Meriem Benchabane - Business Development Director, Feldan Therapeutics, Canada
more
  • To fulfill a need for additional delivery methods for ex vivo cell therapy, Feldan Therapeutics developed a proprietary peptide technology that enables efficient entry of proteins in therapeutic cells.
  • Our carrier peptides combine a cell penetration function with an endosomal leakage activity. They are rationally designed and optimized for their ability to deliver different proteins (e.g. antibodies, transcription factors, CRISPR ribonucleoprotein) in mammalian cells by a simple co-incubation.
  • Exempt from chemical modifications, the peptides are degraded after the delivery, thus decreasing the regulatory burden associated with human cell therapy.
  • Using this peptide-based technology, we delivered SpCas9 or AsCpf1 ribonucleoprotein complexes in adherent cells and in cell suspension cultures, namely human primary T cells, myoblasts and NK cells.
  • With a simple 90-second incubation of cells with a mix of peptides and CRISPR ribonucleoproteins, we reached insertion/deletion rates of 45% in notoriously hard-to-modified NK cells.
  • The flexibility, efficiency and simplicity of our peptide-based technology, the Feldan Shuttle, open new avenues in cell therapy, namely in the production of genetically-modified NK cells for immune therapy.
14:35 - 15:10 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Please move to another stream
15:10 - 15:30 20 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
From a study on autologous brain cell transplantation to clinical trial: GMP cell production
  • Jean-François Brunet - Director, Centre de production cellulaire /DL/CHUV, Switzerland
more

Restoring function of the central nervous system is a challenging task. The large cell replacement experience has offered promising results with different types of cells. Here we propose the adult brain cell auto transplantation as an attractive restoration alternative.

The strategy of autologous reimplantation was investigated in two monkey models:motor cortex lesion as a model of stroke and MPTP-treated monkeys as a parkinsonian model. Primocultures were obtained from cortical biopsy. Cells were grown in vitro as neuralcell ecosystem that consist in astrocytes and neural progenitors. In both models the cells survived and were migrated to the affected structures, respectively to the lesion area and the striatal structures. Significant functional recoveries were thus observed in both models.

Based on such preclinical result, a phase I-II clinical trial is planned for application inpatients affected by stroke.

Prior to that clinical trial, the cell production has to be performed in GMP conditions.For that purpose, and other applications in cell therapy, our public institution, the Lausanne University hospital, implemented a Cell Production Center (CPC) that is now accredited by Swissmedic (authorization n°507482). At the CPC an original concept with a module for production, Isocell Pro 1.8, was developed and qualified. This equipment concept allows to maintain a hermetically culture production in a class A environment by working in class D for operators.

As we all know, the way is long and costing from research to clinical applications. The Lausanne University hospital (CHUV) found a way to help teams of clinicians and researchers to approach that challenge in regenerative medicine

15:10 - 15:30 20 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Ancillary materials for ATMP manufacturing: Quality, regulatory and supply issues
  • Jan Castonguay - Regulatory Affairs Manager, CellGenix, Germany
more

Ancillary materials (AM, also termed raw materials) are crucial for the quality, safety and efficacy of a cell or gene therapy product.

An increasing number of regulatory guidance documents helps to develop risk-mitigation strategies and qualification programs to select appropriate reagents.

The use of fully traceable, GMP manufactured and preferrably animal-derived component-free (ADCF) AM, significantly reduces qualification and validation efforts and helps to ensure consistency, safety and purity of cell & gene therapy medicinal products.

With the first approved cell and gene therapy medicines with significant market volume, the need of high quality AM is increasing strongly. Thus the AM suppliers have to maintain a high quality standard at significantly larger manufacturing scale.

15:10 - 15:25 15 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Clinical Applications of MaxCyte’s Cell Engineering Technology for Gene Editing
  • Jessica Carmen - Director, Business Development, Cellular Therapies, MaxCyte, Inc., USA
more
  • 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
15:25 - 15:40 15 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
TLA Technology & targeted complete NGS sequencing of (trans)genes and gene editing events in cell line development and quality control
  • Max Van Min - CEO, Cergentis, The Netherlands
more
  • TLA technology for targeted complete Next Generation Sequencing
  • TLA-based sequencing of transgenes, integration sites and gene editing events
  • The use of TLA data for clone selection in cell line development
  • TLA to determine the genetic stability and clonality of production cell lines
  • TLA-based sequencing of CAR-T cells & pools
15:30 - 15:45 15 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Development of Large-scale Stem Cell Production System
  • Wen Bo Wang - Senior Vice President, Cell Therapy Research and Development, Cellular Dynamics International, USA
more

In order to meet the growing demand for induced pluripotent stem cell (iPSC)-derived cells for research and therapeutic applications, we have systematically explored the application of traditional and novel cell culture technologies to increase the scale and automation of iPSC expansion, and both adherent and suspension iPSC differentiation. This presentation will summarize our lessons learned from commercializing different iPSC-derived research products and developing candidate therapeutic products.

15:30 - 15:45 15 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Accurate, automated characterization and purity control of gene therapy vectors
  • Nina Forsberg - Marketing Director, Vironova, Sweden
more

In gene therapy manufacturing typical product-related impurities are vectors with deleted or rearranged DNA sequences. Empty particles can have lost their integrity and be prone to form aggregates and furthermore have impact on the immunogenicity profile when administered to patients. Transmission electron microscopy analysis of negatively stained AAV samples (nsTEM) is unmatched in providing detailed information of particle integrity. MiniTEM automatically images and quantifies the portion of broken capsids as well as process related impurities such as host cell debris. The MiniTEM analysis also provides the size distributions of the particles to give accurate data on sample purity.

15:40 - 16:10 30 mins
Main agenda
Afternoon Coffee Break
16:08 - 16:10 2 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Chairperson's Opening Remarks
  • Brian Murphy - Director, Bioprocess Development, Celgene, USA
more
16:10 - 16:45 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Transportation strategies from manufacture to the patient: Elevating bottle necks
  • Carmen Brenner - QC Manager, Bone Therapeutics, Belgium
more
  • Off the shelf ATMPs: from production to patient

  • Cryopreservation of cellular therapeutics: critical quality attributes

  • Storage and delivery on site

  • Case study: allogenic product of Bone Therapeutics


16:10 - 16:45 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Validation expectations for entering the clinic
  • Orly Amiran - Vice President Quality Affairs, Pluristem, Israel
more
  • How do we approach to validation requirements for the various stages of clinical development ( we run trials from phase I to III in our company)
  • Challenges in terms of availability of relevant regulations and regulators expectations (our CMC is approved by US, EU and Japan)
  • Validation readiness of a company in late stages towards marketing.
  • How to plan, manage the samples for testing when batch size is limited
16:10 - 16:45 35 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Designing late stage clinical trials for success
  • Shaun Stapleton - Head of Regulatory Affairs, ReNeuron, UK
more
  • Key design features to consider
  • Special challenges for cell and gene therapies including clinical and manufacturing aspects
  • KOL and Regulatory Agency input into study design
  • Impact of regulatory agency expedited programmes on study design, including impact of 21st Century Cures Act
16:10 - 16:45 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Selection and Acceptance of GMP Raw material for an Allogeneic Universal CAR-T-cell product
  • Sabrina Cogoni - Quality Assurance Manager, Cellectis, France
more
16:45 - 17:20 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Efficient qualification of raw material suppliers
  • Jakub Cierny - Senior Quality Compliance Manager, Sotio AS, Czech Republic
more
  • Case study
  • How do you ensure you are getting the right quality of raw materials?
  • Clinical grade products
  • Supply chain
  • Quality agreements with suppliers
16:45 - 17:20 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Logistics round-tables: Analysing where the gaps lie
  • Lior Raviv - Development Director, Pluristem Therapeutics Inc., Israel
  • Brian Murphy - Director, Bioprocess Development, Celgene, USA
  • Valerie Steenwinckel - Industrialization Director, Celyad, Belgium
  • Martin Lamb - Executive Vice President Sales and Marketing, TrakCel Ltd., UK
more

Informa is looking for 4 round-table leaders to host round-tables on the below topics:

  • Product distribution
  • Injection technologies/ Administration techniques
  • Thawing technologies
  • Global supply chain strategies (including raw materials)
  • Forward thinking and preparations for commercialisation
16:45 - 17:20 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Process development strategies early stage when anticipating GMP
  • Marcos Langtry - Outsourced Manufacturing and Development Director, Tigenix NV, Belgium
more
  • What is the link between early stage process development and GMP production?
  • What do you have to take into consideration in development?
16:45 - 17:20 35 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Please move to another stream
17:20 - 17:21 1 mins
Main agenda
End of Conference