AGENDA 

 

Pre-Conference Workshop Day - Monday 30th March 2020

PRE-CONFERENCE WORKSHOP DAY - Monday 30th March 2019

12:00

Networking Lunch

WORKSHOP A - BIOPROCESS

WORKSHOP B - STANDARDS DEVELOPMENT

WORKSHOP C - RARE DISEASE

WORKSHOP D - CORD BLOOD

Towards CellTherapy4.0 self-adaptive and self-managed manufacturing pipelines

Chair: Dawn Henke, Senior Technical Program Manager, Standards Coordinating Body

Chair: Moshe Israeli, Medical Director, Rabin Medical Center

STANDARDS DEVELOPMENT
13:00

Standards for Advanced Therapies

  • Discussing the current landscape of standards applicable to advanced therapies and standards under development
  • Discussion of needed standards for advanced therapies
  • How to be involved in developing advanced therapies standards and the Standards Coordinating Body
CORD BLOOD
13:00

Keynote: Novel cellular therapies derived from cord blood monocytes

  • Studies in children with selective inborn errors of metabolism have shown that cord blood cells, administered intravenously after myeloablative therapy, and engraft in the brain.
  • DUOC-01, a cord blood derived cellular therapy that promotes myelination, is undergoing testing to augment standard umbilical cord blood treatment in children with leukodystrophies.
  • These observations led us to hypothesize that cord blood cells might also have efficacy treating patients with acquired brain injuries.
BIOPROCESS
13:50

Turn cells into therapies by end-to-end innovation strategies

  • Enable personalized ATMP manufacturing
  • Quality-by-design based ATMP processes development
  • Digital ATMP factory
STANDARDS DEVELOPMENT
13:50

USP Standards for Cell and Gene Therapies

  • Existing applicable USP standards relevant to Cell and Gene Therapies
  • Standards in Development
  • Other needed standards and future development
RARE DISEASE
13:50

The role of patient organisations in precision medicine development

  • A new strategy for research into rare diseases and the roles that patient organisations play
  • The unmet need of precision medicine for scleroderma and other rare orphan conditions
  • Working together with industry to enable development and access to therapies
STANDARDS DEVELOPMENT
14:15

Global Alliance on iPSC Therapies: challenges in developing international consensus standards

  • If cellular therapy products derived from induced pluripotent stem cell (iPSC) lines are to be available across international regulatory jurisdictions then a common set of criteria need to be agreed for defining what constitutes clinical grade lines.
  • Similarly, if different iPSC lines are used for manufacture of a single cellular therapy product, for example in the autologous or HLA-matched allogeneic context, then comparability will need to be demonstrable.
  • GAiT has been working with international partners to develop a consensus set of quality guidelines for clinical grade iPSC lines and standardise the assays which support them.
CORD BLOOD
14:15

CartiCure – An ATMP based on mesenchymal stromal cells for orthopaedic

  • Best tissue for isolation of MSC from human tissue
  • Application of MSC for treatment of cartilage defect in knee
  • Preclinical and clinical trial
14:45

Afternoon Networking Break

WORKSHOP A - BIOPROCESS

WORKSHOP E - GENE EDITING/IMMUNOTHERAPY

WORKSHOP E - GENE EDITING/IMMUNOTHERAPY

WORKSHOP C - RARE DISEASE

WORKSHOP D - CORD BLOOD

Manufacture & Bioprocess of Gene Modified Cell Therapies

Chair: Hansjörg Hauser, Head of Department Gene Regulation and Differentiation, Helmholtz Centre for Infection Research

Chair: Hansjörg Hauser, Head of Department Gene Regulation and Differentiation, Helmholtz Centre for Infection Research

Scientific advice and early dialogue to achieve clinical success and data collection later on

Chair: Moshe Israeli, Medical Director, Rabin Medical Center

RARE DISEASE
15:15

Patient Advocacy Groups and their Evolving & Unique Business Models

  • How the role of patient organizations is changing in the drug development process
  • A case study from the Charcot-Marie-Tooth Association in accelerating drug development for CMT
  • Access to preclinical testing services, key opinion leaders, and the patient community is changing the landscape of drug development in CMT
CORD BLOOD
15:40

Beyond Biostorage: How Cord Blood Banks Can Stay Relevant to Customers

  • How can cord blood banks best serve clients and clinicians as utility (perceived and real) of perinatal stem cells evolves
  • What is the role of personalized medicine and proactive health care in deciding to bank perinatal stem cells
  • What additional meaningful services can banks provide besides biostorage
Jaime Shamonki, Chief Medical Officer, CBR by Generate Life Sciences
17:30

Close of Conference for the day - Opening Networking drinks reception at the Business Design Center

last published: 05/Mar/20 17:05 GMT

Conference Day 1 - Tuesday 31st March 2020

CONFERENCE DAY 1 - TUESDAY 31st March 2020

Opening Keynotes Day 1 – CAR-T & Immunotherapy focus

08:45

Welcome – Jessica Robinson, Project Director, Advanced Therapies 2020, Terrapinn

Prasad Adusumilli
09:00

Keynote presentaton: Autologous Mesothelin-targeting CAR-T to tackle solid tumours in the clinic

  • The presentation will highlight results to date of mesothelin-targeted CAR T-cell clinical trial results
  • Advances in preclinical research of CAR T-cell therapy for solid tumors will be presented
  • Novel strategies for overcoming hurdles to solid tumor CAR T-cell therapy will be discussed
Panel discussion
Bradley Campbell
10:00

Importance of patient focus in developing gene therapy for rare diseases

Start-up Stage - Day 1

Innovation Stage

10:20

Morning Coffee Break

START-UP STAGE
10:20

HAEP – A Novel Format to Foster Innovation through Partnering

  • technology and innovation scouting service
  • sourcing from a vast network
  • successful track record
  • custom-fit search, care-free match, enabling innovation
Martin Pule
11:20

Keynote presentation: War in the Blood - the latest in the front line of CAR-T clinical research

Mark Sawicki
11:40

Effective Integration Strategies Through a Compliance Unified Ecosystem (CUE)

  • Systems Integration continues to be a major issue in the production and distribution of Advanced Therapies
  • Well-defined strategies for the integration of independent informatics platforms will be critical for future standardization and workflow optimization
  • Cryoport’s Compliance Unified Ecosystem (CUE) is driving standard practices for systems integration in the industry
round tables
12:00

Technology and Strategy Roundtables will run from 12:00-12:45 with 15 minutes for a quick summary from each table at the front of the room. These will take place in the conference rooms: look for you

Technology and Strategy Roundtables will run from 12:00-12:45 with 15 minutes for a quick summary from each table at the front of the room. These will take place in the conference rooms: look for your table number to participate.
1. Raw material supply for the commercial manufacturing of cell and gene therapies
Frank Hecht

Frank Hecht, Vice President Marketing And Sales, CellGenix GmbH

10. Exploring the crossover between human and veterinary applications for stem cells
Joanna Miller

Joanna Miller, Chief Scientific Officer, Cell Therapy Sciences

15. Industry-no profit alliances: how to optimize synergies
Elena Beltrami

Elena Beltrami, Business Development Manager, Fondazione Telethon

17. How companies can interact with the regulator and various pitfalls to be aware of
John Johnston

John Johnston, Clinical Assessor, Medicines & Healthcare Products Regulatory Agency

19. Roundtable available
2. Physiological conditions and analytical and monitoring tools for 3D cell cultivations
Cornelia Kasper

Cornelia Kasper, Professor, Biopharmaceutical Production And Technology, BOKU

3. Ensuring safe delivery to patients and fast time-to-market of advanced therapies - How to address challenges of drug administration, fill & finish, packaging & stability?
Diana Lober

Diana Lober, Product Manager, Schott AG

5. Considerations for manufacture scale-up from academia to commercial manufacturing
Johannes Van Der Loo

Johannes Van Der Loo, Clinical Vector Core Director, The Children's Hospital of Philadelphia

7. Reserved for Fujifilm Diosynth Biotechnology
13:00

Lunch

INNOVATION STAGE
13:20

An ODE Model for Optimal Operation of T-Cell Culture in Suspension Systems

  • ODEmodelling approach to control T cells bio process
  • Mechanistic T cell model allows prediction of variability in process outcomes with respect to input variability including cell seeding density, dilution rate and frequency and Glucose concentration
  • Defining first and secondary limiting factors in T cells growth and exhaustion and their effect on the sub-set phenotype
  • Process optimisation and reduce the risk in T cell bioprocessing

IMMUNOTHERAPY

RESEARCH & DEVELOPMENT

STEM CELLS & REGEN MED

CELL THERAPY MANUFACTURE

VIRAL VECTOR MANUFACTURE

PATIENT DELIVERY

PITCH AND PARTNER

INNOVATION

GENE EDITING

EVIDENCE, PRICING AND MARKET ACCESS

GENE MODIFIED CELL THERAPY

GENE THERAPY

Precision medicine for immunotherapy

New clinical data: updates from across the ATMP space

From clinic to commercialisation

Manufacturing strategies

Process development

Chair: Kris Elverum, SVP, Business Development & Strategy, Rubius Therapeutics

Investor Forum & company pitches

Special Session: RNA Targeted Therapeutics

Latest global developments

Chair: Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)

Solid Tumours

New techniques and technologies

IMMUNOTHERAPY
14:20

PD-1 antibodies are transforming cancer therapy both as mono- and in combination

  • PD-1 antibody activity in monotherapy may be enriched with precision medicine tools.
  • Precision medicine may help define resistance biology and enable rational combinations.
  • Certain combinations (e.g. chemotherapy or TKIs + PD-1 antibodies) may be broadly active without regard for biomarker-based selection.
VIRAL VECTOR MANUFACTURE
14:20

Developing robust manufacturing processes for novel gene therapies

INNOVATION
14:20

Translation Control Therapeutics: discovery of small molecule drugs that control mRNA translation into proteins

  • The novel target space of mRNA translation regulation
  • Discovery of small molecule drugs that selectively control mRNA translation
  • Anima Biotech:bi-directional and tissue selective translation controlling drugs
GENE EDITING
14:20

Directed evolution of novel aden-associated viral vectors for clinical gene therapy

  • Adeno-associated viral vectors are increasingly succeeding in the clinic, leading to several regulatory approvals
  • Vectors based on natural versions of AAV suffer from numerous shortcomings, requiring high dosages, invasive routes of administration, and/or limited efficacy for many targets
  • Our directed evolution approach involves high throughput selection of highly optimized AAVs from a library of a billion variants
PRICING & MARKET ACCESS
14:20

Pricing and market access of gene therapies

Mike Fraser, Head of EMEA Region, AveXis
GENE MODIFIED CELL THERAPY
14:20

Exploiting Natural Receptors to broaden the horizon of CAR-T

  • New clinical data for non-gene edited allogenic CAR-T targeting NKG2D in solid tumour applications
  • Update on data for autologous CAR-T for AML
  • SHRNA platform preclinical update and IND status
IMMUNOTHERAPY
14:40

From big patient-level data to actionable insights and answering right business questions in oncology

  • How Patient-Level data can help us to solve business questions from Advanced Therapies
  • Oncology case studies
  • How to optimize the business with data driven insights
STEM CELLS & REGEN MED
14:40

Industrialized personalized regenerative medicine, promising but very challenging

  • ATMPs in personalized regenerative medicine
  • GMP and remote manufacturing as part of GMP-ATMP and one batch-one patient strategy
  • Safety and autologous stem cell products and regulatory hurdles
CELL THERAPY MANUFACTURE
14:40

Exploring Manufacturing platforms and Ancillary Materials to address Cell and Gene Therapy Product needs

  • Exploring Manufacturing platforms and Ancillary Materials to address Cell and Gene Therapy PAiming for commercialization of cell and gene therapy products require a robust process development addressing “manufacturability” and “sustainability”, balancing: critical quality attributes, CoGs, needle-to-needle logistics and scale
  • As a Biotech Manufacturing Organization (BMO) we proactively look on how to address the industry bottlenecks. Here among the different parameters we present the impact of culture media (PRIME-XV T Cell CDM) and manufacturing platforms.
VIRAL VECTOR MANUFACTURE
14:40

Large Scale Production of LV and RV Vectors for T- and CD34+ Cells Transduction

  • Molmed capability for vector production and purification at different scales
  • Challenges for production and purification of LV and RV vectors: from development to GMP
  • Challenges for scaling-up/scaling-out production and purification of LV and RV vectors
IMMUNOTHERAPY
15:00

1st-in-Human CAR T Targets MUC1 Cleavage Product Expressed on Solid Tumors

  • 1st-in-Human Trial for metastatic breast cancer open at the Fred Hutchinson Cancer Research Center.
  • huMNC2-CAR44 recognizes a cancer-specific MUC1 cleavage product called MUC1* (muk 1 star).
  • Antibody huMNC2 recognizes a conformational epitope formed when MUC1 is cleaved to MUC1* by an enzyme that is overexpressed in most solid tumors
PITCH AND PARTNER
15:00

Multi-perspective discussion: How to partner with public/private organizations

  • How to approach big pharma/government organisations for support
  • How can companies correctly identify a fit and interest?
  • Can partnering prevent biotech start-ups from getting stuck before Ph1?
  • Do and don’ts with partnerships
GENE EDITING
15:00

The promise of advanced therapies for 21st Century Medicines

  • Novartis is re-imagining how we treat and care for patients: Innovative platforms for Advanced Therapies
  • Genome editing technologies with a focus on CRISPR/Cas9: Opportunities and challenges of CRISPR as therapeutic modality
  • Genome editing clinical trials: Current status
PRICING & MARKET ACCESS
15:00

Recent developments on Gene Therapy in Germany

  • HTA: Recent developments and processes so far
  • Reimbursement: Contracting models and implementations
  • Data-requirements / registries – an outlook
GENE THERAPY
15:00

Novel approaches to building gene therapy programs: Lessons from BridgeBio

  • Asset identification
  • What to build and what to outsource
  • Putting patients first
CELL THERAPY MANUFACTURE
15:20

Fully closed vs isolator approach in C&GT: how can they collaborate and complement for a successful and sustainable future

  • Are the closed systems really fully closed?
  • In complex processes, how do we manage upstream and downstream?
  • Is there a way to make isolators and closed system collaborating, instead of conflicting?
IMMUNOTHERAPY
15:40

Novel immunotherapy targeting tumor initiating cells

  • 72% 2-year survival, 54% 5-year survival in stage 3 recurrent and stage 4 cancer
  • 96% manufacturing success rate
  • Extremely low cost of goods
CELL THERAPY MANUFACTURE
15:40

Lessons learned from potency assays for beta-thalassemia and sickle cell disease autologous gene therapy drug products

  • Quantitative potency assays were developed to demonstrate correction of b-thalassemia and sickle cell disease properties in an in-vitro cell culture system.
  • Potency was found to be specific to the beta-globin lentiviral vector and dependent on transduction efficiency of the autologous gene therapy drug product, demonstrating ability to reject sub-functional drug products.
  • Considerations for assay development, qualification results, and redundancy to transduction efficiency methods will be discussed
PRICING & MARKET ACCESS
15:40

Getting ready: ARM and access to ATMPs in Europe

  • Access status and expectations for the years to come
  • Challenges and solutions identified by stakeholders
  • RWE infrastructure and new payment models
VIRAL VECTOR MANUFACTURE
16:00

Case study: In-house manufacture vs. outsourcing – why and when?

  • When should you think about in-house manufacture instead of outsourcing?
  • What components do you have to monitor
  • Managing complex supply chain with different suppliers
  • How do you prioritise pushing multiple products
  • When to transition from out to in? if ever?
PATIENT DELIVERY
16:00

Enabling precision healthcare through patient-centric, model-based supply chain optimisation

Maria Papathanasiou, Research Associate, Imperiai College London
16:20

Afternoon Break

CELL THERAPY MANUFACTURE
17:00

The importance of robust process development and characterization early in clinical development of autologous cell therapies

  • Challenges associated with autologous manufacturing and patient variability
  • Defining and establishing critical quality attributes in the context of patient variability
  • Challenges associated with comparability if you go into Phase one with little to know characterization
PITCH AND PARTNER
17:00

Why Specialised Logistics

Timothy Davies, Business Development Manager, A4P Bio
GENE EDITING
17:00

Target optimized variants of CRISPR associated nuclease enables allele-specific gene-editing therapy

  • High precision genome editing is key when developing a genetic cure for congenital diseases.
  • To achieve high precision, optimization of the nuclease per target sequence is a must. Our approach to achieving high precision editing with no off-targets is to employ advanced protein engineering using directed evolution.
  • The approach we present here is novel and broad; it opens up unlimited opportunities for gene editing therapy in cases of dominant negative mutations, compound heterozygous mutations and haploinsufficiency.
PITCH AND PARTNER
17:10

Angicyte, a unique cell therapy for blood vessel regeneration

  • There is an urgent need for new therapies for wound healing.
  • VascVersa’s vision is to create the next generation of living advanced therapies using Angicyte.
  • Angicyte cell therapy creates new blood vessels to improve blood supply and promote healing.
IMMUNOTHERAPY
17:20

Panel discussion: Precision medicine for immunotherapy

  • Using biomarkers and diagnostics to start correlating success rates in immunotherapy: how to know when to apply these
  • Which is more important: stratification of patients using biomarkers or identifying the correct cells for a specific application?
  • Gene editing therapeutics
CELL THERAPY MANUFACTURE
17:20

Panel discussion (potency + assays): Predictive clinical functionality - guaranteeing the success of your cell therapy product

a.TowardsQuantitative-biology mechanistic CQAs to prevent failure of late stage developmentb. From ‘potency assays’ to ‘design spaces’ through ‘CQAs’c.When do we have enough quality and predictive data to assess product potency and effectiveness?d. From ‘black box’ to ‘how much is enough’?e. Realtime evaluation of release criteria?
STEM CELLS & REGEN MED
17:40

In Pursuit of the First Clinical Trial for a Chinese iPSc Derived ATMP (iPSc-Endothelial Progenitor Cell) in the UK

  • Manufacturing protocols and QC standards for iPSc-EPC have been developed in Allife Medicine.
  • Pre-clinical studies have shown positive effects on neuron re-myelination in rats and that iPSc-EPC is able to trigger endothelium lesion recovery in nude mice, with little safety concern in Toxicity and Tumorigenesis.
  • A Phase I/IIα clinical trial to use autologous iPSc-EPC for Ischemic Stroke is planned to start in early 2021.
PITCH AND PARTNER
17:40

The role of AI in development of immunotherapies: opportunities and challenges

  • Biological drug development: a niche waiting to be explored
  • AI in prediction of side effect of immunotherapies
  • Immunotherapy as a tool for precision medicine
INNOVATION
17:40

Using big data applications to transform any cell type

  • Network-based algorithms to find transcription factors that lead to a change is cell state
  • Generating cell conversion protocols
  • Characterise and understand the cell type using microscopic and genetic markers
PITCH AND PARTNER
17:50

Title TBC (PITCH AND PARTNER)

Juergen Weisser, Chief Executive Officer, RheinCell Therapeutics
18:00

Close of Conference for the day - Please join us for an evening drinks reception

last published: 05/Mar/20 17:05 GMT

Conference Day 2 - Wednesday 1st April 2020

CONFERENCE DAY 1 - WEDNESDAY 1st April 2020

Opening Keynotes Day 2 — The latest science in gene therapy and gene editing

Federico Mingozzi
09:00

Gene therapy 2.0: New clinical data, therapeutic applications and next generation technologies

  • Latest updates on LUXTURNA studies to treat blindness, haemophilia and rare disease
  • Tackling the challenges of viral gene therapies i.e. immunogenicity
  • Developing the next generation of gene therapies, what does the future hold?
Steve Kanner
09:20

Next-generation gene editing technology for allogeneic immune cell therapeutics

  • Next-generation CRISPR-Cas9 technology
  • Significantly enhanced editing specificity
  • Editing in immune cells for generation of functionally tuned therapeutics
09:40

Repairing the human genome with designed zinc finger editing reagents

  • Overview of Sangamo’s zinc finger protein gene editing technology, including new innovations that facilitate specificity, precision, and gene editing efficiency
  • Will address specific examples of defined therapeutic applications including the generation of reagents suitable for use in tauopathies, Huntington’s disease, and lysosomal storage diseases
Frederic Chereau
10:00

In vivo gene editing paediatric patients with rare diseases

Start-up Stage - Day 2

10:20

Networking Refreshments

INNOVATION STAGE
11:00

The efficacy of Micro-fragmented adipose tissue in the treatment of Knee Osteoarthritis: A 1-Year Follow-up Study

IMMUNOTHERAPY

RESEARCH & DEVELOPMENT

STEM CELLS & REGEN MED

CELL THERAPY MANUFACTURE

VIRAL VECTOR MANUFACTURE

PATIENT DELIVERY

PITCH AND PARTNER

INNOVATION

EVIDENCE, PRICING & ACCESS

GENE MODIFIED CELL THERAPY

GENE THERAPY

Immuno-oncology

Clinical developments and strategies for Immuno-oncology

Chair: Stephen Badylak, Professor, Department of Surgery, Director, McGowan Center for Pre-Clinical Studies, Deputy Director, McGowan Institute for Regenerative Medicine (MIRM)

Control Strategies

Chair: Manuel Carrondo, Professor of Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET

Exosome Therapeutics

Approval and evidence

Off the shelf: Moving towards allogeneic models

Clinical focus

STEM CELLS & REGEN MED
11:20

Challenges and Troubleshooting of starting auto-islet cell transplant program

  • Institutional, Financial and administrative challenges
  • Clinical challenges with different providers and patients
  • Different options and broad cost analysis of putting together facility and islet isolation team
CELL THERAPY MANUFACTURE
11:20

Adjusting control strategies using Digital Twins

  • Which Quality Attributes of the raw materials do we need to monitor to demonstrate no mix up during parallel processing?
  • Adaptive Digital Twin strategies for varying raw materials as inherent in personalized medicine
  • Integrated Digital Twins for full process chain analysis
VIRAL VECTOR MANUFACTURE
11:20

Plasmid DNA Control Strategy & AAV Production

  • Specifications, Comparability, Potency assays, Stability, etc.
  • Method & Process Validation
  • Safety Risk vs Business Risk
  • Current Regulatory Guidance
GENE EDITING
11:20

CRISPR/Cas9 Gene Editing Therapies for Cystic Fibrosis

  • Cystic fibrosis is caused by more than 200 different mutations in the CF gene, many of which are likely to remain untreatable by small molecule drugs.
  • Gene editing by CRISPR/Cas9 offers many different approaches to the correction of CF gene mutations which could lead to new therapies
  • The big challenge for therapeutic gene editing in CF is delivery of the therapy to the lung, the main affected organ.
  • Nanoparticle delivery and cell therapy methods offer alternative delivery strategies
GENE MODIFIED CELL THERAPY
11:20

Creation of off-the-shelf TCR-less CAR T cell therapies

Dr Bob Valamehr, Chief Development Officer, Fate Therapeutics
PITCH AND PARTNER
11:30

Bringing continuous production to cell manufacturing

  • Batch VS continous cell production
  • Overcoming scaling issues
  • Serum free production
Leo Groenewegen, Chief Executive Officer, CellulaRevolution
INNOVATION
11:40

An Exosome-based Drug Delivery Platform Derived from a Human Neural Stem Cell (hNSC) Line

  • To ensure the scale required for clinical research and commercialisation producer cell immortalisation and clonal isolation is a practical strategy to produce consistent, functionally bioactive exosomes for use as therapeutic agents.
  • The cell line can be rapidly modified to alter exosome cargo or reprogrammed to change its phenotype while maintaining its conditional immortalisation.
  • hNSC derived exosomes demonstrate favourable distribution across the blood brain barrier.
GENE MODIFIED CELL THERAPY
11:40

Automating and scaling manufacture of “off-the-shelf” engineered allogeneic CAR-T therapies

  • The presentation will highlight process steps that are a challenge to automate and scale.
  • Recent advances in developing our manufacturing process platform will be presented.
  • Analytical strategies to gain understanding and control of manufacturing process and product quality will be discussed.
IMMUNOTHERAPY
12:00

Small activating RNAs as a novel approach for immunotherapy

  • Small activating RNAs can be designed to a wide range of targets to activate gene transcription
  • MiNA Therapeutics’lead saRNA to CEBPA has demonstrated proofof concept in the clinic
  • Novel saRNAs have been designed to specifically up-regulated immune targets for cancer immunotherapy
VIRAL VECTOR MANUFACTURE
12:00

Challenges and concepts of designing a new GMP manufacturing facility at an academic centre

  • GMP manufacturing at the Children’s Hospital of Philadelphia
  • Facility design challenges, concepts, and considerations
  • Process of Safety Risk Assessment and FDA interaction
  • Challenges associated with the growing field of gene therapy
APPROVAL & EVIDENCE
12:00

Yescarta’s journey and beyond

  • Kite is providing CAR-T therapy to lymphoma patients in US, EU and other markets through the commercial product, Yescarta
  • Yescarta reached the market through the FDA’s and EMA’s expedited development programmes (Breakthrough Therapy and PRIME)
  • Kite is expanding global access to Yescarta and developing next generation products for lymphoma and solid tumours
STEM CELLS & REGEN MED
12:20

Clinical translation in regenerative medicine

  • Define the Patient and its relevant Preclinical Development Path
  • Characterization and Production of Cell based products for Regenerative Medicine
  • Reimbursement and cost-effectiveness, willingness to pay
CELL THERAPY MANUFACTURE
12:20

Bioinspired Manufacturing of hPSC-based Therapy Products

  • Development of novel cell culturing strategies that recreate environmental conditions to excel hPSC proliferation and differentiation/maturation into functional cardiomyocytes and hepatocytes;
  • Advancing manufacture of cell therapy products through metabolic and process understanding
  • Multi-parametric techniques including advanced “-omics” technologies (proteomics, transcriptomics, metabolomics and fluxomics) as complementary analytical tools to support bioprocess understanding and optimization as well as to unveil the mechanism of action of cell therapy products
STEM CELLS & REGEN MED
12:40

Discovery of metabolic critical quality attributes for pluripotent stem cell-derived cardiomyocytes

  • Defined scalable processes for manufacturing cardiomyocytes from human pluripotent stem cells have been developed, but monitoring cell quality during manufacturing is challenging.
  • Profiling intracellular metabolites during cardiomyocyte differentiation and maturation has identified quality attributes related to cardiomyocyte state.
  • Monitoring medium composition during cardiomyocyte differentiation can predict successful and failed batches.
VIRAL VECTOR MANUFACTURE
12:40

Assay development for oncolytics

  • Early data
  • Trials and tribulations
  • How to transform data analysis
PITCH AND PARTNER
12:40

EVerZom: a large scale bioproduction platform of extracellular vesicles

  • Game changing technology to produce EV at large scale
  • Caracterisation of EVs produced with this new technology
  • Applications in fistula and cardiac repair
GENE MODIFIED CELL THERAPY
12:40

Genetic engineering of drug-resistant gamma delta T cells for eradication of residual glioblastoma

  • Haploidentical gamma delta post-transplant immune “boost” and strategy for off the shelf therapy in glioblastoma trial
  • Drug Resistant Immunotherapy (DRI) - gamma delta T cells in combination with chemotherapy – allows superior targeting of residual glioblastoma.
  • Accrual and logistical information from ongoing trials.
GENE THERAPY
12:40

A virtual model for gene therapy R&D to target the complement system

  • Introduction to Apellis: History, APL-2 and gene therapy pipeline expansion.
  • Components and operation of a virtual gene therapy network.
  • Ongoing gene therapy R&D at Apellis
PITCH AND PARTNER
12:50

Ultroxa® Polymer platform for advanced drug and gene delivery

  • Poly(2-oxazoline)s (PAOx, POZ) as versatile polymer platform for sustained and targeted drug delivery
  • Ultroxa® technology and unique ability to design and produce ultra-defined polymers for pharma and biotech
  • Ultroxa® Polyethyleneimines (PEI) and new superior polymer vectors for transfection
Victor R De La Rosa, Chief Scientific Officer, Avroxa bvba - Ultroxa Polymers
13:00

Neworking Lunch

INNOVATION STAGE
13:00

Artificial Membrane Binding Proteins for Next-Generation Cell Therapies

  • A new non-GM methodology for re-engineering the membranes of therapeutic cells
  • Cell augmentation provides hypoxia resistance, tissue homing and adhesion, and extracellular matrix formation
  • Platform technology is facile and can be used to rapidly display any protein on any cell or extracellular vesicle

RESEARCH & DEVELOPMENT

STEM CELLS & REGEN MED

CELL THERAPY MANUFACTURE

VIRAL VECTOR MANUFACTURE

PITCH & PARTNER

INNOVATION

EVIDENCE, PRICING & ACCESS

GENE MODIFIED CELL THERAPY

GENE THERAPY

Clinical & preclinical updates: Cell therapy

Chair: Manuel Carrondo, Professor of Chemical & Biochemical Engineering, FCT-UNL, Vice-President, IBET

Business models and commercialisation

Gamma Delta T-Cells

CELL THERAPY MANUFACTURE
14:00

Building your own closed manufacturing system

  • Are CMOs really needed?
  • What happens to IP when working with CMOs?
  • Is in-house GMP production really an option?
VIRAL VECTOR MANUFACTURE
14:00

Phase-Appropriate Potency Assays for AAV Gene Therapy Products

Aisleen Mccoll-Carboni, Senior Scientist, Sanofi
INNOVATION
14:00

Developing the First External Quality Assurance (EQA) Program for ATMPs

  • Design and development of the first External Quality Assurance (EQA) programme for ATMPs. Adapting the acquired experience from other fields of knowledge.
  • Promoting the harmonization of relevant Quality Control methods to guide manufacturers to develop safe and effective ATMPs.
  • State of development. Joining the programme and benefits to participants.
APPROVAL & EVIDENCE
14:00

Evaluating pharma partnerships: how & why

  • Blend of partnerships
  • Financing exits and acquisitions
  • Examples from BlueRock
GENE MODIFIED CELL THERAPY
14:00

Gamma Delta T cells engineered with targeting moieties (CARs and TCRs) directed to cell surface and intracellular antigens

  • Development of engineered off-the-shelf allogeneic γδ T cell products
  • Preclinical efficacy and safety of chimeric antigen receptor-modified γδ T cell products
  • Large-scale cGMP-compliant manufacture of γδ T cell products
CELL THERAPY MANUFACTURE
14:20

The role of metabolism in cellular therapeutics biomanufacturing: can metabolomics be used for quality assurance & control?

  • The application of metabolomics at all manufacturing stages (input, bioprocess, output) for the monitoring quality control and optimization of cellular therapies for clinical application.
  • Metabolomics can accurately and sensitively capture alterations in the cellular physiological state such as the loss of pluripotency of embryonic (ESCs) and induced pluripotent stem cells (iPSCs) prior to the detection by other techniques
  • The application of metabolomics for the selection of optimal differentiation protocols and for the design of bioactive materials that can enhance osteogenic differentiation of mesenchymal stem cells (MSCs)
  • We have shown that metabolomics can reveal differences in the immunosuppressive properties of MSCs
VIRAL VECTOR MANUFACTURE
14:20

Industrialization of gene therapy manufacturing

Overcoming manufacturing challenges faced by therapy developers- Effectively preparing for later stage trials and commercialization
PITCH AND PARTNER
14:20

Accelerating drug development by immune monitoring

Our vision is to decrease the risk of drug development failure by:
  • Maximizing the mechanistic information obtained from all phases of drug development
  • Identifying customer needs and tailoring the solutions delivered accordingly
  • Giving access to frontline expertise as part of our service
Thomas Mørch Frøsig, Chief Executive Officer, ImmuMap
GENE THERAPY
14:20

Developing Liver directed gene therapy for paediatric indications

  • Acute unmet need in paediatric liver based disorders
  • New approaches are needed to provide long-term efficacy
  • Challenges in translation of laboratory discoveries into clinical practice
PITCH AND PARTNER
14:30

Innovative plant-based solution for chronic wound care

  • EvoBiotech discovered an innovative plant-derived active ingredient which promote regeneration of skin and blood vessel.
  • The active ingredient can be incorporated into an advanced dressing used for chronic wounds, improving its therapeutic efficacy.
  • This product can fill the urgent need for new medications that can cure ulcers faster and with an accessible price compared to products currently on the market.
Chiara Gai, Chair, EvoBiotech
GENE MODIFIED CELL THERAPY
14:40

Engineering NK Cell Resistance to the Tumor Microenvironment

  • Role of TME-mediated immune suppression in tumor progression and its negative effects on NK cell function
  • Engineering NK cell resistance to TGF-beta
  • In vitro and in vivo function of gene modified NK cells
PITCH AND PARTNER
14:50

Reserved for OpenCell.bio

Thomas Meany, Director, OpenCell.bio
APPROVAL & EVIDENCE
15:00

Towards affordable and sustainable ATMP costs

a. Why do these therapies cost so much?b. Costs for continuous manufacturing?c.Patient vs profit margind. Sustainability and willingness to paye. Regulatory perspectives
GENE MODIFIED CELL THERAPY
15:20

World’s first off-the-shelf NK cellular immunotherapy making cancer a chronic disease

  • How and why off-the-shelf?
  • Why are NK-cells important in cancer treatments?
  • Cancer management a viable option?
APPROVAL & EVIDENCE
15:40

Orphan Drugs Gene and Cell therapies product: The difficulties encountered and the supports proposed by the EC, EMA and National Agencies for the development and registration.

The development of these products is worldwide or at least European, we will highlight some of the problems met regarding missing of harmonization or the design of clinical trials. The regulators have developed some supports and expedited programs topromote the development of these orphan drugs. We will describe this during our presentation.
GENE MODIFIED CELL THERAPY
15:40

Different approaches to allogeneic therapies

  • Allogeneic CAR approaches are being developed based on gene-edited T-cells, or different cell types, such as NK, NKT and gamma delta T-cells. Several of those are now in the clinic.
  • There are different technologies being used to generate these products, including for engineering, manufacturing and cell source.
  • This talk will offer a broad overview of the various efforts in the space and their key features.
GENE THERAPY
15:40

LUMEVOQ Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Subjects

  • Set the scene: LHON is a rare disease affecting young males
  • Main clinical results
  • Indirect comparison with Natural History data
  • Regulatory Pathways
16:00

Networking refreshments

STEM CELLS & REGEN MED

CELL THERAPY & VIRAL VECTOR MANUFACTURE

CELL THERAPY & VIRAL VECTOR MANUFACTURE

EVIDENCE, PRICING & ACCESS

Chair: Ioannis Papantoniou, Principal Investigator, FORTH (Foundation of Research and Technology); Adjunct Professor, KU Leuven

Chair: Ioannis Papantoniou, Principal Investigator, FORTH (Foundation of Research and Technology); Adjunct Professor, KU Leuven

Chair: Raghuram Selvaraju, Managing Director, H.C. Wainwright

STEM CELLS & REGEN MED
16:30

The influence of immunomodulatory biomaterials upon stem cell fate

  • The primary determinant of stem cell fate
  • Dynamic reciprocity
  • Endogenous vs. exogenous stem cell therapy
CELL THERAPY MANUFACTURE
16:30

Panel discussion: moving towards ‘smart’ and ‘flexible’ integrated process pipelines

a. What are the common bioprocess units suitable for all ATMP manufacture – technology and scale perspectives? b..…and what are the specialisations when it comes to different therapy types? i.Technologiesii.Tools iii. Data c. Inherently variable field eliminating sources of systemic uncertainty towards process streamliningd. Whole-process intensification in cell therapy manufacturing: room to improve
VIRAL VECTOR MANUFACTURE
16:30

Panel discussion: moving towards ‘smart’ and ‘flexible’ integrated process pipelines

a. What are the common bioprocess units suitable for all ATMP manufacture – technology and scale perspectives? b..…and what are the specialisations when it comes to different therapy types? i.Technologiesii.Tools iii. Data c. Inherently variable field eliminating sources of systemic uncertainty towards process streamliningd. Whole-process intensification in cell therapy manufacturing: room to improve
GENE THERAPY
16:30

Maximising value from the expression cassette in gene therapy product design

  • Expression cassette design attributes
  • Synthetic promoter design and validation
  • Regulatable gene expression
STEM CELLS & REGEN MED
16:50

Development of Combinatorial Culture Technology for cell and gene therapy applications

  • Combinatorial screening of diverse variables in process development for optimisation of cell and gene therapies.
  • Expansion of hematopoietic stem cells for allogeneic and autologous transplantation.
  • Enhancement of viral transduction efficiency for autologous gene therapy to treat rare diseases.
18:00

Close of conference

last published: 05/Mar/20 17:05 GMT