AGENDA 

 

Pre-Conference Workshop Day - Monday 30th March 2020

PRE-CONFERENCE WORKSHOP DAY - Monday 30th March 2019

12:00

Networking Lunch

LOGISTICS/DELIVERY TO PATIENT
13:00

Tech transfer forum: tools, tactics, strategies and approaches

As the cell therapy manufacturing sector matures, novel directions regarding its outlook in the coming decade should be explored.Automated manufacturing process units are increasingly being adopted by product developers and/or CMOs allowing for the gradual build-up of real-time data sets. This will gradually result in whole-bioprocesses that could eventually operate in a semi-autonomous and self-adaptive manner. Given the, flexibility, agility of future manufacturing schemes in low footprint equipment and potentially personalised processes, the question of whether we reach a point where real-time release can become a reality remains to be answered.
  • Flirting with the control aspects, machine learning and AI tools and linking these to the actual process
  • From mechanistic exploration to data-driven implementation
  • High throughput Analytics for Cell Therapy Process Optimization
  • Producing high volumes of high-quality data for ‘era of 4.0 industry’ and ‘smart bioprocessing’ without good data, which we currently don’t have a lot of.
  • Data acquisition and structuring: useful while building clinical portfolio
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.
PRICING & MARKET ACCESS
13:00

Title TBC (Pricing and Market Access)

BIOPROCESS
13:20

Title TBC (BIOPROCESS)

PRICING & MARKET ACCESS
13:20

Title TBC (Pricing and Market Access)

BIOPROCESS
13:40

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
14:00

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
14:00

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
CORD BLOOD
14:00

CB Expansion breakthrough

BIOPROCESS
14:20

Title TBC (BIOPROCESS)

CORD BLOOD
14:20

TBC Driving public banked cord blood inventory utilization through research and development

BIOPROCESS
14:40

Title TBC (BIOPROCESS)

RARE DISEASE
14:40

Title TBC (RARE DISEASE)

CORD BLOOD
14:40

Title TBC (CORD BLOOD)

15:00

Afternoon Networking Break

CORD BLOOD
15:50

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
17:30

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

last published: 21/Jan/20 11:25 GMT

Conference Day 1 - Tuesday 31st March 2020

CONFERENCE DAY 1 - TUESDAY 31st March 2020

Opening Keynotes Day 1 – CAR-T & Immunotherapy focus

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
09:20

Realizing the promise of ATMPs for patients around the world

  • Working with regulators to navigate scale up challenges in manufacturing
  • Strategies in supply chain and patient delivery
  • Tackling post manufacturing commercialization challenges such as pricing and finding highly skilled experts
  • Durability
10:20

Morning Coffee Break

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

11. Cord blood as a source of new cells
12. Tissue engineering for organ regeneration
Vaidehi Joshi

Vaidehi Joshi, Scientist II, Therapeutics, Organovo

13. TBC
14. Making allogeneic cell therapy a reality
Devyn Smith

Devyn Smith, Chief Operating Officer And Head Of Strategy, Sigilon

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

18. Ensuring ATMP access to patients
Delphine Roulland

Delphine Roulland, Director Government Affairs And Public Policy, European Confederation of Pharmaceutical Entrepreneurs (Belgium)

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

Cornelia Kasper, Professor, Biopharmaceutical Production And Technology, B.O.K.U.

21. Neurological applications for ATMPs
22. Development of regenerative medicines for cardiovascular indications
David Mazzo

David Mazzo, Chief Executive Officer, Caladrius Biosciences

23. Renal applications for ATMPs
24. Tackling diabetes with ATMPs
3. TBC (Reserved for Schott)
Sebastian Kress

Sebastian Kress, Post Doc, BOKU

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

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

Joanna Miller, Chief Scientific Officer, Cell Therapy Sciences

13:00

Lunch

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

Palani Palaniappan, Head Of Technical Operations And Andover Site, Sarepta Therapeutics
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
GENE MODIFIED CELL THERAPY
14:20

Broadening CAR-T’s horizons with NKG2G

  • 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
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
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
PRICING & MARKET ACCESS
14:40

Improving market access for USA, UK and Europe

GENE MODIFIED CELL THERAPY
14:40

Macrophages to tackle solid tumors: innate and adaptive immunity

IMMUNOTHERAPY
15:00

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
RESEARCH & DEVELOPMENT
15:00

TBC (RESEARCH & DEVELOPMENT)

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
IMMUNOTHERAPY
15:20

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
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?
GENE MODIFIED CELL THERAPY
15:20

TBC Trilogy cells: a new approach to tackling solid tumours

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

What incentives are available for developing orphan drugs? Is the current orphan drug legislation & incentives enough?

Delphine Roulland, Director Government Affairs And Public Policy, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
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?
GENE EDITING
16:00

Development of therapeutic genome editing strategies

Seokjoong Kim, Executive Director Of R&D And Strategic Alliances, ToolGen
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
PATIENT DELIVERY
17:00

Traceability in the clinic: best practices, new opportunities

PITCH AND PARTNER
17:00

Why Specialised Logistics

Timothy Davies, Business Development Manager, A4P Bio
INNOVATION
17:00

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
PRICING & MARKET ACCESS
17:00

Panel discussion: Finding the right framework for reimbursement and market access for cell and gene therapies

  • Updates on current EU pricing and contracts for Kymriah, Yescarta and Luxturna
  • Structured reimbursement system for hospital-based treatments in Europe: how is this working so far in 2020?
  • How should be looking at transformative therapy pricing in the next 5 years?
  • What is possible for statutory health insurance framework in the EU?
GENE THERAPY
17:00

AAV gene therapy

PITCH AND PARTNER
17:10

Providing a novel off-the-shelf implant to repair damaged organs

  • Regenerating damaged organs with an engineered off-the-shelf biomimetic medical device implant
  • Regenerating a damaged esophagus with a bioresorbable medical device implant to treat esophageal cancer
  • Utilizing bioresorbable engineered polymers and stem cell derived factors to develop the above mentioned therapeutic medical device implant
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?
GENE THERAPY
17:20

Panel discussion: Immunogenicity for gene therapy

Key unresolved clinical issues in gene therapy: immunosuppression, re-dosing, and in-utero dosing
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

Taking a novel next-generation biologic from idea to clinical proof of concept

  • On-site delivery of human proteins to body surfaces by lactic acid bacteria
  • Clinical trials skin wounds-ILP100
  • Preclinical MOA IBD-ILP100
18:00

Close of Conference for the day - Please join us for an evening drinks reception at the iconic Emirates Stadium

last published: 21/Jan/20 11:25 GMT

Conference Day 2 - Wednesday 1st April 2020

08:55

Opening Remarks

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
Adrian Woolfson
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

10:20

Networking Refreshments

IMMUNOTHERAPY
11:20

Immuno-oncology for breast cancer

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
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.
PATIENT DELIVERY
12:00

Delivering allogenic cell therapy to patients in a clinical setting in USA and across Europe

  • How to process allogenic samples
  • Guaranteeing sound cold chain control globally: learnings from phase 2 & 3 trials
  • Acute vs. Chronic indications and the difference of care
  • Addressing difficulties and time constraints when delivering therapies to acute patients in the clinic
GENE MODIFIED CELL THERAPY
12:00

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.
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
VIRAL VECTOR MANUFACTURE
12:20

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
INNOVATION
12:20

Exosome derived from peripheral blood plasma of chronic pancreatitis patients and healthy volunteers and explore therapeutic potential in auto-islet transplant

  • Produce and characterize serum derived exosome products from CP and healthy subjects and to establish the feasibility of cGMP-compliant production.
  • Evaluate if CP patient derived exosome has impaired anti-inflammatory response by comparing the exosome molecular profile and potency with healthy individuals.
  • Evaluate in mouse model therapeutic potential
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.
13:00

Neworking Lunch

IMMUNOTHERAPY
14:20

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
STEM CELLS & REGEN MED
14:20

Measurement of potency in cellular therapies – correlation with in vivo outcomes

  • Technology to assess in vitro phenotype and likely in vivo performance
  • In vivo performance in models of combined tissue and cell products
  • Initial Results from first in human use of new regenerative cell therapies
John Campbell, Associate Director, Tissues, Cells And Advanced Therapeutics, Scottish National Blood Transfusion Service
CELL THERAPY MANUFACTURE
14:20

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
14:20

Assay development for oncolytics

  • Early data
  • Trials and tribulations
  • How to transform data analysis
APPROVAL & EVIDENCE
14:20

Evaluating pharma partnerships: how & why

  • Blend of partnerships
  • Financing exits and acquisitions
  • Examples from BlueRock
GENE THERAPY
14:20

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
IMMUNOTHERAPY
14:40

Gene modified Cell Therapy: Gene modified T-Cells for solid tumours

CELL THERAPY MANUFACTURE
14:40

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
INNOVATION
14:40

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.
GENE EDITING
14:40

Analytical development in gene editing

  • Current trends and challenges for analytical development in gene editing
  • Delivery vehicle characterization: viral and non-viral
  • Characterization of cargo: new modalities
Bo Yan, Senior Scientist, Analytical Development, Beam Therapeutics
GENE MODIFIED CELL THERAPY
14:40

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.
STEM CELLS & REGEN MED
15:00

Commercialising the first cell therapy in Europe, lessons learned

CELL THERAPY MANUFACTURE
15:20

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
APPROVAL & EVIDENCE
15:20

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

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
16:20

Networking refreshments

RESEARCH & DEVELOPMENT
17:00

CAR T-cell immunotherapy of solid tumours: parallel learning from the clinic and lab

  • An update will be provided on a Phase I clinical trial of panErbB CAR T cells in refractory head and neck cancer
  • A new generation CAR technology known as parallel (p)CAR will be presented
  • Examples of efficacious pCARs will be presented across a spectrum of indications, including avb6 integrin, MUC1 and panErbB
INNOVATION
17:00

Stem cells secretome and their efficiency in the treatment of soft tissue injury and skin rejuvenation

GENE MODIFIED CELL THERAPY
17:00

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
STEM CELLS & REGEN MED
17:20

Stem Cells and their microenvironment: Seed vs. Soil

  • The primary determinant of stem cell fate
  • Dynamic reciprocity
  • Endogenous vs. exogenous stem cell therapy
INNOVATION
17:20

Cell manufacturing for cell therapy and regenerative medicine and cell expansion for iPSCs

GENE MODIFIED CELL THERAPY
17:20

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.
RESEARCH & DEVELOPMENT
17:40

Therapeutic potentials of activated islet progenitor cells for the treatment of diabetes

CELL THERAPY MANUFACTURE
17:40

Industrial automation of cell therapy manufacturing

GENE EDITING
17:40

mRNA characterization

GENE MODIFIED CELL THERAPY
17:40

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?
RESEARCH & DEVELOPMENT
18:00

In vivo models, safety and preclinical development of engineered exosome therapeutics for NPC1 and Lysosomal Storage Disease

CELL THERAPY MANUFACTURE
18:00

Panel discussion: Building your own manufacturing facility Vs. outsourcing the process to partners

GENE EDITING
18:00

Closing panel discussion

Bo Yan, Senior Scientist, Analytical Development, Beam Therapeutics
last published: 21/Jan/20 11:25 GMT