Full Agenda

 

London, 15 - 17 May 2019

Schedule

Create your personal agenda –check the favourite icon

May 1509:00
Conference pass

Welcome remarks

Process Considerations for Cryogenic and Regenerative Medicine Commercialization
May 1509:00
Conference pass

ATMP Therapy Bioprocessing and Manufacture Session

CAR-T manufacture and imaging
Dr Qasim Rafiq, Associate Professor, Bioprocessing Of Cell And Gene Therapies, University College London
May 1509:00
Conference pass

Focus group: To advance process, measurement, and analytical techniques of regenerative medicine products and services

Standards development process/clinical trials
Moderator: Sven Kili, Vice President And Head Of Gene Therapy Development, Sven Kili Consulting
Richard Mcfarland, Chief Regulatory Officer, Advanced Regenerative Manufacturing Institute
Kevin Carrick, Director, Science, Standards And Global Biologics, United States Pharmacopeia
May 1509:05
Conference pass

Lentiviral Vector Production for Advanced Therapies

CAR-T manufacture and imaging
May 1509:20
Conference pass

Panel Discussion: Integration and data continuity throughout the regenerative medicine production process

Process Considerations for Cryogenic and Regenerative Medicine Commercialization
Amy Hines, Senior Manager, Collection Network Management, Be The Match BioTherapies
Alex Nancekievill, Chief Business Officer, GE Healthcare
May 1509:25
Conference pass

Manufacturing Considerations for Cell and Gene Therapy

CAR-T manufacture and imaging
May 1509:45
Conference pass

Process Development and Manufacture of CAR-T cells in an automated, stirred-tank bioreactor

CAR-T manufacture and imaging
May 1511:00
Conference pass

Panel discussion: Systems compliance and traceability during product distribution

Process Considerations for Cryogenic and Regenerative Medicine Commercialization
May 1511:00
Conference pass
May 1511:00
Conference pass

Focus group (continued): To advance process, measurement, and analytical techniques of regenerative medicine products and services

Standards development process/clinical trials
Moderator: Sven Kili, Vice President And Head Of Gene Therapy Development, Sven Kili Consulting
Kevin Carrick, Director, Science, Standards And Global Biologics, United States Pharmacopeia
Richard Mcfarland, Chief Regulatory Officer, Advanced Regenerative Manufacturing Institute
May 1511:25
Conference pass

AI and Image analysis in pathology

AI in Healthcare Summit
May 1511:40
Conference pass

Automated Clinical Coding

AI in Healthcare Summit
Goran Nenadic, Group Leader, The Farr Institute’s Health eResearch Centre
May 1513:00
Conference pass

In vivo genome and base editing treatments of a human PCSK9 knock-in hypercholesterolemic mouse model

Gene Editing Summit
· Generation of humanized hypercholesterolemic in vivo model · Understanding off-target effect of genome and base editing in vivo
Pinar Akcakaya, Senior Research Scientist, AstraZeneca
May 1513:00
Conference pass

Panel discussion: Regenerative medicine globalization; considerations for effective launch activity beyond your initial geographic launch

Process Considerations for Cryogenic and Regenerative Medicine Commercialization
Mark Flower, Vice President, Business Development and Strategic Partnerships, Be The Match BioTherapies
Alain Vertes, Vice President, Alliance Management, Mesoblast Inc
May 1513:00
Conference pass

Advanced Therapy – Future Leader Perspectives

CAR-T manufacture and imaging
Moderator: Dr Qasim Rafiq, Associate Professor, Bioprocessing Of Cell And Gene Therapies, University College London
May 1513:00
Conference pass

Rhian Carter, Oxford BioMedica

CAR-T manufacture and imaging
May 1513:00
Conference pass

Panel: AI at the Bedside: Trust me I’m a machine

AI in Healthcare Summit
  • Developing systems synergistically to a whole health ecosystem
  • How AI can be of assistance in the ICU, Nursing Facilities, Out Patients, Home Patients etc…
  • Understanding what the pinpoints are in a clinical system and how they can be alleviated with AI
May 1513:15
Conference pass

Manufacturing personalised living implants - a quest for predictive functionality

CAR-T manufacture and imaging
May 1513:20
Conference pass

A true understanding of the biology of stem cells

Cord Blood Workshops
Dr Irene Martini, Lecturer, University La Sapienza
May 1513:25
Conference pass

CRISPR-mediated genome engineering of CHO cell lines for production of therapeutic proteins

Gene Editing Summit
· Discuss the latest development in genome editing tools for CHO cell line development· Recognize the impact that CRISPR-mediated genome editing has on accelerating the development of CHO cell lines· Understand how the combination of genome editing and systems biology can be applied to improve the production of therapeutic proteins
May 1513:30
Conference pass

Process Development for CAR T cell manufacturing

CAR-T manufacture and imaging
Emma Chan, Senior Scientist, Autolus
May 1513:45
Conference pass

Panel Discussion: Future Needs of the Advanced Therapy Industry

CAR-T manufacture and imaging
May 1513:50
Conference pass

Precise genome engineering to empower drug discovery

Gene Editing Summit
May 1515:00
Conference pass

Panel: Gene editing and synthetic biology in pharmaceutical development: Potentials and hurdles

Gene Editing Summit
· Moving from autologous to allogenic function · Where are we really headed?
May 1515:00
Conference pass

Scalability (Series of 30 min panel discussion sessions)

Process Considerations for Cryogenic and Regenerative Medicine Commercialization
May 1515:00
Conference pass

Using imaging to improve the success rate of cell therapies on solid tumors

CAR-T manufacture and imaging
May 1515:00
Conference pass

Clinical trial focus workshop: What needs to be done to get these experimental treatments to patients in the clinic?

Standards development process/clinical trials
· Centralized and decentralized models· Sourcing starting material: apheresis, tumor, bone marrow· From manufacture to back into local environment· Design of clinical trial
May 1515:00
Conference pass

Cord blood bank sustainability

Cord Blood Workshops
  • Provoking greater use of cord with high quality impact
  • Understanding the hurdles in selecting cord in the clinical transplant setting
  • Diversification of products and services
  • Creating a sustainable collection model – strategies that have bene adopted
Irina Evseeva, Head Of Specialist Services, Anthony Nolan
Daniel Gibson, Head Of Innovation And Commercialation, Anthony Nolan
Chris Leonforte, Processing And Qc Manager, Anthony Nolan
May 1516:30
Conference pass

Operational, strategic and economic impact of FDA licensure for HPC, cord blood

Cord Blood Workshops
  • Operational impact of FDA Licensure experience that facilitated process improvement, facility design and manufacturing productivity.
  • FDA license economic impact on cost per cord blood unit, price per unit, capital and re-curing expenses
  • Strategically leveraging FDA license to enter new markets and optimize manufacturing

Create your personal agenda –check the favourite icon

May 1608:50
Conference pass

Extending cord blood to regenerative therapies for the brain

Cord Blood
  • The history and evolution of cord blood banking and transplantation
  • Quality measures of cord blood units
  • New clinical applications for cord blood therapies
May 1608:55
Conference pass

Chair’s opening remarks

Cord Blood
Dr Moshe Israeli, Medical Director, Rabin Medical Center
May 1609:00
Conference pass

Chair's opening remarks

Keynotes
Vivienne Parry, Head Of Engagement, Genomics England
May 1609:10
Conference pass

Cord blood based adoptive immunotherapy for patients with relapsed/refractory acute myelogenous leukemia

Cord Blood
  • Anti-leukemic effect of Cord Blood grafts
  • Selecting CB units to enhance antileukemic effects
  • Feasibility of using CB units for refractory AML
Machi Scaradavou, Medical Director Of The National Cord Blood Program, New York Blood Center
May 1609:15
Conference pass

Fireside chat: A new era in medicine- introducing gene therapy

Keynotes
  • What we’ve learned – lessons science has taught us as we create the path for the first gene therapy for an inherited disease in the U.S.
  • Staying true to our roots while preparing the patient, medical and policy maker communities for a new chapter in the retinal disorders textbook
  • Assessing the impact of potential one-time treatments like gene therapies for patients with rare diseases
Interviewer: Vivienne Parry, Head Of Engagement, Genomics England
May 1609:30
Conference pass

Intravenous infusion of umbilical cord tissue (UC) derived Mesenchymal Stem Cells (MSCs) versus bone marrow (BM) derived MSCs to evaluate cytokine suppression in Patients with chronic inflammation due

Cord Blood
  • A Phase I/II, Randomized, Placebo-controlled Comparative Study to compare Umbilical Cord Tissue (UC) Derived Mesenchymal Stem Cells (MSCs) Versus Bone Marrow (BM) Derived MSCs
May 1609:45
Conference pass

(ex vivo) Gene edited therapeutics and (in vivo) gene therapy: how can gene technology be used now and, in the future, to treat and cure diseases in the clinic

Keynotes
  • Current opportunities and limitations in gene therapy
  • Moving into larger diseases from rare diseases, what challenges need to be overcome?
  • Risk/benefit acceptance of the general population and the medical system
  • How do we want to use it in the future and what challenges need to be overcome to do so?
  • Tackling market access challenges and reimbursement hurdles
  • How do you move towards that goal in a way that engages stakeholders?
  • Supply chain and logistics
Vivienne Parry, Head Of Engagement, Genomics England
Doug Danison, Head of Global Pricing, Reimbursement, and Market Access, bluebird bio
Michael Binks, Vice President, Rare Disease Clinical Research, Pfizer
May 1609:50
Conference pass

Keynote Panel Discussion - The future of the cord blood industry – what are the advances that need to be made?

Cord Blood
  • Cord blood banking
  • Research and collaborative efforts
  • Regulation
  • Public awareness and education
Dr Joanne Kurtzberg, Director, Carolinas Cord Blood Bank At Duke
Mayur Abhaya, Managing Director And Chief Executive Officer, Lifecell Ltd
May 1611:30
Conference pass

Keynote presentation: Recent Progress in iPS Cell Research and Application

Keynotes
May 1611:40
Conference pass

Roundtables:

Cord Blood
Amnon Pelz, Chief Executive Officer, Taburit
Chris Bond, Director, Future Health Biobank
May 1611:50
Conference pass

State of the industry address: future state regulatory and compliance considerations for cold chain distribution of regenerative therapies

Keynotes
  • Current compliance and regulatory practices are insufficient in managing risk during distribution
  • Informatics integration, data continuity, and leveraging real time data collection will be key considerations
  • Compliance practices including supply chain traceability are likely to be regulated in the near future
May 1612:10
Conference pass

Technology and Strategy Roundtables

Keynotes
25 senior level tables hosted by thought leaders on key challenges and opportunities in next-generation therapeutic development. Participants are invited to join the group discussions on a topic of importance to them.

· At each roundtable, the moderator will talk for 5 minutes to introduce the topic highlighting particular challenges specific to that area· The table will then move on to discussion for 25 minutes.· There will then be one rotation, giving participants the opportunity to have join two round tables in total

(2 x 30-minute rotations and max 15 PAX)
Table 1. Raw material supply for the commercial manufacturing of cell and gene therapies
Table 10. Purification of stem cells
Stefano Menegatti, Assistant Professor In Chemical And Biomolecular Engineering, North Carolina State University
Table 11. Autologous VS Allogeneic - What it really comes down to?
Nick Gaddum, Lead Device And Delivery Scientist, Cell and Gene Therapy Catapult
Ohad Karnieli, Chief Executive Officer, Atvio Biotechnology
Table 12. New strategies for renal failure
Table 13: Making allogeneic cell therapy a reality
Martha Rook, SVP of Manufacturing, Sigilon Therapeutics
Rogerio Vivaldi, Chief Executive Officer, Sigilon Therapeutics
Table 14. Exploring the crossover between human and veterinary applications for stem cells
Joanna Miller, Chief Scientific Officer, Cell Therapy Sciences
Table 15. How to monitor the safety of viral vectors and other genome modifying therapies in clinical trials?
Table 16. Process development and scale-up strategy for a cryo-product in the context of a broad allogeneic cell therapy
Benoit Champluvier, CMC Senior Advisor,, Icosavax
Table 2. How to manage cell and gene therapy delivery from ‘end to end’
Table 3: Navigating the pathway to clinical success
Table 4. Stem cells for neurological applications
Table 5. MSCs
Alain Vertes, Vice President, Alliance Management, Mesoblast Inc
Table 6. Small molecule drug discovery to selectively stimulate regenerative processes
Table 8. Tissue engineering for organ regeneration
Jeff Ross, Chief Executive Officer, Miromatrix
Table 9. How companies can interact with the regulator and various pitfalls to be aware of (aimed at start-up companies and those from outside the EU (not used to EU or UK ways)
May 1612:40
Conference pass

An overview of World Cord Blood day

Cord Blood
Rachel Manley, Head Of Global Outreach, Save the Cord Foundation
May 1614:10
Conference pass

Chair's remarks

Pricing and Market Access: Value, Pricing, Affordability
May 1614:10
Conference pass

Chair’s remarks

Gene Modified Cell Therapy
Sven Kili, Vice President And Head Of Gene Therapy Development, Sven Kili Consulting
May 1614:10
Conference pass

Chair's remarks

Gene Therapy
Antonio Regalado, Senior Editor, M.I.T. Technology Review
May 1614:10
Conference pass
May 1614:10
Conference pass

Chair's remarks

Cell Therapy Manufacturing
Dr Qasim Rafiq, Associate Professor, Bioprocessing Of Cell And Gene Therapies, University College London
May 1614:10
Conference pass
May 1614:10
Conference pass

Chair's remarks

Pitch and Partner
Vivienne Parry, Head Of Engagement, Genomics England
May 1614:15
Conference pass

Tackling the issue of pricing reimbursement and market access for ATMPs

Pricing and Market Access: Value, Pricing, Affordability
Robert Beckman, Managing Partner, The Channel Group, LLC
May 1614:15
Conference pass

Engineering T cells to specifically enhance activation via co-stimulation and the duration of response via IL-2 independent switches

Gene Modified Cell Therapy
Neill Moray Mackenzie, Chairman And Board Mamber, Cellular Therapeutics Ltd
May 1614:15
Conference pass

Translating in vivo genome editing capabilities into therapeutics

Gene Therapy
May 1614:15
Conference pass

Case study: IC-T immunotherapy programme, implementing clinical trials and translation to patient care

Clinical Trials: Cell Therapy
  • Examples of working with various sizes of biotech and pharma
  • Logistics, coordination, communication cooperation
  • Setting a benchmark of requirements for protocols to streamline operations from both sides
May 1614:15
Conference pass

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

Cell Therapy Manufacturing
When should you think about in-house manufacture instead of outsourcing? 
Martha Rook, SVP of Manufacturing, Sigilon Therapeutics
May 1614:15
Conference pass

Manufacturing of a new Gene therapy product: from early decision to selection for Phase I to Phase III

Viral Vector Manufacturing
  • Academics to CMO: early choice for long term impact?
  • Risk & Opportunities of potential options & current trends
  • What could be an industrial partnership offer without building our own plant?
May 1614:15
Conference pass

Investor panel: How to attract investment into cell and gene therapy: hear how different investors decide on who and what they invest in, and why

Pitch and Partner
  • Compare and contrast different VC methodologies
  • What other types of innovative funding is there?
  • Unorthodox ways to get money
  • What do pharma look for when in-licensing?
Owen Smith, Principal, 4Bio Capital
Alan Muir, Director And Co-Founder, Seven Hills Venture Partners
May 1614:20
Conference pass

Unique features of reconstituting immune cells after cord blood transplantation; opportunities for adjuvant immunotherapies

Cord Blood
  • Immune reconstitution after cord blood transplantation is distinct from reconstitution after other cell sources
  • Early immune reconstitution after cord blood transplantation is excellent and has unique features to get better infectious and disease control
  • Immune reconstitution monitoring should be standardized / harmonized across clinical trials
May 1614:35
Conference pass

Implementing strategic development and innovating disruptive business models for transformative cell and gene therapies

Pricing and Market Access: Value, Pricing, Affordability
· Prioritizing opportunities within the complex landscape of advanced therapeutics· Developing innovative healthcare business models for in-vivo and ex-vivo manufactured gene and cell therapies· Maintaining robust and sustainable growth in rare disease· Perspectives from investors, biotech and pharma
May 1614:35
Conference pass

Phase I evaluation of Pan-ErbB targeted CAR T-cell immunotherapy of head and neck cancer

Gene Modified Cell Therapy
John Maher, Chief Scientific Officer, Leucid Bio
May 1614:35
Conference pass

Panel discussion: Delivering on the Promise of Gene Therapy - Making genetic therapies a reality for patients

Gene Therapy
Attendees will have the opportunity to hear from a panel of experts as they discuss the importance of commercial, scientific and clinical working together, every step of the way, from development to commercialization, to bring a new treatment to patients.
May 1614:35
Conference pass

Panel discussion: Clinical development of next generation cellular therapeutics

Clinical Trials: Cell Therapy
  • Clinical updates and discussion
May 1614:35
Conference pass

Case study: Developing an allogenic product - procurement to patient

Cell Therapy Manufacturing
  • Platform selection – Why Gamma-delta T cells?
  • Understanding of the translational challenges to allogeneic treatments
  • Appreciation of operational challenges
Angela Scott, Chief Operating Officer, TC BioPharm
May 1614:35
Conference pass

Manufacturing Viral Vectors on a Commercial Level - Process Considerations

Viral Vector Manufacturing
Paul S Carter, Cell & Gene Therapy Platform Cmc, GlaxoSmithKline
May 1614:45
Conference pass

Optimization of a stem cell agonist cocktail for the expansion of cord blood hematopoietic stem cells

Cord Blood
  • Review advances in HSC expansion technologies
  • Optimization of small molecules-based HSC agonist cocktail by statistical design of experiment
  • Engraftment properties of cord blood hematopoietic stem cells expanded by small molecules
May 1614:55
Conference pass

Engineering B cells as a living and evolving drug

Gene Modified Cell Therapy
  • CRISPR/Cas9 B cell engineering
  • in vivo activation of adoptively transferred engineered B cells
  • Affinity maturation of engineered B cells to counteract HIV evolution
May 1614:55
Conference pass

How global harmonization will impact the future of cell & gene manufacturing

Cell Therapy Manufacturing
May 1614:55
Conference pass

Gene Therapy Product Analytics: Where Historical and Modern Techniques Meet

Viral Vector Manufacturing
As the acceptances of viral vectors as a delivery system for therapeutics grows, biomanufacturers are looking for an alternative to the classical adherent cell production models
Suparna Taneja-Bageshwar, Principal Scientist and AD Group Leader, Fujifilm Diosynth Biotechnologies
May 1614:55
Conference pass

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

Pitch and Partner
  • How to approach big pharma/governmentorganisationsfor 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
Glenn F Pierce, CMO, Third Rock Ventures, LLC
Robert Beckman, Managing Partner, The Channel Group, LLC
Gareth King, Manager, Catapult Ventures
May 1615:10
Conference pass

Improvement of functional preservation of cord blood stem and progenitor cells stored at +4°C after collection or after ex vivo expansion: development of a potentially injectable conservation solution

Cord Blood
  • After ex vivo expansion, the hematopoietic stem cells (Scid Repopulating Cells) rapidly decline in human-albumin solution (used routinely) and their activity is almost completely lost after 48h. This can be prevented by using an appropriate culture medium instead of human albumin (antioxidants, nutritive elements, microelements).
  • The decline of hematopoietic stem cell activity (although to a lesser extent) detected after cord blood collection using the standard routine procedure can be prevented by using the same culture medium.
  • This medium is not an optimal choice since it should be removed before injection of the cells into the patient. Thus, we have developed a conservation solution composed of already registered injectable pharmaceutical products, able to reproduce the same beneficial effect using both freshly collected cord blood cells and ex vivo expanded ones
May 1615:44
Conference pass

INVESTOR JUDGES:

Pitch and Partner
Glenn F Pierce, CMO, Third Rock Ventures, LLC
Sam Hall, Partner, Apple Tree Partners
Robert Beckman, Managing Partner, The Channel Group, LLC
Ian Mckay, Innovation Lead Advanced Therapies, Innovate UK
May 1615:45
Conference pass

Value, pricing and affordability of Advanced Therapies

Pricing and Market Access: Value, Pricing, Affordability
Doug Danison, Head of Global Pricing, Reimbursement, and Market Access, bluebird bio
May 1615:45
Conference pass

Clinical and commercial application of scaled human stem cell derivates in immune- oncology

Gene Modified Cell Therapy
May 1615:45
Conference pass

Implementation of cross-border healthcare: can you guarantee a patient access to therapies in another country?

Gene Therapy
May 1615:45
Conference pass

Conditioning for CAR-T reconstitution: early clinical data and analysis

Clinical Trials: Cell Therapy
May 1615:45
Conference pass

Cell Therapy and Cell – Gene Bio-Manufacturing: A SWOT Analysis of CDMO Market Demand and Future Capacity

Cell Therapy Manufacturing
  • Cell Therapy and Cell – Gene Bio-Manufacturing: A SWOT Analysis of CDMO Market Demand and Future Capacity
  • Market analytics will be presented to identify Industry Strengths, Weaknesses, Opportunities and Threats
  • A comprehensive view of market trends will be presented
May 1615:45
Conference pass

Continuous, integrated processes supporting robust and cost-effective viral vector productions

Viral Vector Manufacturing
  • Real data from constitutive production and from continuous downstream purification of, eg., lentivirus and oncolytic viruses confirms these as key steps to improve consistency, robustness and reduced costs of goods for cell and gene therapy.
May 1615:45
Conference pass

Pitch 1. Harnessing synthetic nanoclay gels for regenerative medicine

Pitch and Partner
May 1616:00
Conference pass

Pitch 2. Regenerative medicine – a small molecule approach

Pitch and Partner
May 1616:05
Conference pass

Panel discussion: Innovative reimbursement & financing models for rare disease treatments in Europe and the USA

Pricing and Market Access: Value, Pricing, Affordability
May 1616:05
Conference pass

Miguel Forte - Company Presentation

Gene Modified Cell Therapy
May 1616:05
Conference pass

Short (10 minute) company presentations followed by panel discussion: Development strategies for CAR-T and TCR therapies with the late stage in mind

Gene Modified Cell Therapy
  • Allogenic vs autologous
  • Understanding different opportunities: selecting the correct product for the appropriate purpose
  • Improving outcomes tackling solid tumours with CAR-T therapy
  • Moving towards a more off the shelf approach for better cost of goods
Sean Ainsworth, Chief Executive Officer And Chairman, Immusoft Corporation
Stefanos Theoharis, Senior Vice President Of Corporate Development And Partnering, Cell Medica Ltd
Neill Moray Mackenzie, Chairman And Board Mamber, Cellular Therapeutics Ltd
May 1616:05
Conference pass

Preclinical and clinical development of tolerogenic nanoparticles to mitigate immunogenicity against enzyme and gene therapies for orphan diseases

Gene Therapy
  • Immunogenicity is a common cause of treatment failure and hypersensitivity reactions with biologic therapies, particularly for enzyme therapies and gene therapies for orphan and rare diseases
  • We have developed technology to induce antigen-specific immune tolerance to biologic therapies
  • I will present case studies from our lead clinical program in severe refractory gout and preclinical program in gene therapy
May 1616:05
Conference pass

Single Administration of CD34 cell Reverses Cardiovascular disease: Evidence from >700 patients in randomized clinical trials

Clinical Trials: Cell Therapy
  • CD34 cells are naturally occurring, pre-programmed vascular repair cells
  • Pre-clinical models document the ability of locally delivered CD34 cells to induce the formation of new micro vessels in ischemic tissue, resulting in improved perfusion and function
  • Clinical studies in multiple cardiovascular diseases provide evidence of durable clinical benefit following a single administration of autologous CD34 cells
Douglas Losordo, Chief Medical Officer, Caladrius Biosciences
May 1616:05
Conference pass

Novel closed systems for CAR T-cell production

Cell Therapy Manufacturing
  • Closed non-magnetic selection technology (automated T-CATCH)
  • Soluble reversible activation technology (Expamer)
  • Short production process
Mateusz Poltorak, Research Scientist, Juno Therapeutics
May 1616:05
Conference pass

Viral vector manufacturing case study

Viral Vector Manufacturing
May 1616:15
Conference pass

Neill Moray Mackenzie - Company Presentation

Gene Modified Cell Therapy
Neill Moray Mackenzie, Chairman And Board Mamber, Cellular Therapeutics Ltd
May 1616:15
Conference pass

Pitch 3. Caladrius Biosciences will discuss its commitment to the development of innovative therapies addressing unmet medical needs

Pitch and Partner
May 1616:25
Conference pass

Evren Alici - Company Presentation

Gene Modified Cell Therapy
May 1616:25
Conference pass

Strategies for communicating to multiple stakeholders: rare diseases in the spotlight

Gene Therapy
May 1616:25
Conference pass

Panel discussion: Advanced Therapies Treatment Network: facilitating adoption, proof of adoption and how we get there, challenges and adoption

Clinical Trials: Cell Therapy
- What are your challenges?
Doug Danison, Head of Global Pricing, Reimbursement, and Market Access, bluebird bio
Ian Mckay, Innovation Lead Advanced Therapies, Innovate UK
Marc Turner, Medical Director, Scottish National Blood Transfusion Service
May 1616:25
Conference pass

PRIME XV defined media solutions for Cancer immunotherapy applications

Cell Therapy Manufacturing
  • Impact of culture setup on cell culture media performance
  • Understanding the benefits of using chemically-defined media in immunotherapy applications
  • Introduction to PRIME XV cancer immunotherapy portfolio
May 1616:25
Conference pass

Accurate purity data in time helps to avoid late surprises when scaling up. Automated TEM analysis data of gene therapy vectors and VLPs

Viral Vector Manufacturing
Challenges with scale up of gene therapy processes include surprises in purity profiles. Robust analytical solutions are required to to avoid late surprises. Transmission electron microscopy (TEM) provide unmatched insights in characterization of viral gene vectors.MiniTEM automatically provides:
  • Comparative metrics on purity profiles of viral particle samples
  • Automatically differentiates intact viral particles from process related impurities
  • Correlation between potency and viral particle morphology
May 1616:30
Conference pass

Pitch 4. A new TCR approach to take solid tumours

Pitch and Partner
May 1616:30
Conference pass

Advances and challenges in umbilical cord blood and tissue bioprocessing

Cord Blood
  • Umbilical cord tissue and blood is the rapidly advancing field of regenerative medicine for both autologous and allogeneic therapeutic applications
  • Optimization of GMP-compliant manufacturing strategies, collection and bioprocessing of cord blood and tissue
  • Comparison with other type of cells
  • Regulatory challenges and considerations for minimal manipulation and homologous use
  • Future direction; iPSCs or exosomes
Aisha Khan, Executive Director Of Laboratory Operations, Interdisciplinary Stem Cell Institute
May 1616:35
Conference pass

Stefanos Theoharis - Company Presentation

Gene Modified Cell Therapy
Stefanos Theoharis, Senior Vice President Of Corporate Development And Partnering, Cell Medica Ltd
May 1616:45
Conference pass

Michael Dombeck - Company Presentation

Gene Modified Cell Therapy
May 1616:45
Conference pass
May 1616:45
Conference pass

Series of 10-minute updates from gene therapy organisations followed by a panel discussion on strategically improving issues in vivo gene therapy 

Gene Therapy
  • In vivo gene therapy – moving away from the bias of AAV  
  • Why the field has accelerated so rapidly catalyzed CAR-T 
  • Solving the problem of high manufacturing costs 
  • AAV vs Lenti platforms pros and cons 
How industry, academia and patient organizations can work together to improve outcomes for patients. 
Bodil Willumsen, Supplier Relationship Manager, External Supply Operations, Cell And Gene Therapy, Novartis
Fraser Wright, Chief Technology Officer, Axovant Sciences
May 1616:45
Conference pass

The use of enhanced GMP manufacturing techniques boosts the success of Large-Scale production of EMA-approved Cartilage Substitute

Cell Therapy Manufacturing
May 1616:45
Conference pass

BAC-to-AAV: The Technology for Large Scale AAV Manufacturing

Viral Vector Manufacturing
  • Current AAV production systems and their challenges
  • First generation system for AAV production in Sf9 cells and its deficiencies
  • Virovek's serum-free "BAC-to-AAV" technology and its advantages
  • Novel three-phase partitioning (TPP) as upstream process for large scale AAV purification
May 1616:45
Conference pass

Pitch 5. Taking the bull by its horns: regenerating human hearts with human heart cells

Pitch and Partner
May 1616:50
Conference pass

Toticyte – a revolution in cord blood processing

Cord Blood
  • >99.5% haematocrit removal
  • At least 3x more viable stem cells at point of use than any other method
  • Maximum cell recoveries, even for small volumes
  • Primitive cell types retained
  • Clinically compatible
  • HTA authorised use, CE marked, ISO13485 certified
May 1617:00
Conference pass

Use of lentiviral vectors for the lung: approach, challenges and potential indications

Gene Therapy
May 1617:00
Conference pass

Pitch 6. Pioneering a new class of medicines: shielded living therapeutics

Pitch and Partner
Rogerio Vivaldi, Chief Executive Officer, Sigilon Therapeutics
May 1617:05
Conference pass

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

Pricing and Market Access: Value, Pricing, Affordability
· Structured reimbursement system for hospital-based treatments in Europe· What is possible for statutory health insurance framework in the EU?· Contract possibilities with manufacturers, hospitals and insurance providers
Dorothee Andres, Senior Policy Adviser, Federal Ministry of Health Germany
Katja Schmidt, Head Of Market Access And Public Affairs, Novartis
Carla Deakin, Programme Director - Commercial And Managed Access, NICE
Michèle De Guise, Director Of Health Services And Technology Assessment, Inesss
May 1617:05
Conference pass

Panel discussion: Development strategies for CAR-T and TCR therapies with the late stage in mind

Gene Modified Cell Therapy
Neill Moray Mackenzie, Chairman And Board Mamber, Cellular Therapeutics Ltd
Stefanos Theoharis, Senior Vice President Of Corporate Development And Partnering, Cell Medica Ltd
May 1617:05
Conference pass

Manufacturing and delivering CAR-T cell therapy for cancer

Clinical Trials: Cell Therapy
May 1617:05
Conference pass

Dynamic Storage: A New Perspective on Advanced Therapy Supply Chain

Cell Therapy Manufacturing
  • Current and future required quality systems standards for the regenerative medicine industry
  • A comprehensive review of the basic quality systems requirements
  • Regulatory considerations critical in the successful launch and management of the risks in distribution
May 1617:05
Conference pass

PANEL: progress and challenges in Viral Vector Manufacturing: assays and analysis

Viral Vector Manufacturing
How to look at the percentage empty vs filled viral particles  Looking a potency of construct, how can this be measured and what are the tools? Infection of cell: analysing gene expression, functionality, level and potency 
May 1617:10
Conference pass

Panel discussion: the operational challenges of cord blood collection and cryopreservation

Cord Blood
  • Review of collection procedures
  • Best practices recommendations: modifying infrastructure and support
  • Methods to improve conversion rate
May 1617:15
Conference pass

Greg MacMichael - Update

Gene Therapy
Greg MacMichael, SVP of Technical Operations, Axovant Sciences
May 1617:15
Conference pass

Pitch 7. Bioengineering Transplantable Whole Organs

Pitch and Partner
Jeff Ross, Chief Executive Officer, Miromatrix
May 1617:25
Conference pass

New clinical data: Effects of an autologous cell therapy in patients with advanced chronic kidney disease associated with diabetes mellitus

Clinical Trials: Cell Therapy
  • Translational medicine of a cellular therapy for chronic kidney disease
  • Delivering cellular therapies from-and-to the patient
  • Defining a dosing regimen
May 1617:25
Conference pass

Supply chain management, logistics and scale out for T-Cell products

Cell Therapy Manufacturing
  • Case study of a soon to be market authorised product
  • Manufacture and logistics of delivery to the patient
  • Maintaining quality product at every stage of the delivery process
May 1617:30
Conference pass

Bodil Willumsen - Update

Gene Therapy
Bodil Willumsen, Supplier Relationship Manager, External Supply Operations, Cell And Gene Therapy, Novartis
May 1617:30
Conference pass

Pitch 8. A Next Generation Cell Therapy Company

Pitch and Partner
Ross Macdonald, Chief Executive Officer, Cynata Therapeutics Ltd
May 1617:45
Conference pass

Panel discussion: Using imaging to improve the success rate of cell therapies

Gene Modified Cell Therapy
May 1617:45
Conference pass

Luca Alberici - Update

Gene Therapy
May 1617:45
Conference pass

Panel discussion: Ensuring safe clinical development of Cell Therapies (CAR-T, DCs, Tregs etc.)

Clinical Trials: Cell Therapy
  • Safety of new T-Cell Therapies while going through trials
  • Safety signals: how can we feed this back to developers?
  • What should the regulators be looking for?
  • Challenges and problems when developing new T-Cell therapies in the clinic
Alberto Sanchez Fueyo, Professor In Hepatology, King's College London
John Maher, Chief Scientific Officer, Leucid Bio
Douglas Losordo, Chief Medical Officer, Caladrius Biosciences
May 1617:45
Conference pass

BIOPROCESSING CLOSING PLENARY: Panel discussion: How to manage cell and gene therapy delivery from ‘end to end’

Cell Therapy Manufacturing
  • Sample collection, handling and logistics 
  • Regulation 
  • Product distribution 
  • Cryo-shipping strategies 
  • Manufacturing automation and cell therapy 
  • Reimbursement and integrated care 
May 1617:45
Conference pass

BIOPROCESSING CLOSING PLENARY: Panel discussion: How to manage cell and gene therapy delivery from ‘end to end’

Viral Vector Manufacturing
  • Sample collection, handling and logistics 
  • Regulation 
  • Product distribution 
  • Cryo-shipping strategies 
  • Manufacturing automation and cell therapy 
  • Reimbursement and integrated care 
May 1617:45
Conference pass

Judges deliberation, winners will be announced at the networking drinks reception.

Pitch and Partner
Sponsorship opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, erica.baeta@terrapinn.com) for further details
May 1618:00
Conference pass

Panel discussion: strategically improving issues in vivo and ex vivo gene therapy

Gene Therapy
Bodil Willumsen, Supplier Relationship Manager, External Supply Operations, Cell And Gene Therapy, Novartis
Greg MacMichael, SVP of Technical Operations, Axovant Sciences
May 1618:05
Conference pass

Closing remarks

Pricing and Market Access: Value, Pricing, Affordability
· Structured reimbursement system for hospital-based treatments in Europe· What is possible for statutory health insurance framework in the EU?· Contract possibilities with manufacturers, hospitals and insurance providers
May 1618:20
Conference pass

Closing remarks

Gene Modified Cell Therapy
Sven Kili, Vice President And Head Of Gene Therapy Development, Sven Kili Consulting

Create your personal agenda –check the favourite icon

May 1708:30
Conference pass

Chair's opening remarks

Keynotes
Vivienne Parry, Head Of Engagement, Genomics England
May 1708:40
Conference pass
May 1709:00
Conference pass

Keynote panel discussion: Exploring advances in gene editing technology, therapeutics and research

Keynotes
  • Best strategies for implementing new methods across the field of clinical research, now and for the future
  • Optimisation of the delivery of gene editing technology
  • How to minimise off-target effects and mutations for the safe and effective delivery of gene editing technology
  • Discussion of ethical and regulatory challenges, current legislation, how to navigate ethical concerns in light of the growing scale of genome editing. Most recently the CRISPR babies that were born in 2018
May 1709:00
Conference pass

Using the whole cord blood to make novel therapies

Cord Blood
  • Use of umbilical cord blood cells for immunotherapies
  • Potential uses of cord blood plasma and platelets
  • Uses of cord blood derived mesenchymal stem cells
May 1709:20
Conference pass

The application of hyperbaric oxygen therapy to improve umbilical cord blood stem/progenitor cell homing and engraftment

Cord Blood
  • Erythropoietin signaling mediates umbilical cord blood CD34+ cell differentiation, bone marrow homing, and engraftment
  • bloodHyperbaric oxygen (HBO) therapy in the setting of clinical umbilical cord blood transplantation is well-tolerated and is potentially associated with improved long-term outcomes
  • HBO reduces erythropoietin serum level and improves blood count recovery in the setting of clinical umbilical cord blood transplantation
May 1709:40
Conference pass

Keynote presentation: CAR T Immunotherapy – Opportunities and Challenges

Keynotes
  • Understand the role of CAR-T cell therapy in relapsed ALL
  • Understand the risk factors and therapies for Cytokine Release Syndrome (CRS)
  • Understand the importance of T cell proliferation and persistence in CAR-T cell efficacy
May 1709:40
Conference pass

Cord blood for prevention of type 1 diabetes

Cord Blood
  • CD4+CD25+ regulatory T cells (Treg) in umbilical cord blood provide potential as an immunotherapeutic tool
  • Considerable evidence indicates that type 1 diabetes is characterised by abnormalities in Treg number and function
May 1710:00
Conference pass

Keynote panel: Innovations in cellular and combination immunotherapy: current learnings, challenges and future applications

Keynotes
  • Using the immune system to identify and fight cancer cells: where are we and what do we need to maximise patient benefit in the future?
  • Improving cellular immunotherapy success by using genetic biomarkers to create precision medicines and improve success rates
  • Where are we with combination immunotherapy, what needs to be done to improve efficiency and effectiveness in the future
  • Moving from oncology to non-oncology applications, how can we make the shift to new therapy indications
  • Preventing relapse and resistance
May 1710:00
Conference pass

Therapeutic potential of UCB-derived extracellular vesicles

Cord Blood
  • Clinical use of Extracellular vesicles: Opportunities and Threats
  • How to go from UCB to a GMP-compliant product based in extracellular vesicles for Clinical application
  • Therapeutic potential of Exogenus Therapeutics’ proprietary small extracellular vesicles
  • Pipeline development and future Roadmap
May 1711:00
Conference pass

Chair's remarks

Pricing and Market Access: Approval and Evidence
Igor Rudychev, Senior Director, Business Insights, AstraZeneca
May 1711:00
Conference pass
May 1711:00
Conference pass
May 1711:00
Conference pass

Chair’s remarks

Clinical Development: Gene Therapy
Annie Hubert, Director, European Public Policy, Alliance for Regenerative Medicine
May 1711:00
Conference pass
May 1711:00
Conference pass

Chair's remarks

Viral Vector Manufacturing
May 1711:05
Conference pass

The newest changes in regulation and market access for ATMPs in Germany

Pricing and Market Access: Approval and Evidence
Dorothee Andres, Senior Policy Adviser, Federal Ministry of Health Germany
May 1711:05
Conference pass

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

Immunotherapy
  • PD-1 antibodies have shown significant activity across more than 25 major cancer types.
  • 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 + PD-1 antibodies) may be broadly active without regard for biomarker-based selection.
May 1711:05
Conference pass

Human liver bioengineering: from implantable liver to whole organ engineering

Cell Therapy and Regenerative Medicine
May 1711:05
Conference pass

TBC Clinical trials case study 1 - Telethon

Clinical Development: Gene Therapy
May 1711:05
Conference pass

Enabling commercial scale-out of T-cell manufacturing

Cell Therapy Manufacturing
  • What are the key challenges in scaling an autologous process for commercial?
  • The role of closed systems in scalability
  • Understanding and controlling Key Quality Attributes
May 1711:05
Conference pass

Application of the new EU GMP guidelines specific to ATMPs – a quality assessor’s perspective

Viral Vector Manufacturing
May 1711:20
Conference pass

Translational medicine in Russia: how it works

Cord Blood
  • Transitioning from a cord blood bank to a centre of fundamental research and regenerative medicine
  • Preliminary research results (morphofunctional properties of mesenchymal stromal cell during long-term expansion and methods of intravital MMSC labeling with superparamagnetic nanoparticles)
  • Experience in clinical application of cell technologies (traumatology, liver diseases) as well as particularities of national federal legislation in the field of regenerative medicine will be discussed
May 1711:25
Conference pass

Value-based pricing and reimbursement for orphan drugs

Pricing and Market Access: Approval and Evidence
May 1711:25
Conference pass

Cellular medicines for inflammatory diseases

Cell Therapy and Regenerative Medicine
Alain Vertes, Vice President, Alliance Management, Mesoblast Inc
May 1711:25
Conference pass

Key Considerations in Clinical Development of Advanced Therapy Medicinal Products

Clinical Development: Gene Therapy
May 1711:25
Conference pass

Creating a roadmap for CAR T-cell therapies to achieve commercial success

Cell Therapy Manufacturing
Reserved for Hamilton Bonaduz AG
May 1711:25
Conference pass

Scaling up adherent viral vector manufacturing

Viral Vector Manufacturing
May 1711:30
Conference pass

Combining a soluble LAG-3 protein with an anti-PD-1 antibody in phase I-II trials

Immunotherapy
May 1711:40
Conference pass

Success story of the largest cord blood bank in Europe

Cord Blood
  • European CB banking market
  • Key Success factors
  • FamiCord medical activity
Tomasz Baran, Board Member, PBKM/FamiCord Group
May 1711:45
Conference pass

Case study: building a cell therapy business in Japan

Pricing and Market Access: Approval and Evidence
Reserved for Cardinal Health
May 1711:45
Conference pass

Moving from autologous towards fully allogenic cell therapy

Cell Therapy and Regenerative Medicine
May 1711:45
Conference pass

Gene therapy clinical development case study

Clinical Development: Gene Therapy
May 1711:45
Conference pass

Optimizing CGT manufacturing workflows for industrial scale manufacturing

Cell Therapy Manufacturing
May 1711:45
Conference pass

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

Viral Vector Manufacturing
  • LV and RV production development experiments using Pall iCellis nano instrument
  • Scale up to iCellis500 for about 200L RV/LV production
  • Results and comparability data versus iCellis nano and standard GMP Cell Factories LV /RV vectors
May 1711:55
Conference pass

Precision medicine in oncology: challenges and possible solutions from a payer’s perspective

Immunotherapy
Lucy Hoppe, Clinical Effectiveness Manager (Evidence Synthesis) In The Clinical Effectiveness Team, Bupa
May 1712:00
Conference pass

Can frequency of therapeutic application of stem cells be influenced?

Cord Blood
  • The meaning of availability, reimbursement, gaining scientific reputation and clinical evidence
May 1712:05
Conference pass

Reimbursement within the world of Oncology

Pricing and Market Access: Approval and Evidence
Igor Rudychev, Senior Director, Business Insights, AstraZeneca
May 1712:05
Conference pass

Personalisation of Research

Clinical Development: Gene Therapy
May 1712:05
Conference pass

Manufacturing advances and scale-up for simple and complex ATMPs

Cell Therapy Manufacturing
  • Starting with the end in mind – moving from early to later phase trials with a core validated protocol
  • Incremental improvement in processes to achieve large increases in output
  • Automation in advanced therapeutics manufacturing
May 1712:05
Conference pass

Development and manufacture of therapeutic vaccines against cancer

Viral Vector Manufacturing
May 1712:20
Conference pass

Panel discussion: Precision medicine for immunotherapy 

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?
Lucy Hoppe, Clinical Effectiveness Manager (Evidence Synthesis) In The Clinical Effectiveness Team, Bupa
Iraj Ali, Chief Executive Officer, Achilles Therapeutics
May 1712:20
Conference pass
May 1712:25
Conference pass

Starting materials and standards in relation to advanced therapies

Pricing and Market Access: Approval and Evidence
May 1712:25
Conference pass

Panel Discussion: Harnessing the body's regenerative capabilities – therapeutic development from the clinic to commercialization

Cell Therapy and Regenerative Medicine
Speaking opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, erica.baeta@terrapinn.com) for further details 
May 1712:25
Conference pass
May 1712:25
Conference pass

Moving from artisanal to robust and broad manufacturing models

Cell Therapy Manufacturing
Richard Mcfarland, Chief Regulatory Officer, Advanced Regenerative Manufacturing Institute
May 1712:25
Conference pass

Panel discussion: Assays - specific challenges, duration, standardization

Viral Vector Manufacturing
  • Potency assays for comparability,characterizationand release
  • What type of release assays are adequate for a regulator to approve the product in both cell and gene therapies?
  • How do regulators define quality in release assays and how can the industry use this as a guide for regulatory approval?
  • How do regulators validate assays? What kind of assays can actually be validated?
  • Viral vectors and gene modified cell products: are these release assays really necessary? Do you need one for your starting material and one for your end product?
  • Advice on extractables and leachable - what is the regulatory guidance on determining the purity of the final product? Which tests have worked well for products that have made it to market?
  • Different regulations for EU and US- can weharmoniseon the quality aspect of release testing?
  • How do you predict if a batch will be successful at a clinical level?
May 1712:45
Conference pass

US Cell & Gene Therapy Regulatory Policy Update

Pricing and Market Access: Approval and Evidence
May 1712:45
Conference pass

Vector safety in gene and immune gene therapies

Clinical Development: Gene Therapy
  • Comprehensive integration profile of your viral vectors
  • Pharmacovigilance and vector safety in pre- and clinical trials
  • Determine potential on-/off-target effects of genome editing
May 1712:45
Conference pass

Transforming cell therapy manufacturing by enabling point of care affordable automated manufacturing

Cell Therapy Manufacturing
Ohad Karnieli, Chief Executive Officer, Atvio Biotechnology
May 1713:40
Conference pass

Application of Cord Blood NK cell therapies in the clinic

Cord Blood
  • Clinical studies suggest that adoptive transfer of allogeneic natural killer (NK) cells represent a promising treatment for patients with hematological malignancies and solid tumors
  • Celularity is developing an allogeneic, off-the-shelf NK cell product that is derived from placental hematopoietic stem cells and exhibits substantial cytolytic activity against various cancer cell lines, primary AML and primary MM cells
  • Clinical and translational development of Celularity NK cell product will be discussed
May 1714:00
Conference pass

Novel therapies using expanded cord blood

Cord Blood
  • MSCs for regenerative medicine and GVHD
  • NK cells for high-risk myeloma
May 1714:05
Conference pass

Common pitfalls and hurdles when reaching registration and market access for a new next generation therapeutic

Pricing and Market Access: Approval and Evidence
  • What are the best ways to approach getting a new therapeutic modality registered and on the market?
  • Key lessons learned from Strimvelis
  • How we can overcome key challenges and get these therapies registered?
May 1714:05
Conference pass

Next generation immuno-oncology

Immunotherapy
New clinical data for 3 immune-oncology clinical trials for ovarian, glioblastoma and melanoma treatment
May 1714:05
Conference pass

Collaborative phenotyping at King’s: the stem cell hotel

Cell Therapy and Regenerative Medicine
May 1714:05
Conference pass

A multi-stakeholder’s collaborative model to improve the success of translational research: a charity’s perspective

Clinical Development: Gene Therapy
Annamaria Merico, Head Of Technology Transfer, Fondazione Telethon
May 1714:05
Conference pass

How much process risk am I carrying forward into clinical stage production?

Cell Therapy Manufacturing
  • Introducing a road map to move the entire industry forward
  • Educational work force development projects
  • Technical projects such as automated control of microfluidics to measure, control and develop processes better
May 1714:05
Conference pass

How much process risk am I carrying forward into clinical stage production?

Viral Vector Manufacturing
May 1714:20
Conference pass

Driving public banked cord blood inventory utilization through research and development

Cord Blood
  • Optimizing HPC,Cord Blood inventory for use in stem cell transplant
  • Developing clinical pathways and product pipelines through Research
  • Leveraging existing infrastructure, experience and resources to diversify cord blood utility with a Cell Therapy Incubator Laboratory
May 1714:30
Conference pass

Panel discussion: ‘Getting it right first time’ – Taking steps early on to ensure success in the later stages

Pricing and Market Access: Approval and Evidence
May 1714:30
Conference pass

Case study of regulatory challenges and achievements for ilixadencel

Immunotherapy
  • ATMP Classification as somatic cell therapy medicinal product – December 2015
  • Scientific Advice (national and EMA, pre-IND)
  • IND cleared by FDA – December 2016
  • ilixadencel (INN) – from WHO January 2017
  • EMA ATMP Certification – March 2018
Margareth Jorvid, Head Of Regulatory Affairs And QA, Immuniucum AB
May 1714:30
Conference pass

Immunology of the stem cell: host response and effect of cell therapy

Cell Therapy and Regenerative Medicine
  • With graft versus host disease success is related to patient’s response to the materials, what is the innate immune response? 
  • What is the effect of repetition of treatment? 
  • Mechanism of action
Frank Barry, Scientific Director, Regenerative Medicine Institute
Ross Macdonald, Chief Executive Officer, Cynata Therapeutics Ltd
May 1714:30
Conference pass

The difficulties encountered for the development and registration of a product of GT and supports proposed by the European Commission and the European Medicines Agency

Clinical Development: Gene Therapy
  • European Commission (EC) offers some grants for OD designated products in Europe.
  • A new regulation for clinical trial authorization is proposed by the EC: a common system for EU clinical trial authorizations
  • The EMA puts in place several possibilities of meetings (scientific Advices common with HTA or FDA) and a continuing support like PRIME system
May 1714:30
Conference pass

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

Cell Therapy Manufacturing
Which is the best path to commercial scale? Real life stories from people who have their own facility Vs. people who have outsourced. Is there a middle ground? What to learn from outsourcing parts of your process, where do you invest and who do you bring in as partners? Do CMOs have the ability to work at global scale? 
May 1714:30
Conference pass

Plenary with Viral Vectors Manufacturing: Panel discussion: Building your own manufacturing facility Vs. outsourcing the process to partners 

Viral Vector Manufacturing
Which is the best path to commercial scale? Real life stories from people who have their own facility Vs. people who have outsourced. Is there a middle ground? What to learn from outsourcing parts of your process, where do you invest and who do you bring in as partners? Do CMOs have the ability to work at global scale? 
May 1714:40
Conference pass

Cellular mechanisms in support of allogenic human amnion epithelial stem cell transplantation without immunosuppression

Cord Blood
  • Stem cell properties typically expressed by human amnion epithelial (AE) stem cells
  • How to isolate and identify placenta-derived AE cells
  • AE transplantation in preclinical model of liver diseases
  • Route of infusion to enhance liver engraftment
  • How allogenic AE cells may avoid immunorejection without immunosuppressant in support
May 1714:50
Conference pass

Allogeneic gamma-delta CAR-T

Immunotherapy
  • Clinical strategy, transition from autologous to allogeneic
  • Manufacture of GMP allogeneic banks
  • CAR-T modification of allogeneic cells
May 1714:50
Conference pass

Maximising benefits from clinical trials developing safe and efficacious gene therapies as quickly as possible

Clinical Development: Gene Therapy
May 1715:10
Conference pass

Chair's closing remarks

Pricing and Market Access: Approval and Evidence
Igor Rudychev, Senior Director, Business Insights, AstraZeneca
May 1715:10
Conference pass

Cellular Mechanisms in support of allogenic human Amnion Epithelial Stem Cell Transplantation without Immunosuppression

Cord Blood
  • Stem cell properties typically expressed by human amnion epithelial (AE) stem cells
  • How to isolate and identify placenta-derived AE cells
  • AE transplantation in preclinical model of liver diseases
  • Route of infusion to enhance liver engraftment
  • How allogenic AE cells may avoid immunorejection without immunosuppressant in support
May 1715:40
Conference pass

Targeting PI3K-gamma with IPI-549, a tumor macrophage-reprogramming small molecule, in patients with advanced solid tumors

Immunotherapy
  • IPI-549 is the only potent, oral, and selective PI3K- inhibitor in clinical development
  • In preclinical studies, IPI-549 reprograms tumour macrophages from an immune-suppressive (M2) phenotype to an immune-activating (M1) phenotype and can overcome intrinsic resistance to checkpoint inhibitors.
  • In an ongoing Phase 1/1b study in approximately 225 patients, IPI--549 is well tolerated and associated with a favourable safety profile and shows clinical activity both in monotherapy and in combination with nivolumab in indications not expected to respond to nivolumab alone. Additionally, a robust translational medicine program has demonstrated IPI-549’s on-target effects, including a reduction of myeloid derived suppressor cells and increased proliferation of previously-exhausted T-cells
May 1715:40
Conference pass

Overcoming barriers to translation of tissue engineered therapies in the UK and EU: defining better routes to clinic for complex therapies

Cell Therapy and Regenerative Medicine
May 1715:40
Conference pass

Panel discussion: Improving patient outcomes in clinical trials and using technology for patient monitoring

Clinical Development: Gene Therapy
May 1715:40
Conference pass

Autostem - Fully automated stem cell production as an enabler for cell-based therapies

Cell Therapy Manufacturing
May 1715:40
Conference pass

Autostem - Fully automated stem cell production as an enabler for cell-based therapies

Viral Vector Manufacturing
May 1715:40
Conference pass

A snapshot of current research into Haploidentical transplants

Cord Blood
  • Summary of the advancements in Haplo-SCT in recent years and comparison of other transplantation modalities
  • Strategies for improving clinical outcomes
  • Donor selection and graft-versus-host disease
Dr Moshe Israeli, Medical Director, Rabin Medical Center
May 1716:00
Conference pass

Personalised cancer immunotherapy, tumour-derived T cell therapy for non-small cell lung cancer

Immunotherapy
Iraj Ali, Chief Executive Officer, Achilles Therapeutics
May 1716:00
Conference pass

Regenerative medicine (chaired by Dr. Stephen Badylak): Current carriers and storage solutions for cell therapies 

Cell Therapy and Regenerative Medicine
  • The ancillary components of cell therapy and its importance on clinical outcomes  
  • Creating the ideal microenvironment for cells during and after introduction to the patient  
  • Counteracting viability risk factors: taking out of the frozen environment, suspension, and injection 
  • What is the best suspension solution to use? How is the cellular treatment dissipated after injection?  
  • How can we make this process more robust? 
May 1716:00
Conference pass

Panel discussion: Moving towards full automation and closed systems

Cell Therapy Manufacturing
  • Manufacturing processes
  • Quality processes
  • Logistics processes
  • How do we do improve all these to decrease the cost of manufacture?
May 1716:00
Conference pass

Panel discussion: Moving towards full automation and closed systems

Viral Vector Manufacturing
  • Manufacturing processes
  • Quality processes
  • Logistics processes
  • How do we do improve all these to decrease the cost of manufacture?
May 1716:00
Conference pass

Panel Discussion: Haploidentical vs cord blood transplantations

Cord Blood
  • Pros and cons of both – case study examples
  • Is there sufficient data to support one donor type over another?
  • Is institutional practice influenced by experience and research interest?
Dr Moshe Israeli, Medical Director, Rabin Medical Center
Andy Peniket, Consultant, Haematologist And Bone Marrow Transplant Physician, NIHR Oxford Biomedical Research Centre
Kavita Raj, Consultant Haematologist, Guy's And St Thomas' Hospital
May 1716:40
Conference pass
May 1716:40
Conference pass
May 1716:40
Conference pass

Chair's closing remarks

Clinical Development: Gene Therapy
Annie Hubert, Director, European Public Policy, Alliance for Regenerative Medicine
May 1716:40
Conference pass
May 1716:40
Conference pass
May 1716:50
Conference pass

Chair's closing remarks

Cord Blood
May 1716:55
Conference pass

Close of conference

Cord Blood
last published: 14/May/19 13:35 GMT

 

The World Advanced Therapies and Regenerative Medicine Congress and Exhibition is an international conference and exhibition featuring the latest in advanced therapies, bioengineering and surrounding technology. To attend the conference which features global leaders in the biopharma and regenerative medicine industries, there is a registration fee. The exhibition is free to visit.

Register your pass here.