Full Agenda

 

London, 15 - 17 May 2019

Schedule

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May 1509:00
Conference pass

Using CRISPR for preclinical drug discovery

Gene Editing Summit
May 1509:00
Conference pass

Welcome remarks

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

Workshop: Automating CAR-T manufacturing, a step by step guide

CAR-T manufacture and imaging
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
Kevin Carrick, Director, Science, Standards And Global Biologics, United States Pharmacopeia
May 1509:10
Conference pass

Developing an NHS Example Hospital

AI in Healthcare Summit
David Sloman, Chief Executive Officer, Royal Free London NHS Foundation Trust
May 1509:20
Conference pass

Precise genome engineering to empower drug discovery

Gene Editing Summit
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
May 1509:20
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
Jim Fackler, Associate Professor Of Anesthesiology And Critical Care Medicine, John Hopkins School Of Medicine
May 1509:20
Conference pass

TBC: Late preclinical and early clinical updates on Sanofi’s rare disease projects

Gene Therapy for Rare Disease Summit
May 1509:40
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 1509:40
Conference pass

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

Gene Therapy for Rare Disease Summit
  • 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 1510:00
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 1511:00
Conference pass

Synthetic biology for cell and gene therapies presentations followed by panel discussion

Gene Editing Summit
· Moving from autologous to allogenic function · Where are we really headed?
Dr Robert Deans, Chief Technology Officer, Blue Rock Therapeutics
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

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
May 1511:00
Conference pass

Data analytics and AI for Healthcare Transformation

AI in Healthcare Summit
May 1511:00
Conference pass

A true understanding of the biology of stem cells

Cord Blood Workshops
Dr Irene Martini, Lecturer, University La Sapienza
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

Gene editing workshop

Gene Editing Summit
Sponsorship opportunity available. Please contact Erica Baeta (+44 (0)207 092 1152, erica.baeta@terrapinn.com) for further details
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
Alain Vertes, Vice President, Alliance Management, Mesoblast Inc
May 1513:00
Conference pass

Manufacture and bioprocess of CAR-T

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

The EBMR (electronic batch manufacturing record)– product quality in a black box

Standards development process/clinical trials
· Ensuring product delivery, quality control, traceability. · How can we reduce manufacturing man hours and increase efficiency using software?· Focus group to develop software to trace product back to patient
May 1513:00
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
Chris Leonforte, Processing and QC Manager, Anthony Nolan
Daniel Gibson, Head Of Innovation And Commercialation, Anthony Nolan
Irina Evseeva, Head of Specialist Services, Anthony Nolan
May 1513:20
Conference pass

Panel discussion

AI in Healthcare Summit
Goran Nenadic, Group Leader, The Farr Institute’s Health eResearch Centre
David Sloman, Chief Executive Officer, Royal Free London NHS Foundation Trust
Jim Fackler, Associate Professor Of Anesthesiology And Critical Care Medicine, John Hopkins School Of Medicine
May 1514:00
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
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 CAR-T 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
Sven Kili, Vice President And Head Of Gene Therapy Development, Sven Kili Consulting
May 1515:00
Conference pass

AI IN HEALTHCARE WORKSHOP SLOT AVAILABLE

AI in Healthcare Summit
Please contact Erica Baeta (+44 (0)207 092 1152, erica.baeta@terrapinn.com) for further details
May 1515:00
Conference pass

Panel discussion: Tackling the ongoing challenges of research and development of rare disease medicines

Gene Therapy for Rare Disease Summit
  • As an industry how can we position ourselves specifically as small companies?

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May 1608:45
Conference pass

Chair's opening remarks

Keynotes
Vivienne Parry, Head Of Engagement, Genomics England
May 1608:55
Conference pass

Chair's opening remarks

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

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

Keynotes
Interviewer: Vivienne Parry, Head Of Engagement, Genomics England
May 1609:00
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
Dr Joanne Kurtzberg, Director, Carolinas Cord Blood Bank At Duke
May 1609:20
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 1609:30
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
  • How does gene editing apply to the clinic?
  • How is it currently being applied?
  • How can we improve delivery of AAVs into the cell?
  • Improving the volume of viral particles: what stages do we need to cover to get to a commercial scale?
  • Moving into larger diseases from rare diseases, what challenges need to be overcome?
  • Risk/benefit acceptance of the general population and the medical system.
  • What is the real risk of CRISPR causing cancer as published by Novartis and KI?
  • How do we want to use it in the future and what challenges need to be overcome to do so? (immunogenicity, off target effects and efficacy, being vigilant with safety profiles, potency and manufacturing, moving towards direct, in vivo [bluebird bio live delivery usingnanoparticles], viral and non-viral)
  • How do you move towards that goal in a way that engages stakeholders? Applications
May 1609:40
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
Elizabeth Shpall , Director, MD Anderson Cancer Center
May 1610:30
Conference pass

Australian Regenerative Medicine Opportunities

On-Floor Seminar
May 1610:40
Conference pass

Introducing MedCity and the Advanced Therapies Network

On-Floor Seminar
May 1611:00
Conference pass

Cellbox - The Live Cell Shipper Transport at 37°C

On-Floor Seminar
  • Status Quo for shipping cell cultures - Cold chain technologies.
  • From Innovation to Product - The Cellbox portable CO2 incubator as a solution for shipping live cell cultures.
  • The advantages of live cell shipping - A case study of the Cellbox in action.
May 1611:20
Conference pass

Roundtables:

Cord Blood
Amnon Pelz, Chief Executive Officer, Taburit
Chris Bond, Director, Future Health Biobank
May 1611:20
Conference pass

Artificial membrane-binding proteins for cell therapies

On-Floor Seminar
  • The biodistribution of therapeutic cells
  • Artificial membrane-binding protein (AMBP) technology
  • Augmentation of therapeutic cells by AMBPs
May 1611:30
Conference pass

Keynote presentation: Recent Progress in iPS Cell Research and Application

Keynotes
May 1611:50
Conference pass

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

Keynotes
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. How companies can interact with the regulator and various pitfalls to be aware of
Table 11. Purification of stem cells
Stefano Menegatti, Assistant Professor In Chemical And Biomolecular Engineering, North Carolina State University
Table 12. Challenges and opportunities of developing cell therapy in Latin America
Table 15: Making allogeneic cell therapy a reality
Rogerio Vivaldi, Chief Executive Officer, Sigilon Therapeutics
Table 16. Exploring the crossover between human and veterinary applications for stem cells
Joanna Miller, Chief Scientific Officer, Cell Therapy Sciences
Table 17. Roundtable discussion available (If you are interested in being involved, please contact Erica Baeta at: Erica.baeta@terrapinn.com or +44 207 092 1152)
Table 18. Building a diversified portfolio of C&GT medicines
Table 19. How to monitor the safety of viral vectors and other genome modifying therapies in clinical trials?
Table 2. How to manage cell and gene therapy delivery from ‘end to end’
Table 20. Process development and scale-up strategy for a cryo-product in the context of a broad allogeneic cell therapy
Benoit Champluvier, Chief Technology And Manufacturing Officer, Bone Therapeutics
Table 21. Automation and continuous manufacture
Table 22. Using ATMPs to tackle aging
Table 23. Process analytics and control
Table 4. Stem cells for neurological applications
Table 5. MSCs
Alain Vertes, Vice President, Alliance Management, Mesoblast Inc
Table 6. A holistic approach to analytical support, for the biotech industry
Sergey Piletsky, Professor, University of Leicester
Table 7. Small molecule drug discovery to selectively stimulate regenerative processes
May 1612:20
Conference pass

An overview of World Cord Blood day

Cord Blood
Rachel Manley, Head Of Global Outreach, Save the Cord Foundation
May 1612:50
Conference pass

Cytoplasmic membrane re-engineering for next-gen cell therapies

On-Floor Seminar
    Describes a new methodology for the synthesis of novel artificial cell membrane binding proteins.Technology can be readily applied to therapeutic cells to provide tissue homing, adhesion, extracellular matrix formation and hypoxia resistance.Platform is extremely versatile and could be utilized to display virtually any protein on any cell.
May 1613:10
Conference pass

Lunchtime keynote: The Future of Health & Medicine: Where Can Technology Take Us?

Keynotes
From the perspective of a leading physician, scientist, inventor and innovator this presentation examines rapidly emerging, game changing and convergent technology trends and how they are and will be leveraged to change the face of healthcare and the practice of medicine in the next decade.Daniel Kraft offers a fast-paced look at the next few years of innovations in medicine, powered by new tools, tests and apps that bring diagnostic information right to the patient's bedside.A deep dive into where emergent fields such as low-cost personal genomics, the digitization of health records, crowd sourced data, molecular imaging, wearable devices & mobile health, synthetic biology, systems medicine, robotics, artificial intelligence, nanotechnology, 3D printing and regenerative medicine are transforming healthcare, and have the potential to enable clinicians, empower patients, and deliver better care and outcomes at lower cost.
May 1613:10
Conference pass
May 1613:30
Conference pass

Bioengineering Transplantable Whole Organs

On-Floor Seminar
Miromatrix is a biotechnology company engaged in the development of fully biological human organs to solve the chronic shortage of transplantable organs utilizing its patented perfusion decellularization/recellularization technology, as well as the development of the next generation of acellular based products.
Jeff Ross, Chief Executive Officer, Miromatrix
May 1613:40
Conference pass

Challenges with advanced therapies in clinical trials

On-Floor Seminar
May 1613:50
Conference pass

BioRN initiatives in leveraging the unique combination of global pharma and leading academic institutions in the region

On-Floor Seminar
  • HAEP – a new instrument for facilitated technology transfer between SMEs, academia and global healthcare companies
  • Health Axis Europe critical mass: a large international network of innovators
  • The vibrant Rhine/Main/Neckar life science region
May 1614:00
Conference pass

A new 3D cell culture platform (encapsulation) led to unprecedented population doubling (> x100/passage) while improving genetic stability and cell differentiation (functional neurons)

On-Floor Seminar
  • C-Stem™ encapsulation, cultivation and formulation platform enabled:
  • Pluripotent Stem Cell amplification factor of >100 x per passage
  • Improved iPSC genetic stability after high number of doubling
  • Dramatic improvement of neuron functionalization demonstrated on animal model
  • Scalable differentiation of iPSC into numerous cell/tissue type (cardiomyocytes, chondrocytes, gut crypt)
May 1614:10
Conference pass

Chair's opening remarks

Clinical Trials: Cell Therapy
May 1614:10
Conference pass
May 1614:10
Conference pass

Chair's opening remarks

Pitch and Partner
Vivienne Parry, Head Of Engagement, Genomics England
May 1614:10
Conference pass

Cord blood expansion for engraftment and regenerative medicine

Cord Blood
  • Current state of the art for cord blood expansion in the transplant setting
  • MSCs for regenerative medicine and GVHD
  • Will discuss the GMP production of cord blood tissue derived MSCs for clinical use
Elizabeth Shpall , Director, MD Anderson Cancer Center
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

Getting genetic therapies from the bench to the bedside: Meeting the challenges of commercialisation

Gene Modified Cell Therapy
· What are the biggest challenges to making gene therapy commercially viable· What system change is needed· What have we learned as an industry so far
May 1614:15
Conference pass
May 1614:15
Conference pass

TBC: 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? 
May 1614:15
Conference pass

Case study: building an in-house baculovirus 200l GMP manufacturing facility

Viral Vector Manufacturing
  • Volume/application vs safety
  • Haemophilia A/B
  • How do you tackle the high treatment burden?
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?
May 1614:30
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 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

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

Gene Modified Cell Therapy
Speaking opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, erica.baeta@terrapinn.com) for further details)
May 1614:35
Conference pass

Panel discussion: Delivering on the Promise of Gene Therapy 

In Vivo Gene Therapy
Making genetic therapies a reality for patientsAttendees 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

Viral Vector Manufacturing
Paul Carter, Cell & Gene Therapy Platform CMC, GlaxoSmithKline
May 1614:50
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

How the allogeneic approach will make cell therapies accessible for more patients through gene editing

Gene Modified Cell Therapy
May 1614:55
Conference pass

Are we asking the right questions to achieve deliverable advanced therapies? Lessons from the past and the challenges of the future

Cell Therapy Manufacturing
  • An overview of almost forty years of cell therapeutics development
  • Establishing the potential of cell-based therapies, enabling technologies and organizations
  • Developing an applicable regulatory framework and guidance and a better appreciation of drug development principles
  • Themes of the present and future as we advance deliverability of cell-based therapies
May 1614:55
Conference pass

TBC: Suspension Vero cell line for production of viral vaccines and viral therapeutics

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
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
Gareth King, Manager, Catapult Ventures Group
May 1615:10
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 1615:45
Conference pass

The Rise of CAR-T: Ensuring Access to Transformative Cancer Therapies

Pricing and Market Access: Value, Pricing, Affordability
The rise of immuno-oncology, most recently CAR-T, represents a new generation of transformative cancer therapies offering hope for the patients of today and tomorrow. However, with this revolutionary approach to treating cancer, there are potential hurdles to patient access that must be acknowledged and overcome. This session will focus on the transformative potential of CAR T as well as key challenges ahead for patients, providers, payers, and innovators. Topics will include: value and cost (to the patient and the healthcare system), coverage and reimbursement, and the potential for innovative payment arrangements.
May 1615:45
Conference pass

Using immunotherapy to target HIV

Gene Modified Cell Therapy
May 1615:45
Conference pass

Case study: TIGEM’s gene therapy projects

In Vivo 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
  • Market analytics will be presented to identify Industry Strengths, Weaknesses, Opportunities and Threats
  • Industry weaknesses and opportunities are described
  • A comprehensive view of future 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 1616:00
Conference pass

Best practice in successfully commercializing an academic spin-out company

On-Floor Seminar
  • Guidance for both the investor and University side at this early spin-out and seed investment stage to try and help each side understand the other’s perspectives.
  • Best practices from both investor and academic perspectives
  • Suggested advice of how to bridge the different starting points
May 1616:05
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 1616:05
Conference pass

Translating in vivo genome editing capabilities into therapeutics

In Vivo Gene Therapy
  • Optimizing the nuclease to ensure in vivo specificity
  • Determining the best delivery system
  • Evaluating appropriate disease models
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
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
May 1616:05
Conference pass

Viral vector manufacturing case study

Viral Vector Manufacturing
May 1616:15
Conference pass
May 1616:25
Conference pass

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

Pricing and Market Access: Value, Pricing, Affordability
Carla Deakin, Programme Director – Commercial & Managed Access, NICE
Nick Medcalf, Innovation Lead - Advanced Therapies, Innovate UK
May 1616:25
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 tumourswith 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 1616:25
Conference pass

The road to restoring sight in patients with rare inherited retinal diseases

In Vivo Gene Therapy
Phase I & II clinical trials data using gene therapy for retinal diseases
Rabia Ozden, Chief Medical Officer, Nightstar Therapeutics
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?
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
  • As cell-based cancer immunotherapies move from the research to a commercialization stage there is a need to establish a reproducible manufacturing process.
  • Cell culture media is a crucial part of the immunotherapy manufacturing process and discovery process.
  • The use of defined media for cell culture helps meet the challenges currently faced in the cancer immunotherapy field. In the drug discovery part, one needs a media that allows for reproducibility of results, while providing easiness of use and some flexibility of application. In cell therapy the challenge resides in the ability to generate a high number of clinical grade cells consistently.
  • Fujifilm Irvine Scientific has developed chemically defined and serum- free media solutions for drug discovery and ex-vivo expansion of the main cell types used in immunotherapy (Cancer Initiating Stem Cells, T Cells, NK and dendritic cells), as a part of the PRIME XV Cell Therapy product line. Serum-free and chemically-defined media are free of sera (animal or human derived) providing an extra level of safety, consistency and quality, by removing undefined and possible major health treats (such as viruses and prions).
  • In this presentation we will introduce our PRIME XV portfolio for cancer immunotherapy and discuss the benefits of a defined media solution.
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 1616:45
Conference pass

Successfully developing and commercialising a gene therapy

In Vivo Gene Therapy
Speaking opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, erica.baeta@terrapinn.com) for further details
May 1616:45
Conference pass

A cell culture GMP facility of the future implemented in the present

Cell Therapy Manufacturing
Reserved for COMECER and CO.DON AG
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

Reserved for sponsor

Cord Blood
If you are interested in being involved, please contact Erica Baeta at:Erica.baeta@terrapinn.com or +44 207 092 1152
May 1617:00
Conference pass

Presentation

Pricing and Market Access: Value, Pricing, Affordability
Reserved for PRMA Consulting
May 1617:00
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 1617:00
Conference pass

Quality systems for regenerative medicine delivery: basic quality requirement for distribution

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 1617:00
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

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

In Vivo 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, CTO, Axovant Sciences
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 
Paul Carter, Cell & Gene Therapy Platform CMC, GlaxoSmithKline
May 1617:10
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 1617:15
Conference pass

Phase III clinical data

Clinical Trials: Cell Therapy
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

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
Michèle De Guise, Director of Health Services and Technology Assessment, Inesss
Charles De Cidrac, Director Of Health Insurance, AXA
Walter Van Dyck, Associate Professor, Vlerick Healthcare Management Centre
Katja Schmidt, Head of Market Access and Public Affairs, Novartis
Carla Deakin, Programme Director – Commercial & Managed Access, NICE
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

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 1617:45
Conference pass

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

Gene Modified Cell Therapy
May 1617:45
Conference pass

Panel discussion: 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
May 1617:45
Conference pass

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: Bioprocessing 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

Pitch 9.

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

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

Title TBC

Cord Blood
Machi Scaradavou, Medical Director of the National Cord Blood Program, New York Blood Center
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 1711:05
Conference pass

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

Pricing and Market Access: Approval and Evidence
May 1711:05
Conference pass

The changing paradigm for cell therapy

Cell Therapy and Regenerative Medicine
  • How we access partnerships through technology, process development and manufacturing
  • Shifts challenges and opportunities
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

Light distilleries of cells and proteins

On-Floor Seminar
  • Proteins and cells are bound on the adsorbent in dark and are recovered by applying light at a specific wavelength
  • This is a very innovative and safe manner to purify therapeutics that are so biochemically delicate that current purification strategies fail. We call this technology "Light distilleries of cells and proteins
Stefano Menegatti, Assistant Professor In Chemical And Biomolecular Engineering, North Carolina State University
May 1711:25
Conference pass

Breaking into the US market and overcoming regulatory hurdles

Pricing and Market Access: Approval and Evidence
Reserved for Cardinal Health
May 1711:25
Conference pass

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

Immunotherapy
May 1711:25
Conference pass

Clinical trial updates new data and strategies

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 1711:25
Conference pass

Opening presentation

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

TBC: Getting it right first time, viral vector production from end to end

Viral Vector Manufacturing
Presentation from Pall
May 1711:30
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 1711:40
Conference pass

The AUTOSTEM PROJECT

On-Floor Seminar
Mary Murphy, Principle Investigator, Remedi National University of Ireland Galway
May 1711:45
Conference pass

Using Gamma-Delta T-Cells for Immuno-oncology applications

Immunotherapy
  • Using V Delta 2 T-Cells to sense and react to metabolic tumors
  • Using viral vectors to create a catalytic event via T-Cell receptor and kill like an NK Cell
  • Broad spectrum for tumor application and sensitive to tumor inhibition
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

Building a ‘smart city’ for cell and gene

Cell Therapy Manufacturing
  • Concept and design
  • GMP space building
  • Prefab space
  • Commercial GMP & vector production
  • Patient facing/hospital scenario
May 1711:45
Conference pass

TBC: Technology for safe and effective viral vector manufacture

Viral Vector Manufacturing
Reserved MolMed
May 1711:50
Conference pass

Case study of one of the largest cord blood banks in Europe

Cord Blood
  • Success story and challenges met along the way
  • Research updates
Tomasz Baran, Board Member, PBKM/FamiCord Group
May 1712:00
Conference pass

Composite ceramic monolithic columns for chromatographic separations

On-Floor Seminar
  • Flow properties
  • The challenge of functionalization
  • Impact on biomolecule downstream process
May 1712:05
Conference pass

Case study: building a cell therapy business in Japan

Pricing and Market Access: Approval and Evidence
May 1712:05
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 1712:05
Conference pass

Platform technology for regenerative medicine cell therapy, stem cells, immune-oncology

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 1712:05
Conference pass

Personalisation of Research

Clinical Development: Gene Therapy
May 1712:05
Conference pass

Optimizing CGT manufacturing workflows for industrial scale manufacturing”

Cell Therapy Manufacturing
  • Miltenyi Biotec’s generic GMP CAR T Manufacturing protocol as a model for complex CGT manufacturing
  • Ways towards efficient large-scale processing
  • TechTransfer concepts for further scale out
May 1712:05
Conference pass

Improving safety and efficacy of viral vector manufacture

Viral Vector Manufacturing
If you are interested in being involved, please contact Erica Baeta at:Erica.baeta@terrapinn.com or +44 207 092 1152
May 1712:10
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 1712:25
Conference pass

Panel focusing on Japanese regulation and commercial ecosystem

Pricing and Market Access: Approval and Evidence
May 1712:25
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?
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
This panel will examine and discuss the ways regenerative medicine is being revived in the biotech industry and how some companies are using these new techniques and platforms to create better, faster, safer and potentially curative treatment options for a multitude of disease indicationsKey topic: Moving from autologous to fully allogeneic cell therapy
May 1712:25
Conference pass

New clinical data: UK Cystic Fibrosis gene therapy program and CRM

Clinical Development: Gene Therapy
May 1712:25
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 1712:25
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?
  • Consistency: How do you define your critical quality attributes and assays, so you know each batch is the same?
May 1712:30
Conference pass
May 1712:30
Conference pass

3D-print your own chromatographic column

On-Floor Seminar
  • Development of new materials compatible with downstream operations and 3D printing technology
  • Demonstration of protein capture from cell culture supernatant
  • Opportunities and challenges of 3D printed chromatography columns
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

Engineering for cell therapy manufacturing

Cell Therapy Manufacturing
If you are interested in being involved, please contact Erica Baeta at: Erica.baeta@terrapinn.com or +44 207 092 1152
May 1713:50
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 1713:50
Conference pass

Next generation technologies: Implantable robotics, a rapidly moving and exciting field that dovetails with advances in tissue engineering and biomaterials

On-Floor Seminar
May 1714:00
Conference pass

Inside the ethics committee: Ethics behind CRISPR and gene edited therapeutics

On-Floor Seminar
Vivienne Parry, Head Of Engagement, Genomics England
May 1714:05
Conference pass

Starting materials and standards in relation to advanced therapies

Pricing and Market Access: Approval and Evidence
May 1714:05
Conference pass
May 1714:10
Conference pass

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

Immunotherapy
New clinical data for 3 immune-oncology clinical trials for ovarian, glioblastoma and melanoma treatment
May 1714:10
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
Ross Macdonald, Chief Executive Officer, Cynata Therapeutics Ltd
Katarina Le Blanc, Professor/Specialist Physician, Division Of Clinical Immunology, Karolinska Institutet
May 1714:10
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 1714:10
Conference pass

Moving from artisanal to robust and broad manufacturing models

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 1714:10
Conference pass

Designing your viral vector manufacturing facility with the end in mind

Viral Vector Manufacturing
If you are interested in being involved, please contact Erica Baeta at:Erica.baeta@terrapinn.com or +44 207 092 1152
May 1714:10
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 1714:25
Conference pass

US Cell & Gene Therapy Regulatory Policy Update

Pricing and Market Access: Approval and Evidence
Recent or planned updates to the regulatory framework for cell and gene therapy products and experience with the new RMAT (Regenerative Medicine Therapy Designation) will be reviewed and discussed.
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

Developing a viral vector therapeutic for PKU

Clinical Development: Gene Therapy
May 1714:30
Conference pass

Plenary with Viral Vectors Manufacturing: 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 Cell Therapy 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?
Ohad Karnieli, CEO, Atvio Biotechnology
May 1714:30
Conference pass

Success from a phase I clinical trial: cord blood derived mononuclear cells for the treatment of hypoplastic left heart syndrome

Cord Blood
  • What is HLHS?
  • Pre-clinical development using UCB-MNCs in small and large animals
  • Phase I clinical study and results
May 1714:50
Conference pass

Tools and technologies for immunotherapy development

Immunotherapy
Speaking opportunity available Please contact Erica Baeta (+44 (0)207 092 1152, erica.baeta@terrapinn.com) for further details
May 1714:50
Conference pass

Collaborative phenotyping at King’s: the stem cell hotel

Cell Therapy and Regenerative Medicine
May 1714:50
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 1714:50
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 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 1715:10
Conference pass

Brexit: the potential policy, regulatory and public affairs outcomes of Brexit and the possible implications for the life science industry

On-Floor Seminar
Pharmaceutical companies continue to plan for the UK’s exit from the EU. As they do, the terms on which the UK will leave - and the terms of the future relationship – remain uncertain
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

Cell therapy for neurorepair

Clinical Development: Gene Therapy
Pascale Guillot, Senior Lecturer Of Fetal And Maternal Health, University College London
May 1715:40
Conference pass

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

Cell Therapy Manufacturing
May 1715:40
Conference pass

Highlighting and addressing ‘critical path’ issues in bringing vector manufacturing processes from Phase I/II to commercial scale

Viral Vector Manufacturing
If you are interested in being involved, please contact Erica Baeta at:Erica.baeta@terrapinn.com or +44 207 092 1152
May 1716:00
Conference pass

What is going on in Quebec about Market Access?

Pricing and Market Access: Approval and Evidence
  • Examining Quebec life sciences strategy: Integration of medicines and technological innovations
  • Achieving cost reduction and improving access to medicines
  • Re-editing the evaluation process for the introduction of innovative medication and technologies
Luc Boileau, Président-Directeur General, Inesss
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? 
Katarina Le Blanc, Professor/Specialist Physician, Division Of Clinical Immunology, Karolinska Institutet
May 1716:00
Conference pass

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

Clinical Development: Gene Therapy
May 1716:00
Conference pass

BIOPROCESSING CLOSING PLENARY PANEL: 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?
Ioannis Papantoniou, Atmp Bioprocessing Coordinator, KU Leuven
Mary Murphy, Principle Investigator, Remedi National University of Ireland Galway
May 1716:00
Conference pass

BIOPROCESSING CLOSING PLENARY PANEL: 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?
Ioannis Papantoniou, Atmp Bioprocessing Coordinator, KU Leuven
Mary Murphy, Principle Investigator, Remedi National University of Ireland Galway
May 1716:00
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 1716:20
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
  • Preparation for market access
  • Planning early in the clinical trials process to avoid failure later on in the development process
  • Improving the awareness of working with the clinical centre
  • Supply term agreement with hospital and insurance providers
  • Framework administration to provide expanded access and clinical trials
  • Being aware & working well in advance on quality agreements
  • Maintaining chain of custody (both ways) of samples and quality control during transfer of clinical samples
  • Tackling the reluctance to think about this planning early on
May 1716:20
Conference pass

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 1717:00
Conference pass

Chair's closing remarks

Cord Blood
May 1717:05
Conference pass

Close of conference

Cord Blood
last published: 18/Mar/19 11:45 GMT

 

 

There are two parts to the World Advanced Therapies & Regenerative Medicine Congress: a conference and an exhibition featuring solutions for pharma and biotech.

There is a registration fee to attend the conferences. To visit the exhibition is free.

TO SPONSOR / EXHIBIT

Erica Baeta 
+44 (0)207 092 1152 
erica.baeta@terrapinn.com

TO SPEAK

Jessica Robinson 
t/ +44 (0)207 092 1150 
jessica.robinson@terrapinn.com

TO REGISTER

Issa Mauthoor 
+44 (0)207 092 1257 
issa.mauthoor@terrapinn.com