Stem Cells & Cell Therapy Summit Agenda

Summit Registration and Materials Pick-up

Afternoon One of the Summit: Market Analysis and Round-Table Discussion
Enal Razvi, Managing Director, Select Biosciences Inc, United States of America

In this presentation, I will provide a detailed market analysis of the cellular therapy space addressing the following topics:

1. Adult Stem Cells Market Segmentation, Quantitative Market Metrics [market sizing, growth rate], Technology Analysis.

2. Competitive and SWOT Analysis of Industry Participants.

3. Clinical Trials Analysis of Adult Stem Cells Space.

4. Cord Blood Market Landscape: Segments of Utility, Emerging Trends, Disease Classes Addressed, Quantitative Market Analysis

5. Round-Table Discussion.

Description of the Challenges and Opportunities Related to the Clinical Development and Commercialization of Allogeneic Stem Cells in Orphan Disease Market. Illustration with the Practical Case of the Belgian Cell Therapy Company Promethera Biosciences
Philippe Willemsen, Senior Manager, Promethera Biosciences SA, Belgium

Promethera® Biosciences' mission is to discover, develop and commercialize cell therapy products to treat liver diseases in an innovative way using stem cells from healthy human livers.  Promethera® Biosciences develops a new cell therapy product using allogeneic stem cells expanded from healthy human liver tissue. This product called Promethera® HepaStem aims to treat a wide variety of liver genetic and acquired diseases affecting children and adults. The innovation resides in both the simplicity of the treatment, which doesn’t require radical surgery (compared to liver transplantation), and in the wide variety of liver pathologies that can be addressed with the same product. This product has received two orphan drug designations from the European Community and the FDA for the treatment of Crigler-Najjar syndrome and urea cycle disease. In conjunction, Promethera® Biosciences is developing, Promethera® HepaScreen, a unique cell model for the pharmaceutical industry to mimic metabolism and detoxification of new drugs by the human liver. Both products are based on newly discovered and patented stem cell types. Eric Halioua, CEO of Promethera Biosciences, will illustrate the opportunities and challenges in terms of commercialization for an allogeneic stem cell product.

Developing a Next Generation MultiStem Product; a Xeno-free Formulation and Alternative Methods for Production and Scale-up
Jef Pinxteren, Manager & Head, Regenesys, Belgium

ReGenesys is a biopharmaceutical company focused on the research, development and commercialization of stem cell-based therapies and technologies. MultiStem® cells are bone marrow-derived non-hematopoietic adherent stem cells with a large expansion capacity and a remarkable biological plasticity. MultiStem cells have the potential to home and integrate into damaged tissues and provide immunomodulatory, angiogenic and other effects by cell-cell contact and paracrine regulation. Therefore, there is growing interest in the use of MultiStem therapy. The MultiStem product is an allogeneic “off the shelf" stem cell product. Like mesenchymal stem cells (MSC), allogeneic MultiStem cells are non-immunogenic and exert immunosuppressive effects in vitro. On the other hand, MultiStem cells show alterations in phenotype, differences in gene and protein expression and have an extensive proliferation capacity as compared to MSC. MultiStem cells are currently being used in mid-stage clinical trials for ischemic stroke (Phase II) and ulcerative colitis (Phase II). Two phase I trials were earlier concluded successfully (GVHD and AMI) and other trials are being prepared, e.g. in solid organ transplantation.

Coffee and Networking

Clinical Grade iPS Cell Manufacture by Cellectis
Edward Balbirnie, Head, Cellectis, France

Cellectis, via it's 'iPS Engineering HubTM ' has developed the capability to produce clinical grade iPS cells using GMPs which could be used as starting material for future iPS-based therapeutics.

Legal Framework for Stem Cells and Cell Therapy in Europe
Marc Martens, Senior Associate, Bird & Bird LLP, United Kingdom

The presentation will provide a view on the current regulatory state of play for the use of stem cells and the development and marketing of advanced therapies.

- Stem cells used for medical applications

o Directives on quality and safety standards

o Ethical neutrality of the directive: consequences

- Cell therapies and other advanced therapies

o ATMP regulation : principles and scope

o Articulation with the directives on quality and safety standards

o Hospital exemption : scope and diverging national implementation

- Research on stem cells and cell therapy : overview of divergences in national approaches

Lunch

A Day in the Life of a Contract Manufacturer: Together for Happier Cells
Luca Romagnoli, Business Development Manager, Areta International, Italy

Today’s contract manufacturers are expected to deliver excellence and be prepared to address a wide range of challenges that goes well beyond the process development and regulatory aspects. An open and fruitful collaboration with the developer is fundamental to facilitate clinical translation and, ultimately, determine the success of the product.

ATMPs: A Regulatory Route-map
Alison Wilson, Principle Consultant, CellData Services, United Kingdom

The field of cell therapy covers a huge range of concepts and therapeutic approaches. Many of these are at early stages in development, and are frequently associated with clinician-led academic groups and small spin-out companies rather than large pharmaceutical companies. The regulatory frameworks surrounding the clinical development, manufacture and approval for marketing of cell therapies are complex and may be outside the expertise of the early development teams in small organisations. The key to designing an appropriate development programme that will deliver all of the necessary data for clinical use and ultimately commercialisation of a cell therapy product is a clear understanding of how the product will be regulated.

This presentation will discuss the broad concepts that determine whether a cell therapy product will be regulated under the Advanced Therapy Medicinal Product (ATMP) Regulation, and then give an overview of the specificities of ATMP requirements within the EU’s medicinal products regulatory framework. Guidance will be given on the types of advice and support available for development of cell therapies within the ATMP regulation, and the key development stages at which scientific advice should be considered.

Coffee and Networking

Cardiac Atrial Appendage Stem Cells Restore Cardiac Function in a Minipig Infarction Model
Remco Koninckx, Post-doc, Jessa Hospital, Belgium

Researchers in the Jessa Hospital in Belgium recently discovered a new cardiac stem cell (CSCs) population with superior differentiation potential compared to other CSCs. They now successfully investigated the functional characteristics of these cells in the minipig infarct model.

PHD2 Silencing Enhances the Survival and Paracrine Function of Transplanted Adipose-Derived Stem Cells in Infarcted Myocardium
Wei (Eric) Wang, Attending Doctor, Daping Hospital, Third Military Medical University, China

Silencing prolyl hydroxylase domain protein 2 (PHD2) promotes the survival of transplanted adipose-derived stem cells (ADSCs) in infarcted hearts and enhances their paracrine function. The promoted survival was HIF-1alpha dependent, while the enhanced paracrine function was associated with NF-kB signaling.

Intranasal Delivery of Therapeutic NSCs to Target Intracerebral Glioma: Enzyme/Prodrug CE/CPT-11 Therapy
Margarita Gutova, Assistant Research Professor, City of Hope National Cancer Center, United States of America

Despite aggressive multimodal therapy and advances in imaging, surgical and radiation techniques, high-grade gliomas remain incurable, with survival often measured in months. Treatment failure is largely attributable to the diffuse and invasive nature of glioma cells, ineffective delivery of chemotherapeutic agents across the blood-brain barrier (BBB), and associated dose-limiting systemic toxicities. Neural stem cells (NSCs) display inherent tumor-tropic properties that can be exploited for targeted delivery of anti-cancer agents to invasive and metastatic tumors, and may offer an unprecedented advantage over conventional therapeutic approaches. NSCs can overcome the major obstacles limiting the efficacy of current treatments by their ability to cross the BBB, target therapeutic agents to primary and invasive tumor foci throughout the brain, and minimize toxicity to normal tissues. Used as a delivery vehicle, NSCs have been engineered to express a variety of anti-cancer agents, demonstrating >70% therapeutic efficacy in pre-clinical models of glioma, medulloblastoma, melanoma brain metastases and disseminated neuroblastoma.  Cell based therapies for neurodegenerative diseases depend on efficient delivery of the therapeutic cells to the areas of damage. Administration of NSCs intracranially may cause damage of normal tissues and demonstrate poor engraftment into the brain.  Alternatively, when stem cells are injected intravenously immunological reaction and other systemic complications may occur.  Here we demonstrate, for the first time, tumor-specific NSC tropism of therapeutic human neural stem cells (HB1.F3.CDs) to human glioma xenografts in mouse models.  In this study, severely immunodeficient Esl1 were implanted intracranially with U251.eGFP.ffluc glioma cells.  After 15 days, NSCs were administered intranasally and biodistribution of the stem cells was determined via MR and histological imaging.  

Close of Summit