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Stem Cell Research & Regenerative Medicine

2017-09-192017-03-092018-02-13
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2017 Agenda
Day 1 - Wednesday, April 5th, 2017
7:30
Continental Breakfast & Registration
9:00
Opening Remarks
10:00
Morning Networking Break
Advances in Adult & Pluripotent Stem Cell Research
Moderator: Anthony Ting, Athersys
10:30
Healios' Pipelines and Strategy toward Commercializations of iPS Cell
 
Hardy T S Kagimoto
Hardy T S Kagimoto
President and Chief Executive Officer
Healios K.K (RIKEN Venture)
About Speaker: Hardy TS Kagimoto, MD is a serial entrepreneur and obsessed with the concept of curing as many patients as possible through technology. At the first biotech, Aqumen, he launched BBG which gained de-facto-standard status in ophthalmology community, gl... Read Full Bio 
 
 
Hardy T S Kagimoto
President and Chief Executive Officer
Healios K.K (RIKEN Venture)
 
About Speaker:

Hardy TS Kagimoto, MD is a serial entrepreneur and obsessed with the concept of curing as many patients as possible through technology. At the first biotech, Aqumen, he launched BBG which gained de-facto-standard status in ophthalmology community, globally. And then he founded Healios with the vision to create a world leading regenerative cell therapy company.   He brought Healios public in 2015 and currently running P2/3 trials for acute brain stroke in Japan with somatic stem cell’s immune moderation effect to reduce secondary damage of acute brain stroke. Healios also holds various iPS pipelines which is under clinical research to gain proof of concept with human. He is happily living in Tokyo, Japan, with his British wife, 3 children, 2 turtles, 1 tank of tropical fish and various plants.

 
Abstract: Healios would introduce its corporate strategy and pipelines to enable cell therapy in the light of Japanese accelerated approval system. 1...Read More 

Healios would introduce its corporate strategy and pipelines to enable cell therapy in the light of Japanese accelerated approval system.

1st pipeline: Bone marrow derived Somatic stem cell pipeline, which Healios is conducting randomized, double blinded 110 vs 110, P2/3 study for acute brain stroke.

2nd pipeline: iPS based RPE cell, which Riken started world first clinical research using autologous iPS.  It’s been 2 years and 6 months and the patient is free from any anti-VEGF injections for the eye she had implantation but the fellow eye still requires constant injections.  Recently, Riken has gained approval from ministry of health in Japan to start world first clinical research using allogeneic haplo bank iPS cell lines for those who are suffering from wet Age-related Macular degeneration.  Healios will be presenting the consistent flow from clinical research to clinical trials to gain approval for iPS cell based therapy.

3rd pipeline: Healios’s another focus is organ bud technology which is proprietary technology which enables to generate various type of organs.  With Yokohama City University, Healios is planning to start world first clinical research in 2019 for patients suffering from congenital lever enzyme deficiency.

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10:55
The Development of Pluripotent Stem Cell-based Therapies for Regenerative Medicine
 
Erin Kimbrel
Erin Kimbrel
Senior Director of Development
Astellas Institute for Regenerative Medicine
About Speaker: Erin Kimbrel is the Senior Director of Development at Astellas Institute for Regenerative Medicine (AIRM), an indirect, wholly-owned subsidiary of Astellas Pharma located in Marlborough, Massachusetts, USA. Dr. Kimbrel has nearly 15 years of experien... Read Full Bio 
 
 
Erin Kimbrel
Senior Director of Development
Astellas Institute for Regenerative Medicine
 
About Speaker:

Erin Kimbrel is the Senior Director of Development at Astellas Institute for Regenerative Medicine (AIRM), an indirect, wholly-owned subsidiary of Astellas Pharma located in Marlborough, Massachusetts, USA. Dr. Kimbrel has nearly 15 years of experience in the stem cell field, including several years in industry where she has been engaged in research and development of various hESC and iPSC-derived cell-based therapies. She was previously the Director of MSC research at Advanced Cell Technology and Director of Translational Research/Sr. Director of Development at Ocata Therapeutics before the company was acquired by Astellas. She is a co-author on over 20 publications and an inventor on numerous patents. Dr. Kimbrel is a former Fulbright scholar and member of Phi Beta Kappa, receiving her B.A. Magna Cum Laude from Holy Cross College in Worcester, Massachusetts and her Ph.D. in Pharmacology and Cancer Biology from Duke University in Durham, North Carolina. She completed post-doctoral training in Boston, Massachusetts at the Dana-Farber Cancer Institute and Harvard Medical School with a focus on hematopoietic stem cell fate decisions. Her current role at AIRM involves the strategic planning of pipeline programs as well as managing scientists involved in regenerative medicine research and early product development.

 
Abstract: Pluripotent stem cells (PSCs) have important clinical potential in regenerative medicine given their ability to differ...Read More 

Pluripotent stem cells (PSCs) have important clinical potential in regenerative medicine given their ability to differentiate into any cell type in the body. AIRM is focused on developing PSC-derivatives as therapeutic cell-based therapies for diseases with high unmet need. Our most advanced product, human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) is a clinical-stage product for age-related macular degeneration and Stargardt disease, an inherited juvenile form of macular degeneration. Other cell-based ophthalmologic therapies in development include PSC-derived photoreceptor progenitors to treat retinitis pigmentosa and macular degeneration, retinal ganglion progenitors to treat glaucoma and optic neuropathies as well as corneal endothelium to treat diseases and injuries of the cornea. We are also exploring cell-based therapies for indications outside the eye. Vascular progenitors have been generated from PSCs and are being investigated for the treatment of indications such as critical limb ischemia and pulmonary hypertension. Additionally, we have differentiated PSCs into mesodermal precursors called hemangioblasts and used them to generate mesenchymal stem cells. These hemangio-derived mesenchymal cells (HMCs) have been found to possess immunomodulatory and/or tissue-protecting effects in preclinical studies of lupus nephritis, Crohn’s disease, multiple sclerosis, and autoimmune uveitis. We will highlight our efforts in each of these programs and discuss critical issues that should be considered when developing any type of PSC-derived therapy for clinical use.

Key points:

  1. Pluripotent stem cells are a key starting material for the development of off-the-shelf cell-based therapies and regenerative medicine.
  2. Ophthalmology has been a popular first therapeutic area for clinical application of PSC-based therapies yet PSC derivatives are being developed in a variety of areas outside the eye.
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11:20
Regenerative Therapies for Cardiovascular and CNS Degenerative Disease
 
Robert Deans
Robert Deans
Chief Technology Officer
BlueRock Therapeutics
About Speaker: ... Read Full Bio 
 
 
Robert Deans
Chief Technology Officer
BlueRock Therapeutics
 
About Speaker:
11:45
MultiStem for Acute Injury
 
Anthony Ting
Anthony Ting
Vice President
Athersys
About Speaker: Dr. Ting is the Vice President of Regenerative Medicine and Head of Cardiopulmonary Programs. With more than thirty years of experience in cell and stem cell biology, Dr. Ting has developed expertise in translational clinical studies with adult stem ... Read Full Bio 
 
 
Anthony Ting
Vice President
Athersys
 
About Speaker:

Dr. Ting is the Vice President of Regenerative Medicine and Head of Cardiopulmonary Programs. With more than thirty years of experience in cell and stem cell biology, Dr. Ting has developed expertise in translational clinical studies with adult stem cell therapies and has been responsible for all stages of the development of MultiStem® from the bench to the bedside. Dr. Ting manages all programs in the cardiovascular and pulmonary areas at the Company, as well as the evaluation of potential new uses for the cell therapy product. Dr. Ting serves on several regenerative medicine society committees including the International Society for Cell Therapy, the Alliance for Regenerative Medicine and the American Society for Gene and Cell Therapy. From 1995 to 2001, Dr. Ting was a Principal Investigator and Head of the Screening for Novel Inhibitors group at the Institute of Molecular and Cell Biology (IMCB) at the National University of Singapore. Prior to joining IMCB, he was a post-doctoral fellow in the department of Molecular and Cellular Physiology at Stanford University. Dr. Ting received his Ph.D. in Cell Biology from Johns Hopkins University and his B.A. in Biology from Amherst College.

12:10
Lunch Workshop – Speaker: Judith Gorski, Crown Bioscience
12:10
Translational Platforms to Model Pre-Diabetes, Diabetes, and Diabetic Complications
 
Judith Gorski
Judith Gorski
Global Director, Scientific Engagement
Crown Bioscience Inc.
About Speaker: Dr. Judith Gorski is Global Director of Scientific Engagement at Crown Bioscience Inc., and is a pharmacologist with over 18 years of experience in drug discovery and development in a large pharmaceutical environment.  She has extensive experience i... Read Full Bio 
 
 
Judith Gorski
Global Director, Scientific Engagement
Crown Bioscience Inc.
 
About Speaker:

Dr. Judith Gorski is Global Director of Scientific Engagement at Crown Bioscience Inc., and is a pharmacologist with over 18 years of experience in drug discovery and development in a large pharmaceutical environment.  She has extensive experience in basic research and targeted drug discovery in the disease areas of type 1 and 2 diabetes, dyslipidemia, atherosclerosis, obesity, and metabolic syndrome.  Dr. Gorski has co-authored publications in Nature, Obesity, Obesity Research, Cell Metabolism, Journal of Lipid Metabolism.

Prior to joining CrownBio, Dr. Gorski’s work at Merck focused on target identification/validation and small molecule and biologics identification/optimization with the aim of recommending lead candidates for clinical development. Her primary responsibilities were to establish and validate appropriate primary, secondary and tertiary in vivo models of dyslipidemia, obesity, metabolic syndrome, and type 1 and 2 diabetes. In addition to preclinical work, Dr. Gorski has been a member of early development teams to move lead candidates into development and has collaborated across an array of disciplines, including drug safety and metabolism, pharmaceutical sciences, early clinical development, medical affairs, regulatory affairs, and commercial development.

Dr. Gorski received her Ph.D. in Neuroscience from University of Medicine and Dentistry, New Jersey.

 
Abstract: A key challenge in preclinical studies of diabetes is the lack of translational platforms that can model the various aspects of disease progression...Read More 

A key challenge in preclinical studies of diabetes is the lack of translational platforms that can model the various aspects of disease progression and associated complications.  In this talk, Dr. Gorski will present the development and utility of CrownBio’s unique continuum of translational platforms that model various aspects of diabetes and metabolic syndrome, and how such platforms are used to predict the efficacy and safety of anti-diabetic therapies in humans.  Attendees will learn about:

  • The latest rodent and Non-Human Primate translational models of to study diabetes pathology
  • Modeling prediabetes, diabetes progression, and associated complications (e.g., nephropathy) in these models;
  • Examples of how these models are used in anti-diabetes drug development
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Revolution in Immunotherapy
Moderator: Jane Lebkowski, Asterias Biotherapeutics
1:25
Path to Universal "off the shelf" Engineered T Cell Therapy
 
Margo Roberts
Margo Roberts
Sr VP, Discovery Research
Kite Pharma
About Speaker: Dr. Roberts has more than two decades of biomedical research, drug discovery and development experience. From 1999 to 2013, Dr. Roberts held an Associate Professor position at the University of Virginia where she pursued interdisciplinary research in... Read Full Bio 
 
 
Margo Roberts
Sr VP, Discovery Research
Kite Pharma
 
About Speaker:

Dr. Roberts has more than two decades of biomedical research, drug discovery and development experience. From 1999 to 2013, Dr. Roberts held an Associate Professor position at the University of Virginia where she pursued interdisciplinary research in the area of immunity and inflammation. From 1990 to 1998, she served as Principal Scientist and Director of Immune and Cell Therapy at Cell Genesys, Inc. Dr. Roberts is the inventor on the first set of CAR patents, including second-generation CAR constructs that incorporate the domains of costimulatory receptors, such as CD28, aimed at improving CAR T cell survival and function. She is the author of more than 25 scientific publications and inventor on 13 issued US patents and three published US patent applications related to CAR technology and tumor vaccine therapies. Dr. Roberts was a postdoctoral fellow at Yale and at the LGME of the CNRS in France. She received her BSc with honors and her PhD from the University of Leeds in England.

1:50
Placental Natural Killer Cells (PNK-007) for the Treatment of Hematologic Malignancies
 
Wolfgang Hofgartner
Wolfgang Hofgartner
Vice President, Research
Celgene Cellular Therapeutics
About Speaker: Dr. Hofgartner is Vice President of Research & Development at Celgene Cellular Therapeutics, which is a clinical stage company with multiple phase 1/2 programs. The company is focused on developing and advancing a number of distinct cellular immu... Read Full Bio 
 
 
Wolfgang Hofgartner
Vice President, Research
Celgene Cellular Therapeutics
 
About Speaker:

Dr. Hofgartner is Vice President of Research & Development at Celgene Cellular Therapeutics, which is a clinical stage company with multiple phase 1/2 programs. The company is focused on developing and advancing a number of distinct cellular immuno-oncology programs for therapeutic areas with high unmet medical need. Prior to joining Celgene Cellular Therapeutics in 2004, Dr. Hofgartner was Medical Director at Becton Dickinson, a global medical technology company. By background he is a board certified Clinical Pathologist, trained at the University of Washington in Seattle, with subsequent fellowship training in Molecular Pathology at the University of Pittsburgh.

 

 
Abstract: Clinical studies suggest that adoptive transfer of allogeneic natural killer (NK) cells represent a promising treatment for patients with hematolog...Read More 

Clinical studies suggest that adoptive transfer of allogeneic natural killer (NK) cells represent a promising treatment for patients with hematological malignancies and solid tumors. Celgene Cellular Therapeutics has established a cultivation process to generate human NK cells (PNK-007) from umbilical cord blood CD34+ cells with substantial cytolytic activity against several human tumor cell lines, primary AML and primary multiple myeloma cells. Two Phase 1 clinical studies of PNK-007 infusion, one in AML and the other in multiple myeloma, will be discussed.

Bullet Points:

  • Placental Natural Killer Cells (PNK-007) are expanded and differentiated from cord blood CD34 cells
  • PNK-007 constitutes an allogeneic, off-the-shelf product for systemic application
  • PNK-007 is currently in Phase 1 studies in AML and multiple myeloma
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2:15
Pluripotent Stem Cell Derived Dendritic Cells for the Immunotherapy of Lung Cancer
 
Jane Lebkowski
Jane Lebkowski
President, Research & Development
Asterias Biotherapeutics
About Speaker: Jane Lebkowski has been actively involved in the development of cell and gene therapies since 1986 and is currently President of R&D and Chief Scientific Officer at Asterias Biotherapeutics Inc, where she is responsible for all R&D of Asteria... Read Full Bio 
 
 
Jane Lebkowski
President, Research & Development
Asterias Biotherapeutics
 
About Speaker:

Jane Lebkowski has been actively involved in the development of cell and gene therapies since 1986 and is currently President of R&D and Chief Scientific Officer at Asterias Biotherapeutics Inc, where she is responsible for all R&D of Asterias’ products.  From 1998 to 2012, Dr. Lebkowski was Senior Vice President of Regenerative Medicine and Chief Scientific Officer at Geron Corporation.  Dr. Lebkowski led Geron’s human embryonic stem cell program, being responsible for all research, preclinical development, product development, manufacturing, and clinical development activities.  Prior to Geron, Dr. Lebkowski was Vice President of Research and Development at Applied Immune Sciences.  Following the acquisition of Applied Immune Sciences by Rhone Poulenc Rorer (RPR, currently Sanofi), Dr. Lebkowski remained at RPR as Vice President of Discovery Research.  During Dr. Lebkowski’s tenure at RPR, she coordinated preclinical investigations of gene therapy approaches for treatment of cancer, cardiovascular disease and nervous system disorders, and directed vector formulations and delivery development.  Dr. Lebkowski received her Ph.D. in Biochemistry from Princeton University in 1982, and completed a postdoctoral fellowship at the Department of Genetics, Stanford University in 1986.

Dr. Lebkowski has published over 70 peer reviewed papers and has 13 issued U.S. patents.  Dr. Lebkowski has served on the board of Directors of the American Society for Gene and Cell Therapy and as the co-chair of the Industrial Committee of the International Society for Stem Cell Research. Dr Lebkowski serves on several scientific advisory boards and other professional committees.

 
Abstract: AST-VAC2 is a cancer immunotherapy product comprising embryonic stem cell-derived dendritic cells electroporated with ...Read More 

AST-VAC2 is a cancer immunotherapy product comprising embryonic stem cell-derived dendritic cells electroporated with an mRNA encoding a telomerase/lysosome-associated membrane protein 1 (LAMP hTERT) chimeric tumor antigen. Previous studies have shown that AST-VAC2 can elicit a telomerase-specific T-cell response from partially HLA-matched donor peripheral blood mononuclear cells.  AST-VAC2 is being developed as an off-the-shelf allogeneic cancer vaccine for clinical testing in partially HLA matched patients with non-small cell lung carcinoma. The AST-VAC2 product is derived from the H1 human embryonic stem cell line which is expanded and differentiated into mature dendritic cells in a multi-step process.  The dendritic cells are harvested, electroporated with the LAMP hTERT mRNA, cryopreserved and  irradiated. It is anticipated that the proposed Phase 1/2a clinical trial will enroll approximately 24 patients and will include subjects with either advanced or resected disease. Asterias has partnered with Cancer Research United Kingdom (CRUK) for the GMP manufacturing and initial clinical testing of AST-VAC2 in the UK.

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2:40
Monoclonal Antibody Delivery with MSCs
 
John E Davies
John E Davies
Professor, Institute of Biomaterials and Biomedical Engineering (IBBME); President & Chief Executive Officer
University of Toronto; Tissue Regeneration Therapeutics Inc.
About Speaker: Davies is a Full Professor at the Institute of Biomaterials and Biomedical Engineering (IBBME) of the University of Toronto, with cross-appointments to the Faculty of Applied Science and Engineering, the Faculty of Dentistry and the Faculty of Medici... Read Full Bio 
 
 
John E Davies
Professor, Institute of Biomaterials and Biomedical Engineering (IBBME); President & Chief Executive Officer
University of Toronto; Tissue Regeneration Therapeutics Inc.
 
About Speaker:

Davies is a Full Professor at the Institute of Biomaterials and Biomedical Engineering (IBBME) of the University of Toronto, with cross-appointments to the Faculty of Applied Science and Engineering, the Faculty of Dentistry and the Faculty of Medicine (Dept of Surgery). He is also the founding President, and CEO, of Tissue Regeneration Therapeutics Inc (TRT), a Toronto-based mesenchymal stromal cell company. TRT extracts this population of cells from the perivascular tissue of the human umbilical cord to provide 2 platforms: native and engineered cells. These cells are now in pre-clinical trials for multiple therapeutic targets including pediatric GvHD, lung and pancreatic islet transplantation, bone tissue engineering and wound healing.

Davies trained as an anatomist and oral surgeon in the UK. He was awarded a DSc by the University of London, England, for his sustained research contributions over a period of 20 years to the field of Biomaterials. He has edited 2 books, published over 200 hundred scientific papers and book chapters and filed >70 patents.

 
Abstract: Tissue Regeneration Therapeutics Inc. (TRT) has built a robust international patent portfolio based on its discovery, ...Read More 

Tissue Regeneration Therapeutics Inc. (TRT) has built a robust international patent portfolio based on its discovery, harvesting, expansion and engineering of a mesenchymal cell source from the perivascular tissue of the human umbilical cord.

One TRT platform, TXP, comprises a rich homogeneous mesenchymal population currently being employed in several pre-clinical projects from their co-administration with pancreatic islets; to acute respiratory distress syndrome, and an imminent Phase 1 trial in steroid-refractory GvHD.

The second platform, eTXP—the focus of this presentation—comprises engineered MSCs for the delivery of bio-effector molecules including monoclonal antibodies. Our initial focus, and proof-of-principle, was the employment of eTXP to secrete an anti-viral antibody as a new form of biomedical counter-measure. However, among the problems of MSCs as therapeutic delivery vehicles are the challenges of producing clinically relevant numbers of cells at low passage number, the short dwell time of such cells in the lungs following intravenous delivery, the disparate effects of pro-inflammatory and immunosuppressive MSC phenotypes, and the perceived need to culture MSCs to permit recovery from cryogenic storage. These issues are all addressed by TRT’s eTXP platform.

This presentation will show that the eTXP platform can provide sustained delivery of targeted bio-effectors of relevance to immunotherapy.

 

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3:05
Afternoon Networking Break
What’s in the Pipeline: Pre-clinical & Clinical Strategies
Moderator: Alain Vertes, NxR Biotechnologies GmbH
3:35
Chronic Disability after Stroke: Can Neural Stem Cells Provide a Cure?
 
John Sinden
John Sinden
Chief Scientific Officer
ReNeuron
About Speaker: John Sinden is Chief Scientific Officer of ReNeuron. From 1998 to 2015 he was a director of the ReNeuron companies. Prior to founding ReNeuron and becoming its first employee, he was Reader in Neurobiology of Behaviour at the Institute of Psychiatry ... Read Full Bio 
 
 
John Sinden
Chief Scientific Officer
ReNeuron
 
About Speaker:

John Sinden is Chief Scientific Officer of ReNeuron. From 1998 to 2015 he was a director of the ReNeuron companies. Prior to founding ReNeuron and becoming its first employee, he was Reader in Neurobiology of Behaviour at the Institute of Psychiatry at Kings College London. He graduated in Psychology from the University of Sydney and completed a Ph.D. in Neuroscience from the University of Paris at the College de France. He subsequently held post-doctoral appointments at Oxford University and the Institute of Psychiatry prior to joining the tenured staff of the Institute in 1987. Dr. Sinden is an Honorary Professor in the Faculty of Medical Sciences at University College London and has over 140 scientific publications and book chapters. He holds Fellowships of the Royal Society of Medicine and the Royal Society of Biology and is a member of the International Society for Stem Cell Research and the Expert Working Group on Cell and Gene Therapies for the Bioindustry Organization BioSafe Committee.

 
Abstract: CTX is a conditionally immortalised clonal neural stem cell line generating a stable frozen cell therapy product able to be manufactured to GMP at ...Read More 

CTX is a conditionally immortalised clonal neural stem cell line generating a stable frozen cell therapy product able to be manufactured to GMP at large scale and with long shelf life for clinical and commercial use.

Several published preclinical studies have shown recovery of motor function in rodents with stable disability post middle cerebral artery occlusion. Survival and neural differentiation of grafted cells at the implant sites are seen, along with a range of host regenerative mechanisms, including vascular genesis, enhanced post-stroke neurogenesis and reparative modulation of the damaging immune response to the occlusion.

Recently, we have published excellent long-term safety across a range of cell doses in a Phase I trial of intracerebrally delivered CTX in 11 chronic stroke patients with significant and stable motor disability. Improvements in motor function compared to pretreatment baseline were seen in the majority of patients. More recently, a Phase II single arm study looking at a range of outcome efficacy measures in hemiparetic stroke patients has recently reported interim data which are promising across a range of motor, disability and activities of daily life measures.

Future placebo controlled pivotal studies are under discussion with FDA and EMA, including the use of placebo surgery as a control.

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4:00
Bringing CRISPR Therapeutics into the Clinic
 
Chad Cowan
Chad Cowan
Scientific Founder and Research Head
CRISPR Therapeutics
About Speaker: Dr. Chad Cowan is an Associate Professor at Harvard University in the Department of Stem Cell and Regenerative Biology, and at Massachusetts General Hospital, with appointments in the Center for Regenerative Medicine, the Cardiovascular Research Cent... Read Full Bio 
 
 
Chad Cowan
Scientific Founder and Research Head
CRISPR Therapeutics
 
About Speaker:

Dr. Chad Cowan is an Associate Professor at Harvard University in the Department of Stem Cell and Regenerative Biology, and at Massachusetts General Hospital, with appointments in the Center for Regenerative Medicine, the Cardiovascular Research Center and the Center for Human Genetics Research. He is an associate member of the Broad Institute and a principal faculty member of the Harvard Stem Cell Institute, where he directs the Diabetes Disease Program and the iPS Cell Core Facility.

Chad has led or been a member of several large efforts to use stem cells to better understand disease, including the National Heart, Lung, and Blood Institute’s Next Gen iPS Cell Project and the Progenitor Cell Biology Consortium. More recently, Chad has focused on using gene editing tools as therapeutics.

Chad received his B.A. and B.S., with honors, from the University of Kansas. He received his Ph.D. from the University of Texas Southwestern at Dallas, garnering the Nominata award for most outstanding thesis. He subsequently completed a Damon Runyon fellowship with Professor Douglas Melton at Harvard University. He was named a Stowers Medical Investigator in 2006 and has been a faculty member at Harvard University since 2008.

 
Abstract: Only five short years after Emmanuelle Charpentier, Jennifer Doudna and colleagues demonstrated how to use the CRISPR ...Read More 

Only five short years after Emmanuelle Charpentier, Jennifer Doudna and colleagues demonstrated how to use the CRISPR system with a programmable RNA to precisely cleave specific DNA sequences, applications using the technology in stem cells and other cell types are already in development. The adoption of CRISPR-Cas9 gene editing for use in research, and for developing novel medicines, has been remarkably rapid, in part because it is so easy to apply these fundamental discoveries to higher organisms including humans. CRISPR/Cas9 edited T-cells are currently being tested in patients, and numerous other applications of both ex vivo in vivo applications are in late preclinical testing. This talk will review programs in development with a particular focus on an application in hematopoietic stem cells, and provide a discussion of remaining considerations and technical hurdles that still need to be addressed as we bring CRISPR therapeutics into clinical stage testing in patients.

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4:25
Encapsulated Stem Cells Targeting Apoptotic and Immune Modalities for Cancer
 
Khalid Shah
Khalid Shah
Director Center for Stem Cell Therapeutics and Imaging
Harvard Medical School
About Speaker: Dr. Shah is an Associate Professor at Harvard Medical School. He is also the Director of Center for Stem Cell Therapeutics and Imaging and Vice Chair of Research at Brigham and Women’s Hospital and a Principal Faculty at Harvard Stem Cell Institute... Read Full Bio 
 
 
Khalid Shah
Director Center for Stem Cell Therapeutics and Imaging
Harvard Medical School
 
About Speaker:

Dr. Shah is an Associate Professor at Harvard Medical School. He is also the Director of Center for Stem Cell Therapeutics and Imaging and Vice Chair of Research at Brigham and Women’s Hospital and a Principal Faculty at Harvard Stem Cell Institute in Boston. His laboratory focuses on developing therapeutic stem cells for receptor targeted therapies for cancer and testing their efficacy in clinically relevant mouse tumor models. In recent years, Dr. Shah and his team have pioneered major developments in the stem cell therapy field, successfully developing experimental models to understand basic cancer biology and therapeutic stem cells for cancer, particularly brain tumors. These studies have been published in a number of very high impact journals like Nature Neuroscience, PNAS, Nature Reviews Cancer, JNCI, Stem Cells and Lancet Oncology, validating the use of therapeutic stem cells alone and in combination with clinically approved drugs for cancer therapy.

Recently, Dr. Shah's work has caught the attention in the public domain and as such it has been highlighted in the media world-wide including features on BBC and CNN. Dr. Shah holds current positions on numerous councils, advisory and editorial boards in the fields of stem cell therapy and oncology. The technologies from Dr. Shah’s laboratory have led to the foundation of a biotech company, AMASA Technologies Inc. whose main objective is the clinical translation of therapeutic stem cells in cancer patients.

 
Abstract: Stem cell-based therapies are emerging as a promising strategy to tackle cancer. Using our recently established invasive, recurrent and resection m...Read More 

Stem cell-based therapies are emerging as a promising strategy to tackle cancer. Using our recently established invasive, recurrent and resection models of primary brain tumors and metastatic tumors in the brain that mimic clinical settings, we have shown that receptor targeted encapsulated adult stem cells expressing novel bi-functional proteins or loaded with oncolytic viruses target both the primary and the invasive tumor deposits and have profound anti-tumor effects. Recently we have shown the efficacy of encapsulated stem cell released immunomodulatory agents in syngeneic mouse tumor models that mimic the clinical scenario of tumor resection and growth. These studies demonstrate the strength of employing engineered stem cells in preclinical-therapeutic tumor models and form the basis for their clinical translation. This presentation considers the current status of stem cell-based treatments for cancer and provides a rationale for translating the most promising preclinical studies into the clinic.

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4:50
Progenitor Cell Activation - an Enabling Technology for In-Situ Tissue Regeneration
 
Chris Loose
Chris Loose
Co-founder & Chief Scientific Officer
Frequency Therapeutics
About Speaker: Chris is the Chief Scientific Officer and Co-founder of Frequency Therapeutics. He is an entrepreneurial scientist who has overseen the translation of new medical technologies from concept to approved products. Previously, Chris co-founded Semprus Bi... Read Full Bio 
 
 
Chris Loose
Co-founder & Chief Scientific Officer
Frequency Therapeutics
 
About Speaker:

Chris is the Chief Scientific Officer and Co-founder of Frequency Therapeutics. He is an entrepreneurial scientist who has overseen the translation of new medical technologies from concept to approved products. Previously, Chris co-founded Semprus BioSciences and served as Chief Technology Officer through the company’s acquisition by Teleflex in 2012. He led the technology team in developing medical products with surface modifications designed to reduce infection and clotting, leading to FDA/CE Mark clearance. In 2011, he was awarded the inaugural Peter Strauss Entrepreneurial Award from the Hertz Foundation. Chris has also been named to the Technology Review TR35, the 35 innovators under 35 most likely to impact technology, and was named to the Boston Business Journal’s 40 Under 40. He is currently an Associate Adjunct Professor of Urology in the Yale School of Medicine. Chris holds a Ph.D. in Chemical Engineering from MIT, where he studied under Robert Langer, Sc.D., and a BSE from Princeton University.

 
Abstract: Frequency Therapeutics, is leveraging a proprietary Progenitor Cell Activation screening platform using primary human ...Read More 

Frequency Therapeutics, is leveraging a proprietary Progenitor Cell Activation screening platform using primary human cells to develop innovative disease modifying medicines that temporarily activate cellular regeneration at specific sites within the body to restore healthy tissue. This talk will focus on Frequency’s application of its PCA platform to develop a novel treatment to restore hearing in people with chronic noise-induced hearing loss. Over 360 million people worldwide suffer from hearing impairment, with no effective therapeutic solutions currently available. Hearing loss caused by prolonged exposure to excessive noise can be attributed to many professional and social environments, such as first responders, manufacturing, music concerts or military combat. Sensory hair cells in the hearing portion of the inner ear, or cochlea, are susceptible to damage, and because these cells do not regenerate spontaneously, the number of hair cells in the ear only decreases with age. Recent breakthroughs by Frequency’s co-founders provide an approach to regenerate sensory hair cells using locally-delivered small molecules that activate key regenerative pathways. The highly promising work unlocks the potential to develop restorative treatments for hearing loss, and could eventually be applied to other applications spanning dermatological, ocular, and GI diseases.

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5:15
Networking Reception & Poster Session
Day - 2 Thursday, April 6th, 2017
7:30
Continental Breakfast
Joint Plenary Session: Where do We Stand with Cell Therapy in Diabetes?
Moderator: Mark Zimmerman, ViaCyte
8:30
Developing Cell Therapies for Diabetes
 
Mark Zimmerman
Mark Zimmerman
Vice President, Strategy and Business Development
ViaCyte
About Speaker: Mark Zimmerman received his bachelors in Biology from Syracuse University and his masters and doctorate in Biomedical Engineering from Rutgers University. Mark joined UMD-New Jersey Medical School as an Assistant Professor in 1986 and left in 1996 as... Read Full Bio 
 
 
Mark Zimmerman
Vice President, Strategy and Business Development
ViaCyte
 
About Speaker:

Mark Zimmerman received his bachelors in Biology from Syracuse University and his masters and doctorate in Biomedical Engineering from Rutgers University. Mark joined UMD-New Jersey Medical School as an Assistant Professor in 1986 and left in 1996 as an Associate Professor of Surgery with tenure. His research interests spanned musculoskeletal tissue engineering, sports medicine, trauma, and spine biomechanics and biomaterials.

Mark joined Johnson and Johnson in 1997 as a principal scientist/group leader and transitioned into regenerative medicine projects related to orthopaedic surgery, wound healing, vascular biology, and diabetes. Mark was appointed executive director of a Lifescan incubator, BetaLogics, in July 2002. BetaLogics transitioned to a Johnson and Johnson Internal Venture in 2004. The mission of BetaLogics was to discover and develop a cellular product to treat diabetes. Mark was appointed Venture Leader/Vice President of BetaLogics, a business unit of Janssen R&D LLC in 2009. Janssen completed a business transaction with ViaCyte in 2015 and merged the assets of BetaLogics into ViaCyte. Mark is currently seconded to ViaCyte and serves as the Vice President of Strategy and Business Development.

 
Abstract: ViaCyte’s product candidates are based on the directed differentiation of pancreatic progenitor cells from human pluripotent stem cells. Thes...Read More 

ViaCyte’s product candidates are based on the directed differentiation of pancreatic progenitor cells from human pluripotent stem cells. These pancreatic progenitor cells are implanted in a durable and retrievable encapsulation device. Once implanted, these cells are designed to further mature to the pancreatic endocrine cells, including beta cells, which secrete insulin and other regulatory factors in response to blood glucose levels. ViaCyte has two products in development. The PEC-Direct™ product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients that have severe hypoglycemic episodes, extreme glycemic lability, and/or impaired hypoglycemia awareness. The PEC-Encap™ (also known as VC-01) product candidate delivers pancreatic progenitor cells in an immunoprotective device and is currently being evaluated in a Phase 1/2 trial in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. This presentation will describe first-hand experience with the challenges and progress in developing pluripotent stem cell-derived products, including manufacturing and quality control, methods of delivery, clinical trials, and market opportunities.

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8:55
Development of a Stem Cell Derived Pancreatic Islet Cell Therapy for Diabetes
 
Felicia Pagliuca
Felicia Pagliuca
Vice President, Cell Biology R&D
Semma Therapeutics
About Speaker: Dr. Pagliuca currently serves as Vice President of Cell Biology Research and Development and is scientific co-founder of Semma Therapeutics. She leads cell-based research and development and plays a key role in supporting Semma's preclinical, regulat... Read Full Bio 
 
 
Felicia Pagliuca
Vice President, Cell Biology R&D
Semma Therapeutics
 
About Speaker:

Dr. Pagliuca currently serves as Vice President of Cell Biology Research and Development and is scientific co-founder of Semma Therapeutics. She leads cell-based research and development and plays a key role in supporting Semma's preclinical, regulatory, and manufacturing strategies for its cell therapy products. Felicia also works closely with Semma’s senior leadership on corporate development activities, including key collaborations and partnerships. Previously, Felicia was a postdoctoral fellow in Professor Doug Melton’s laboratory at the Harvard Stem Cell Institute. Felicia was part of the team in the Melton lab that discovered how to generate stem cell derived beta cells and published a seminal paper in Cell in 2014. She is an expert in stem cell biology and diabetes and one of the inventors of Semma Therapeutics’ key technologies. Felicia received a B.S. from Duke University and a Ph.D. from Cambridge University where she was a Marshall Scholar. She is currently on leave from Harvard Business School where she was a Kaplan Fellow.

 
Abstract: Semma Therapeutics is a preclinical stage biotechnology company with the mission to transform the treatment of diabete...Read More 

Semma Therapeutics is a preclinical stage biotechnology company with the mission to transform the treatment of diabetes through development of stem cell derived pancreatic islets, including insulin-producing beta cells, to be used as a cell replacement therapy.   Diabetes results from the dysfunction and/or destruction of the insulin-producing beta cells in the pancreatic islet.  The development of replacement sources of beta cells, combined with effective methods of delivery back into the patient’s body, has the potential to “cure” the disease.  Recent breakthroughs have enabled the virtually unlimited production of replacement beta cells through pluripotent stem cell differentiation.  At Semma, we are focused on further optimization and innovation in differentiation technologies, manufacturing scale-up, and characterization of stem cell derived islets in preclinical studies in order to move into clinical trials.  In parallel, we are engineering innovative encapsulation solutions, using novel materials and device configurations, to solve the challenge of delivering and protecting these therapeutics from immune destruction.

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9:20
Tolerogenic Therapy for Type 1 Diabetes: A Phase 2 Randomized Study of Autologous Regulatory T-cells Adolescents with Recent Onset T1DM
 
Douglas Losordo
Douglas Losordo
Chief Medical Officer, Senior Vice President of Clinical, Medical & Regulatory Affairs
Caladrius Biosciences
About Speaker: Dr. Losordo is the Chief Medical Officer and Senior Vice President of Clinical, Medical and Regulatory Affairs of Caladrius Biosciences, Inc, Clinical Professor of Medicine at  the New York University Langone Medical Center  and Adjunct Professor o... Read Full Bio 
 
 
Douglas Losordo
Chief Medical Officer, Senior Vice President of Clinical, Medical & Regulatory Affairs
Caladrius Biosciences
 
About Speaker:

Dr. Losordo is the Chief Medical Officer and Senior Vice President of Clinical, Medical and Regulatory Affairs of Caladrius Biosciences, Inc, Clinical Professor of Medicine at  the New York University Langone Medical Center  and Adjunct Professor of Medicine at the Northwestern University Feinberg School of Medicine in Chicago, Illinois.

Dr. Losordo’s career has been dedicated to patient care and to the development of novel therapeutics aimed at the reversal or repair of chronic conditions such as heart failure, critical limb ischemia, cancer and diabetes.

A native of Brooklyn, NY, he received his medical degree from the University of Vermont. Dr. Losordo completed an internship, residency and fellowship at St. Elizabeth’s Medical, Boston, Massachusetts, where he subsequently joined the faculty, working with the late Jeff Isner to develop a program in gene therapy and cell-based tissue repair. Dr. Losordo’s group has executed the full “translational medicine” paradigm: identifying potential therapeutic approaches in the laboratory, investigating these strategies in pre-clinical/IND-enabling models and designing and executing first-in-human and proof-of-concept clinical trials as the study sponsor/IND-holder. His work has included developing VEGF gene therapy for myocardial ischemia and diabetic neuropathy, CD34+ cell therapy for refractory angina, critical limb ischemia, severe claudication and coronary microvascular dysfunction and regulatory T cell therapy for autoimmune disease. Two of these candidates advanced to phase 3. At Caladrius Dr. Losordo has initiated a phase 2 study of autologous regulatory T cell therapy for new onset type 1 diabetes in children and recently received Japanese PMDA agreement on a study of CD34 cell therapy for critical limb ischemia targeting conditional approval under the new Japanese regulatory rules governing regenerative therapies. In addition to his own work Dr. Losordo has also mentored numerous scientists and physician-scientists from around the world who now have their own independent programs in translational research.

 
Abstract: T1DM in children is characterized by increased immune activation and a more aggressive clinical course compared to the adult population. Available ...Read More 

T1DM in children is characterized by increased immune activation and a more aggressive clinical course compared to the adult population. Available evidence suggests that adoptive transfer of regulatory T-cells (Tregs) can slow disease progression and potentially lead to remission. Previous phase 1 clinical trials have demonstrated that expanded polyclonal Tregs are safe and well tolerated in both adults and children, supporting the development of a phase 2 trial to assess safety and efficacy of Treg therapy for T1DM in adolescents.

Caladrius Biosciences has launched a phase 2, double-blind, placebo-controlled, multi-center clinical trial to assess the safety and efficacy of autologous Tregs  to modify the T1DM disease course, including preservation of β-cell function and improvements in disease severity in adolescents (aged 8 to 17 years) with recent onset T1DM.

Approximately 111 subjects will be randomized to one of 3 treatment groups in a 1:1:1 ratio (placebo, 2.5 or 20 million Treg cells/kg BW). Key endpoints include the 2-hour and 4-hour Mixed Meal Tolerance Test (MMTT) stimulated C-peptide AUC at various time points through 24 months, daily dose of insulin use, severe hypoglycemia, and the percentage of subjects achieving partial or complete T1DM remission. Subjects will be followed for 2 years. A safety review has been conducted after the initial 19 subjects were followed for 3 months. An interim analysis is planned after approximately 50% of subjects complete the Week 26 visit. Extensive immune-profiling will be performed before and after Treg vs placebo infusion

This phase 2 study will advance insight into the safety and potential efficacy of adoptive transfer of Tregs to modify the T1DM disease course in children with recent onset T1DM as well as providing unprecedented insights into immune regulatory function.

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9:45
Mesenchymal Stromal Cells as Cellular Therapeutics to Enhance Human Pancreatic Islet Transplantation in Type-1 Diabetes
 
John Campbell
John Campbell
Associate Director, Research, Development & Innovation
SNBTS, National Science Laboratory
About Speaker: Professor John Campbell is Associate Director of Research and Development at the Scottish National Blood Transfusion Service (SNBTS) in Edinburgh. He completed his PhD in Pathology at Edinburgh in 1995 on the immunopathogenesis of lymproliferative di... Read Full Bio 
 
 
John Campbell
Associate Director, Research, Development & Innovation
SNBTS, National Science Laboratory
 
About Speaker:

Professor John Campbell is Associate Director of Research and Development at the Scottish National Blood Transfusion Service (SNBTS) in Edinburgh. He completed his PhD in Pathology at Edinburgh in 1995 on the immunopathogenesis of lymproliferative disease, and has worked in the cellular therapy field for over 20 years in academic and industry positions. He is currently the national head of research for SNBTS and holds academic appointments at the Universities of Glasgow and Edinburgh. SNBTS has a substantial cellular therapy research programme, with over 40 full time scientists working on basic cellular function; translation of laboratory protocols to full GMP processes; and production of cellular therapeutics for treatment of patients. SNBTS has a dedicated, fully MHRA licensed, GMP cellular therapy production centre at the Scottish Centre for Regenerative Medicine. This GMP manufacturing and development capacity will be substantially increased when SNBTS moves to the Jack Copland Centre, Edinburgh, in early 2017. Cellular therapeutics in development and early phase clinical trials include, mesenchymal stromal cells, corneal limbal stem cells, macrophages for tissue repair and virus -specific T Lymphocytes.

 
Abstract: Islet transplantation is of proven efficacy in subjects with Type 1 diabetes where glycaemic control is problematic, stabilizing glycaemic control ...Read More 

Islet transplantation is of proven efficacy in subjects with Type 1 diabetes where glycaemic control is problematic, stabilizing glycaemic control and restoring awareness of hypoglycaemia where this has been compromised. However, long term graft survival remains poor and patients typically require two to three islet transplants.  Innate and adaptive immune responses contribute to early and ongoing graft attrition. Therapeutic strategies to improve graft function are urgently needed. Ideally, a therapeutic strategy would modulate the inflammatory and immune response and enhance engraftment of islets. Mesenchymal stromal cells (MSCs) are multipotent cells found in the majority of tissues and have been shown to support regeneration of tissues by supporting blood vessel formation through a broad spectrum of growth factors and extracellular matrix secreted. This tissue building function is accompanied by the ability to suppress T cell responses and modulate inflammatory infiltration. We have investigated the ability of GMP-grade human MSC to support human Islet engraftment and function in vivo pre-clinical models. In this presentation we will discuss the properties of MSC from different source tissues and how they can be used to support and improve islet graft function.

Learning outcomes:

Understanding the impact of islet transplantation on the health of patients with severe Type-1 Diabetes

Understanding the function of Mesenchymal Stromal Cells as cellular therapeutics, including the challenges of GMP manufacturing

In vivo models of human islet transplantation – what can be learned and what are the limitations

Measuring the improvement in transplant function when co-transplanted with cellular therapeutics

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10:10
Morning Networking Break
12:10
Lunch on Your Own