Navigation
banner

Stem Cell Product Development & Commercialization

2017-11-182018-03-092018-02-13
EARLY BIRD DISCOUNT! Register by Feb 13, 2018 to receive 20% off your registration!
Or Register 3 for the price of 2 with the coupon code rcdvb!


2017 Agenda
Day 1 - 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.

 Read Less
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.

 Read Less
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.

 Read Less
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

 Read Less
10:10
Morning Networking Break
12:10
Lunch on Your Own
Regulatory Challenges in Cell Therapy
Moderator: William Sietsema, Caladrius Biosciences
1:30
Monitoring Evolving Regulatory Policy
 
Melody Eble
Melody Eble
Director, Global Regulatory Affairs-Advanced Therapies
Janssen Research & Development, LLC
About Speaker: Melody has over 20 years of regulatory experience and has worked on products at various stages of development, from early through to filing, approval and commercialization. Currently, Melody provides global strategic regulatory oversight for Janssen... Read Full Bio 
 
 
Melody Eble
Director, Global Regulatory Affairs-Advanced Therapies
Janssen Research & Development, LLC
 
About Speaker:

Melody has over 20 years of regulatory experience and has worked on products at various stages of development, from early through to filing, approval and commercialization. Currently, Melody provides global strategic regulatory oversight for Janssen’s lead cell therapy program and is responsible for coordinating Janssen’s advanced therapy regulatory policy efforts. In her prior role, as a Director in Global Regulatory Policy and Intelligence, Melody was responsible for leading and delivering regulatory policy and intelligence analyses to enhance global regulatory strategy for Janssen products across all therapeutic areas. Melody completed her B.S. and Doctor of Pharmacy degrees at University of Buffalo in New York and completed an ASHP Accredited Post-Doctoral Residency in Oncology Pharmacy in San Antonio, Texas.

 
Abstract: This session will review the Regenerative Advanced Therapy (RAT) provisions within the US 21st Century Cures Act including: 1) The RAT Designation ...Read More 

This session will review the Regenerative Advanced Therapy (RAT) provisions within the US 21st Century Cures Act including: 1) The RAT Designation criteria, 2) Guidance to be created regarding devices used in the recovery, isolation, or delivery of regenerative advanced therapies, 3) Annual Report on regenerative advanced therapies, 4) Development of Standards for regenerative medicine and regenerative advanced therapies. Time permitting, a list of anticipated EU and US guidance will be presented.

 Read Less
1:50
Global Regulation of Cell Therapies
 
William Sietsema
William K Sietsema
Executive Director, Global Regulatory Affairs
Caladrius Biosciences
About Speaker: Dr. Sietsema is Executive Director, Global Regulatory Affairs at Caladrius Biosciences, a company that focuses on innovative cell therapies for difficult-to-treat diseases. Prior to Caladrius, he was Global Regulatory Lead at Amgen where he provided ... Read Full Bio 
 
 
William K Sietsema
Executive Director, Global Regulatory Affairs
Caladrius Biosciences
 
About Speaker:

Dr. Sietsema is Executive Director, Global Regulatory Affairs at Caladrius Biosciences, a company that focuses on innovative cell therapies for difficult-to-treat diseases. Prior to Caladrius, he was Global Regulatory Lead at Amgen where he provided strategic guidance to a portfolio of early stage projects in oncology and inflammation. He was also Vice President, Global Regulatory Consulting and Submissions at Kendle International/INC Research and Adjunct Professor of Pharmaceutical Sciences at the University of Cincinnati, College of Pharmacy, where he taught evening classes in drug development. He has 32 years of experience in the pharmaceutical industry. During his sixteen years with Kendle/INC, he brought leadership to several initiatives in the fields of inflammation, skeletal disease, analgesia, gastrointestinal disease, ophthalmology, and women's health. He played a pivotal role in the rapid development of Pharmacia's Celebrex, which transited from beginning of Phase 2 to NDA approval in less than three years. He received his BA, magna cum laude, in Chemistry from the University of Colorado, Boulder in 1977 and his PhD in biochemistry from the University of Wisconsin, Madison in 1982. He is the author of 24 journal articles, 4 book chapters, 42 presentations and posters and is an inventor on 6 patents. He has published six books on regulatory topics ranging from strategic planning to practical aspects of preparing CTAs and MAAs. He is a member of the American Chemical Society, the Regulatory Affairs Professional Society, and the Association for Regenerative Medicine. He was recognized by R&D Directions as one of the top 20 clinical research scientists in 2007

 
Abstract: Regulation of cell therapies presents some challenges, given that regulatory platforms were designed with small molecule drugs and antibody or vacc...Read More 

Regulation of cell therapies presents some challenges, given that regulatory platforms were designed with small molecule drugs and antibody or vaccine biologics in mind. There are a few examples of cell therapies approved under existing platforms. Regulation and guidance globally take into account whether therapies are autologous, allogeneic, or xenogeneic, whether the intended use is homologous or not, and the extent to which cells are manipulated. In the US, cell therapies are regulated by the recently renamed Office of Tissues and Advanced Therapies within CBER at FDA. Cell therapies primarily follow approval pathways for other products but there are regulations specific to cell therapies at 21 CFR §1270/1271. In the US, recent legislation created a designation for Regenerative Advanced Therapy which is expected to provide benefits as for other expedited programs. In Europe, cell therapies primarily follow existing procedures but the EMA has established a special Committee for Advanced Therapies with cell therapy expertise. Europe has regulations and directives specific to cell therapies, for example 1394/2007 and 2001/83/EC. Europe has also established a designation for Advanced Therapeutic Medicinal Product which provides benefits such as fee reductions and access to expertise. Japan has adapted their regulatory platform to include a new regenerative medicine law that is innovative and flexible, allowing conditional approval based on preliminary data which predict efficacy and assure safety. Procedures are similar to those for other medicines. In Canada, cell therapy products are regulated by the Biologics and Genetic Therapies Directorate and follow established procedures for Clinical Trial Applications and New Drug Submissions. Canada has prepared guidance for cell therapy products. A progressive licensing pathway (conditional approval) exists in Canada for all products and has been used for approving at least one cell therapy. Many regions have provisions for a “hospital exemption” which permit administration of an autologous cell therapy when cells are collected, minimally processed, and administered back to the patient within the same institution. Globally, cell therapy regulation continues to evolve, thus regulatory professionals must stay abreast of new legislation and guidance. Attendees will benefit from a high level understanding of how cell therapies are regulated in key regions around the world.

 Read Less
2:10
Navigating the Regulatory Pathway for Cell Therapy Products: Common Problems and How to Avoid Them
 
Scott Burger
Scott R. Burger
Principal
Advanced Cell & Gene Therapy
About Speaker: Scott R. Burger, MD, is the Principal of Advanced Cell and Gene Therapy, a consulting firm specializing in cell and gene therapy product development, manufacturing, and regulatory affairs. Dr. Burger has over 25 years of experience developing cell an... Read Full Bio 
 
 
Scott R. Burger
Principal
Advanced Cell & Gene Therapy
 
About Speaker:

Scott R. Burger, MD, is the Principal of Advanced Cell and Gene Therapy, a consulting firm specializing in cell and gene therapy product development, manufacturing, and regulatory affairs. Dr. Burger has over 25 years of experience developing cell and gene therapy products and has consulted for over 100 industry and academic clients in North America, Europe, Asia, and Australia. He has directed or consulted on process development, manufacturing, and regulatory aspects of a wide range of cell therapy and gene therapy products, including CAR T-cells, gene-edited cells, dendritic cell vaccines, natural killer (NK) cells, and other immunotherapies, hematopoietic stem cells (HSCs) from bone marrow, blood, umbilical cord blood, and regenerative medicine products such as mesenchymal stem cells (MSCs), chondrocytes, pancreatic islet cells, and other somatic cell therapies. Prior to founding Advanced Cell & Gene Therapy in 2002, Dr. Burger was Vice-President for R&D at Merix Bioscience, and director of the University of Minnesota Cell Therapy Clinical Laboratory. His regulatory background includes numerous IND and IDE submissions and productive interactions with FDA-CBER Office of Tissues and Advanced Therapies (OTAT, formerly the Office of Cellular, Tissue and Gene Therapies, OCTGT). Dr. Burger is a member of several scientific advisory boards, and has served on the USP Cell, Gene and Tissue Therapies Expert Committee, the ISCT advisory board and ISCT committees on gene therapy, regulatory affairs, commercialization, and product/process development. A graduate of the University of Pennsylvania School of Medicine, Dr. Burger completed training in clinical pathology and transfusion medicine at Washington University in St. Louis, and is author of over 200 scientific publications and presentations, and recipient of numerous honors and awards.

Funding & Partnering
Moderator: Alain Vertes, NxR Biotechnologies GmbH
2:30
Advancing Stem Cell Research in California and Beyond
 
Gil Sambrano
Gil Sambrano
Vice President of Portfolio Development and Review
California Institute for Regenerative Medicine
About Speaker: Gil leads the team responsible for selecting the highest quality stem cell-based projects for CIRM funding from Discovery to Clinical programs.  His team builds and cultivates a world class team of expert reviewers and directs a rigorous review proc... Read Full Bio 
 
 
Gil Sambrano
Vice President of Portfolio Development and Review
California Institute for Regenerative Medicine
 
About Speaker:

Gil leads the team responsible for selecting the highest quality stem cell-based projects for CIRM funding from Discovery to Clinical programs.  His team builds and cultivates a world class team of expert reviewers and directs a rigorous review process through the governing board-appointed Grants Working Group (GWG).

Gil joined CIRM in 2005 as the first Scientific Officer. During his tenure, Gil has contributed to building the vision of CIRM by leading and advancing the scientific review process, constructing grants administration policies, guiding early development of the Grants Management System, and managing the training grant programs. He has been a key point of contact to help applicants and grantees identify appropriate partnering opportunities and navigate the CIRM solicitation and application process.  He leads the conduct of GWG review meetings and has been the primary liaison with patient advocate and scientific members of the GWG.

Prior to CIRM, he was an assistant professor in the department of Cellular and Molecular Pharmacology at UCSF. In 2001, Gil took on a notable position to coordinate efforts of the Alliance for Cellular Signaling, a multi-institutional and multi-disciplinary consortium of scientists led by the Nobel laureate, Alfred G. Gilman, whose goal is to understand the basic principles that regulate signal transduction in cells.

His scientific education includes a B.S. in biology from the University of Texas at El Paso and a Ph.D. in biomedical sciences from the University of California, San Diego. Gil trained as a postdoctoral fellow at the Cardiovascular Research Institute at the University of California San Francisco.

 
Abstract: The California Institute for Regenerative Medicine (CIRM) was established in 2004 by the passage of a $3 billion State...Read More 

The California Institute for Regenerative Medicine (CIRM) was established in 2004 by the passage of a $3 billion State bond initiative, Proposition 71, with the mission to accelerate stem cell treatments to patients with unmet medical needs.

While overcoming two constitutional challenges to Proposition 71, CIRM initially relied on a loan of $150 million approved by then Governor Schwarzenegger and $35 million in contingent bond anticipation notes, to hire a skeleton staff to begin the process of building a research funding agency from scratch.

Since that time, CIRM has progressively advanced to build a powerful stem cell engine with increased speed, coordination, and focus. The result is a more rigorous process for vetting the best ideas and developing a pipeline of funding opportunities that seamlessly support stem cell-based projects from early discovery through the clinical trial stages. CIRM 2.0 was launched with streamlined processes and expanded reach that includes funding for entities located outside the State of California.

An overview of CIRM’s progress, process, and opportunities for partnering will be presented.

 Read Less
2:50
A Partnering Model for Sustainable Funding
 
Spencer Hoover
Spencer Hoover
Development Manager
CCRM
About Speaker: Spencer Hoover is a development manager at CCRM, a Canadian, not-for-profit organization supporting the development of foundational technologies for commercializing cell and gene therapies. His current focus is on business development and technical ... Read Full Bio 
 
 
Spencer Hoover
Development Manager
CCRM
 
About Speaker:

Spencer Hoover is a development manager at CCRM, a Canadian, not-for-profit organization supporting the development of foundational technologies for commercializing cell and gene therapies.

His current focus is on business development and technical projects directed towards PSC and viral vector manufacture for immunotherapy.

Prior to joining CCRM, Dr. Hoover spent five years developing FDA-cleared multiplex molecular diagnostics for Luminex Corporation in the US and Canada. 

He completed a B.S. in chemistry at the University of Louisville and a Ph.D. in microbiology (focus on virology) at the University of Wisconsin – Madison.  He was then a postdoctoral fellow in the chemical engineering department at UW-Madison (focus on metabolic engineering). 

 
Abstract: Sustainable partnering can be achieved through multiple partnering approaches involving industry, academia and government sources.  The Centre...Read More 

Sustainable partnering can be achieved through multiple partnering approaches involving industry, academia and government sources.  The Centre for Advanced Therapeutic Cell Technologies (CATCT) is a collaboration between GE, CCRM and the Federal Economic Development Agency for Southern Ontario, combining public and private funds to accelerate the industrialization of cellular and gene therapies.  The CATCT seeks to build a sustainable partnership by identifying collaborators with complementary capabilities and then focusing on the synergistic needs of each partner, namely regional economic and job growth for FedDev, market development and technology commercialization for GE, and expertise development and long-term funding support for CCRM.

 Read Less
3:10
Afternoon Networking Break
Stem Cells for Drug Discovery & Disease Modeling
Moderator: Yang (Ted) D. Teng, Harvard University and Spaulding Rehabilitation Hospital
3:55
Using Stem Cells to Model Neurological Disease & Advance Personalized Medicine
 
Evan  Snyder
Evan Y. Snyder
Professor and Director, Program in Stem Cell & Regenerative Biology
Sanford-Burnham Medical Research Institute
About Speaker: Evan Y. Snyder, who is regarded as one of the “fathers of the stem cell field”, earned his M.D. and Ph.D. (in neuroscience) from the University of Pennsylvania and received training in Philosophy, Pscyhology, and Linguistics at Oxford University.... Read Full Bio 
 
 
Evan Y. Snyder
Professor and Director, Program in Stem Cell & Regenerative Biology
Sanford-Burnham Medical Research Institute
 
About Speaker:

Evan Y. Snyder, who is regarded as one of the “fathers of the stem cell field”, earned his M.D. and Ph.D. (in neuroscience) from the University of Pennsylvania and received training in Philosophy, Pscyhology, and Linguistics at Oxford University. He began his career as a physician-scientist at Harvard Medical School and Boston Children’s Hospital where, in addition to running a lab, remained clinically active in pediatric neurology and newborn intensive care medicine. After 23 years at Harvard, he was recruited to the Sanford-Burnham-Prebys Medical Discovery Institute and UCSD as Professor and founding director of the Center for Stem Cells and Regenerative Medicine and the Stem Cell Research Center and Core Facility. His ab studies the basic and translational biology of stem cells (particularly neural) with the goal of understanding normal and aberrant development, tissue homeostasis and plasticity, inter-cellular communication, oncogenesis, and recovery of function as well as using stem cells to model disease for pathway mapping and drug discovery.

 
Abstract: Being able to model development & disease “in a dish” for a specific patient is the future of medicine...Read More 

Being able to model development & disease “in a dish” for a specific patient is the future of medicine – “personalized” or “precision” medicine. It is envisioned that every individual will someday have a “safety deposit box” of their own functional pluripotent stem cells which can be queried for molecular data or expanded for cell-based therapies when differentiated down a particular lineage) as well as an “omics” database that can be mined by health care providers and researchers lifelong. Such a resource will also be the key to drug discovery, toxicity testing, and pathway analysis, not only for the patient in question but perhaps for a given disease more broadly if a sufficient number of patients are profiled. These approaches may well be the “lowest-hanging fruit” in the stem cell field.

Human induced pluripotent stem cells (hiPSCs) have the potential to serve as in vitro models of disease for the purpose of (a) unveiling novel pathophysiological mechanisms; (b) pinpointing biomarkers, prognostics, & diagnostics; (c) elucidating the mechanisms-of-action of extant drugs; (d) revealing novel drug targets; (e) potentially discovering new drugs. These self-renewing pluripotent cells & their derivatives are particularly useful for (i) providing a range of cell types that are otherwise difficult to obtain & scale-up from a specific living patient; (ii) deciphering cellular & molecular differences between patients suffering from a given disease vs. those individuals who are unaffected or affected by another condition; (iii) discerning differences between cells treated under 1 condition vs. another. The value of hiPSCs is dramatically enhanced by being able to map the intracellular signaling pathways responsible for the disease emulating phenotypes they evince. Such maps can then help pinpoint drug targets (often novel & unanticipated), &, in turn, expedite the discovery of new drugs while avoiding drugs that may have off-target toxicities. This approach has the potential to transform the field of pharmacotherapeutics.

In this talk, I will briefly discuss how the stem cell field can contribute to the advancement of personalized medicine as well as focus on how hiPSCs may be used to model the most challenging  kinds of diseases – those for which genetic defects or cellular abnormalities have not yet been defined but are rather polygenic, multifactorial, and complex, for example, neuropsychiatric disorders.

 Read Less
4:35
Defining Recovery Neurobiology of Injured Spinal Cord by Stem Cell-based Multimodal Approaches
 
Yang (Ted) D Teng
Yang (Ted) D Teng
Director, Laboratory of SCI, Stem Cell & Neurofacilitation Research PM&R and Neurosurgery
Harvard University and Spaulding Rehabilitation Hospital
About Speaker: After receiving his Medical Degree and Master of Science in Neuropharmacology, Dr. Teng earned his Ph.D. degree in Cell Biology/Neuroscience at Georgetown University, Washington DC, USA.  For his postdoctoral training, he focused on respiratory neur... Read Full Bio 
 
 
Yang (Ted) D Teng
Director, Laboratory of SCI, Stem Cell & Neurofacilitation Research PM&R and Neurosurgery
Harvard University and Spaulding Rehabilitation Hospital
 
About Speaker:

After receiving his Medical Degree and Master of Science in Neuropharmacology, Dr. Teng earned his Ph.D. degree in Cell Biology/Neuroscience at Georgetown University, Washington DC, USA.  For his postdoctoral training, he focused on respiratory neurobiology, and stem cell biology and neurodegeneration at Georgetown University and Harvard Medical School, respectively.  He is presently Director, Laboratory of Spinal Cord Injury, Stem Cell & Neurofacilitation Research, Departments of Physical Medicine & Rehabilitation and Neurosurgery (for which he holds professorships), Harvard Medical School/Spaulding Rehabilitation Hospital/Brigham & Women’s Hospital, and Director, Division of Spinal Cord Injury Research, VA Boston Healthcare System.  Dr. Teng studies Functional Multipotency of Stem Cells and Recovery Neurobiology of the Spinal Cord through multimodal cross-examination approaches that integrate stem cell biology, neural and glial biology, chemical or genetic engineering, molecular pharmacology, and spinal cord oncology.  Work of his team has received the prestigious 2011 Apple Award of the American Spinal Injury Association (ASIA), the ERF New Investigator Award from the Foundation of Physical Medicine & Rehabilitation (2004), and the Mayfield Awards and Larson Research Award from the Congress of Neurological Surgeons and American Association of Neurological surgeons (CNS/AANS) Joint Section on Disorders of the Spine and Peripheral Nerves (2012, 2015 and 2016).  Dr. Teng has been serving as a reviewer for more than 50 academic and clinical journals, and working as a study section member for federal and private funding agencies as well as for European Union academic organizations. He is elected the Secretary (2014-2016) and President (2013-2014) of the American Society for Neural Therapy and Repair (www.asntr.org).

 
Abstract: Emerging evidence increasingly suggests that stem cells may help repair the central nervous system through multiple me...Read More 

Emerging evidence increasingly suggests that stem cells may help repair the central nervous system through multiple mechanistic strategies that are often concurrent (i.e., Functional Multipotency of Stem Cells). They may serve not only as tissue engineering mediators of cellular reconstitution, but also as vectors for the delivery of molecules and genes. We have now developed a platform technology to determine therapeutic mechanisms of human mesenchymal stromal stem cells (hMSCs) in a dorsal root ganglion coculture system and an intraspinal cord implantation model. The unique poly(lactic-co-glycolic) acid scaffolding augments hMSC stemness, engraftment, and function without neural transdifferentiation or mesenchymal lineage development, resulting in robust motosensory improvement, pain and tissue damage mitigation, and myelin preservation in adult rat spinal cord after injury. The scaffolded hMSC-derived neurotrophism, neurogenesis, angiogenesis, antiautoimmunity, and antiinflammation support the propriospinal network, neuromuscular junctions, and serotonergic reticulospinal reinnervation to activate the central pattern generator for restoring hindlimb locomotion. Our findings illuminate “Recovery Neurobiology” — i.e., the injured spinal cord may deploy polysynaptic neural circuits different from normal adulthood pathways for postinjury improvement. I will discuss that how tailored polymer implants containing hMSCs or human neural progenitor cells (hNPCs) may hold significant promise for providing a broad range of insight regarding essential neurological mechanisms required for repairing the adult mammalian spinal cord after injury. Our findings may provide a stem cell-based multimodal approach to investigating and formulating therapeutic strategies to achieve clinically meaningful improvement for SCI and neurodegenerative diseases.

 Read Less
4:55
Day Concludes
Day - 2 Friday, April 7th, 2017
7:30
Continental Breakfast
Manufacturing and Scalable Processes
Moderator: Jon A. Rowley, RoosterBio Inc
8:00
Scale Up of Pluripotent Stem Cell (PSC) Manufacturing
 
Gary Pigeau
Gary Pigeau
Development Manager, Cell Therapy
GE Healthcare & CCRM (BridGE)
About Speaker: Gary Pigeau received his Ph.D. in Biotechnology from Brock University in St. Catharines, Ontario. He completed a postdoctoral fellowship at the University of Alberta with a visiting scientist appointment at the University of Oxford. Gary moved to pri... Read Full Bio 
 
 
Gary Pigeau
Development Manager, Cell Therapy
GE Healthcare & CCRM (BridGE)
 
About Speaker:

Gary Pigeau received his Ph.D. in Biotechnology from Brock University in St. Catharines, Ontario. He completed a postdoctoral fellowship at the University of Alberta with a visiting scientist appointment at the University of Oxford. Gary moved to private industry in 2008, where his research group focused on process development and scale-up of a proprietary bioprocess technology. He then moved to commercial, large-scale biomanufacturing in 2013. Gary joined GE Healthcare and BridGE @ CCRM in 2016 and leads the pluripotent stem cell process development, scale-up and manufacturing efforts.

 
 
Abstract: Human PSCs can serve as an input cell source for the generation of differentiated cell derivatives for drug screening,...Read More 

Human PSCs can serve as an input cell source for the generation of differentiated cell derivatives for drug screening, disease modelling, and cellular therapies. Laboratory scale PSC culture strategies use anchorage-dependent systems and demand a high level of operator handling to maintain cell quality.  There has yet to be a demonstration of a truly scalable solution for PSC production. As the field is currently working at the scale of approximate 1 L, a suitable solution must enable scaling to production in the 1–10 L range to accommodate near-term needs, while providing a platform that can be readily adapted to scales of 10-1,000 L.  Progress towards these goals by BridGE @ CCRM is presented.

 Read Less
8:25
Large Scale Manufacturing of Cell-Based Commercial Products
 
Zorina Pitkin
Zorina Pitkin
Vice President, Quality Systems
Organogenesis
About Speaker: Dr. Zorina Pitkin is Senior Vice President of Quality Systems at Organogenesis Inc., a regenerative medicine company focused on development and commercialization of both cellular and acellular therapies in the areas of bio-active wound healing and bi... Read Full Bio 
 
 
Zorina Pitkin
Vice President, Quality Systems
Organogenesis
 
About Speaker:

Dr. Zorina Pitkin is Senior Vice President of Quality Systems at Organogenesis Inc., a regenerative medicine company focused on development and commercialization of both cellular and acellular therapies in the areas of bio-active wound healing and bio-surgery.

She has over 20 years of extensive experience in the field of biotechnology including the development of Quality System programs for autologous and allogeneic cellular therapies, biologic/device combination products, and for xenotransplantation. Prior to joining Organogenesis in 2008, Zorina held senior management positions at RenaMed Biologics and Circe Biomedical, the two Companies developing bioartificial organs and therapeutic cell systems for treatment of acute renal failure, and acute liver failure and diabetes, respectively; and before then, a variety of positions of increased responsibility at Cellcor, a Company developing autologous cell-based treatment for renal cell carcinoma and Hepatitis B.

Dr. Pitkin has published 16 original papers in professional journals and has presented at numerous national and international conventions. She holds a Ph.D. in Biological Sciences from the Research Institute of Influenza, Russian Academy of Medical Sciences, and is Regulatory Affairs certified.

 
Abstract: One of the main challenges in cell-based manufacturing is the transition from a pilot to commercial scale production a...Read More 

One of the main challenges in cell-based manufacturing is the transition from a pilot to commercial scale production and the ability to sustain large scale manufacturing at a low cost.  This presentation will provide an overview of the two cell-based products produced by Organogenesis at a commercial scale, Apligraf® and Dermagraft®. Both products are approved by the FDA as class III medical devices to treat diabetic foot ulcers, and Apligraf is approved to treat venous leg ulcers. To date, more than a million units of Apligraf and Dermagraft have been shipped to patients.  We will review the scale-up manufacturing process for Apligraf and we will discuss the role of keratinocyte stem cells used in the manufacturing process to create functional cell-based products.

 Read Less
8:50
A Presentation of Rubius Therapeutics Red Cell Therapeutics Technology
 
Robert Konopacz
Robert Konopacz
Vice President of Manufacturing
Rubius Therapeutics
About Speaker: Robert Konopacz, is responsible for manufacturing and bioprocess development at Rubius Therapeutics, based on his background in Gene Therapy and Biological Manufacturing. Robert has more than 35 years of experience in Biotechnology Therapeutics whic... Read Full Bio 
 
 
Robert Konopacz
Vice President of Manufacturing
Rubius Therapeutics
 
About Speaker:

Robert Konopacz, is responsible for manufacturing and bioprocess development at Rubius Therapeutics, based on his background in Gene Therapy and Biological Manufacturing.

Robert has more than 35 years of experience in Biotechnology Therapeutics which includes work with virus and T cell processing, mammalian, bacterial and yeast expression systems, blood fractionation, and transgenic animals. He most recently served as Head of Advanced Medicines Relationship Management for Novartis AG Global Technical Operations supporting their Cart 19 program.

Prior to Novartis, Robert was Senior Director of Manufacturing at Genetics Institute (Pfizer) responsible for the manufacturing of recombinant Factor VIII, BMP-2, and IL-11.  Robert was an original manufacturing team member at Genentech, developing and launching hGH and tPA.

He has also held positions of increasing responsibility at Chiron, and Baxter Healthcare.

Robert holds has a B.Sc. in Chemical Engineering and a B.Sc. in Microbiology from the Michigan State University.

 
Abstract: Rubius Therapeutics is Pioneering the Creation of The Red-Cell Therapeutics Drug Class. This presentation will provide...Read More 

Rubius Therapeutics is Pioneering the Creation of The Red-Cell Therapeutics Drug Class. This presentation will provide an overview of the application of this new platform technology that covers a broad therapeutic space delivering unique benefits to patients.

 Read Less
9:15
Building Cell Supply Chain How to Standardize Collection, Apheresis Expertise, Cell Delivery
 
Beth Shaz
Beth Shaz
Chief Medical and Scientific Officer, Comprehensive Cell Solutions
New York Blood Center
About Speaker: Beth is Chief Medical and Scientific Officer, Senior Vice President at New York Blood Center, and Adjunct Assistant Professor, Department of Pathology and Cell Biology, Columbia University Medical Center. Beth is leading NYBC’s Comprehensive Cell S... Read Full Bio 
 
 
Beth Shaz
Chief Medical and Scientific Officer, Comprehensive Cell Solutions
New York Blood Center
 
About Speaker:

Beth is Chief Medical and Scientific Officer, Senior Vice President at New York Blood Center, and Adjunct Assistant Professor, Department of Pathology and Cell Biology, Columbia University Medical Center. Beth is leading NYBC’s Comprehensive Cell Solutions. CCS is focused on developing, improving, and implementing regenerative medicine, cell therapies, transfusion medicine, and related scientific and medical endeavors. Previously, she was Associate Professor at Emory University School of Medicine and director of the transfusion service at Grady Memorial Hospital. Also, she was associate director of the transfusion service at Beth Israel Deaconess Medical Center, Harvard Medical School. Beth received her Medical Degree with research distinction from University of Michigan and Bachelor of Science in chemical engineering with distinction from Cornell University. She did a general surgery internship at Georgetown University, an anatomic & clinical pathology residency at Beth Israel Deaconess Medical Center, and a transfusion medicine fellowship at Harvard Medical School.

 
Abstract: The cell therapy area has rapidly expanded. The ability to supply high quality cells and other manufacturing requirements is critical to the succes...Read More 

The cell therapy area has rapidly expanded. The ability to supply high quality cells and other manufacturing requirements is critical to the success of these products. Companies should select partners who understand apheresis collection, donor management, including venous access and personalized care, nursing experience, tailoring procedures to each cell therapy product, quality management systems, flexibility, location, and the regulatory environment. This presentation addresses key pieces of creating a high quality, standardized, and robust cell supply chain.

 Read Less
9:40
Thinking Outside the Vial: Plug & Play Stem Cell Systems for Rapid Manufacturing Process Development
 
Jon Rowley
Jon A. Rowley
Chief Technology Officer
RoosterBio Inc
About Speaker: Jon A. Rowley, PhD, is the Founder and Chief Technology Officer of RoosterBio Inc and has a personal goal of making significant contributions to the commercial translation of living cellular technologies. Jon holds a PhD from the University of Michig... Read Full Bio 
 
 
Jon A. Rowley
Chief Technology Officer
RoosterBio Inc
 
About Speaker:

Jon A. Rowley, PhD, is the Founder and Chief Technology Officer of RoosterBio Inc and has a personal goal of making significant contributions to the commercial translation of living cellular technologies. Jon holds a PhD from the University of Michigan in Biomedical Engineering and has authored over 30 peer reviewed manuscripts and 15 issued or pending patents related to biomaterials development, tissue engineering, and cellular therapy. He most recently spent 5 years as Director of Innovation and Process Development in Lonza’s Cell Therapy CMO business, and currently resides in Walkersville, MD with his wonderful wife and their 3 young children.

 
Abstract: Mesenchymal Stem Cells are the workhorse of Regenerative Medicine, but have met with challenges in scale up and COGS. We have been developing ready...Read More 

Mesenchymal Stem Cells are the workhorse of Regenerative Medicine, but have met with challenges in scale up and COGS. We have been developing ready-to-use cellular starting cell stocks (cell banks) and bioprocess media systems so that anyone can quickly implement highly sophisticated, scalable, and economical MSC production processes. These “Plug & Play” systems are radically shortening the product development timelines for Regenerative Medicine products that incorporate stem cells.

The Key Learnings will be

1) The technology roadmap for scalable manufacturing of adherent stem cells

2) the importance of proper analytics for monitoring comparability during major process changes

3) The impact of manufacturing platforms (flasks vs scaled up planar surfaces vs 3D suspension) on the COGs of stem cell related processes.

4) new products within the industry focused on rapidly implementing cell therapy manufacturing processes

 Read Less
10:05
Morning Networking Break
Biotech Showcase: Emerging Pipelines
Moderator: Robert Deans, BlueRock Therapeutics
10:35
Universal Donor Stem Cells
 
David Russell
David Russell
Chief Scientific Officer
Universal Cells Inc.
About Speaker: Human pluripotent stem cells (PSCs) have the potential to treat diseases affecting almost every organ system. However, the clinical use of PSC-derived products is limited by allogeneic rejection, primarily due to differences in the diverse human leuk... Read Full Bio 
 
 
David Russell
Chief Scientific Officer
Universal Cells Inc.
 
About Speaker:

Human pluripotent stem cells (PSCs) have the potential to treat diseases affecting almost every organ system. However, the clinical use of PSC-derived products is limited by allogeneic rejection, primarily due to differences in the diverse human leukocyte antigen (HLA) genes, and the use of autologous induced PSCs or the establishment of HLA-typed PSC banks are problematic due to the large number of cGMP-grade cell lines that must be prepared, characterized, and approved by regulatory agencies. Here I will describe an approach for generating universal donor PSCs, which will allow a single PSC-derived cell product to be used in multiple recipients. Gene editing with recombinant adeno-associated virus vectors is used to efficiently alter genes involved in HLA expression, without the use of potentially genotoxic nucleases. Through this process, we eliminate cell surface expression of both HLA class I and class II molecules, which prevents peptide presentation to T cells and recognition by anti-HLA antibodies. Gene editing is also used to reintroduce a non-polymorphic class I molecule and thereby prevent lysis by Natural Killer cells. We show that these HLA-engineered universal donor cells resist allogeneic responses of NK, B and T cells, both in vitro and in vivo in humanized mouse models. Universal donor PSCs can be differentiated into diverse therapeutic cell products that are compatible with all recipients, and they allow the production of off-the-shelf cellular therapy products for many indications.

 
Abstract: Human pluripotent stem cells (PSCs) have the potential to treat diseases affecting almost every organ system. However, the clinical use of PSC-deri...Read More 

Human pluripotent stem cells (PSCs) have the potential to treat diseases affecting almost every organ system. However, the clinical use of PSC-derived products is limited by allogeneic rejection, primarily due to differences in the diverse human leukocyte antigen (HLA) genes, and the use of autologous induced PSCs or the establishment of HLA-typed PSC banks are problematic due to the large number of cGMP-grade cell lines that must be prepared, characterized, and approved by regulatory agencies. Here I will describe an approach for generating universal donor PSCs, which will allow a single PSC-derived cell product to be used in multiple recipients. Gene editing with recombinant adeno-associated virus vectors is used to efficiently alter genes involved in HLA expression, without the use of potentially genotoxic nucleases. Through this process, we eliminate cell surface expression of both HLA class I and class II molecules, which prevents peptide presentation to T cells and recognition by anti-HLA antibodies. Gene editing is also used to reintroduce a non-polymorphic class I molecule and thereby prevent lysis by Natural Killer cells. We show that these HLA-engineered universal donor cells resist allogeneic responses of NK, B and T cells, both in vitro and in vivo in humanized mouse models. Universal donor PSCs can be differentiated into diverse therapeutic cell products that are compatible with all recipients, and they allow the production of off-the-shelf cellular therapy products for many indications.

 Read Less
11:00
Sentien Biotechnologies: Developing a Novel Approach to Cell Therapy
 
Brian  Miller
Brian Miller
Chief Executive Officer
Sentien Biotech
About Speaker: Brian Miller brings over 18 years of experience in the life sciences industry, currently serving as Co-Founder and CEO of Sentien Biotechnologies, a regenerative medicine company developing cell based devices for organ failure. Mr. Miller has previou... Read Full Bio 
 
 
Brian Miller
Chief Executive Officer
Sentien Biotech
 
About Speaker:

Brian Miller brings over 18 years of experience in the life sciences industry, currently serving as Co-Founder and CEO of Sentien Biotechnologies, a regenerative medicine company developing cell based devices for organ failure. Mr. Miller has previously held the roles of Executive Director and Head, Corporate Development at FORMA Therapeutics, a clinical stage oncology company, and Head of Business Development – Asia Pacific Region for Quosa, Inc., a life science informatics company, where he led the commercial launch of their product line in the Asia Pacific region. He began his biotech career at Eli Lilly and Company, where he led the identification, evaluation, and in-licensing of research informatics technologies in the Global External Research & Development group. Brian received BA degrees in Chemistry and Business from Goshen College, an MS degree in Health Sciences from the Harvard-MIT Division of Health Sciences Technology, and an MBA from the MIT Sloan School of Management.

 
Abstract: Sentien Biotechnologies, Inc. is a privately owned, early clinical stage company developing novel approaches to cell therapy. Our lead product is d...Read More 

Sentien Biotechnologies, Inc. is a privately owned, early clinical stage company developing novel approaches to cell therapy. Our lead product is designed to allow for controlled, sustained delivery of mesenchymal stromal cell (MSC) secreted factors. Our approach immobilizes the MSCs in an extracorporeal device, allowing for doses of the therapeutic secreted factors that are unattainable by direct injection.

 Read Less
Round Table Discussions
Round Table 1: Gene Editing of Cell Therapeutics
Chair: Robert Deans, Chief Technology Officer, BlueRock Therapeutics
Round Table 2: Skills Required for Translation of Cell Therapies from Bench to Bedside
Co-Chair: Khalid Shah, Director Center for Stem Cell Therapeutics & Imaging, Harvard Medical School
Co-Chair: Bill Lundberg, Chief Scientific Officer, CRISPR Therapeutics
Round Table 3: Regulation of Cell Therapies Evolving Across the Globe
Chair: William Sietsema, Executive Director, Global Regulatory Affairs, Caladrius Biosciences
12:15
Lunch Provided by GTCbio
Commercialization: Creating Sustainable Business Models
Moderator: Robert Deans, BlueRock Therapeutics
1:30
What’s Next?: Technology and Competition
 
Matthew Durdy
Matthew Durdy
Chief Business Officer
Cell Therapy Catapult
About Speaker: Part of the team that created the Catapult, he is also an Executive Director. He is responsible for finding, funding and transacting the business of the Catapult and has been a champion of the early integration of healthcare economics and reimburseme... Read Full Bio 
 
 
Matthew Durdy
Chief Business Officer
Cell Therapy Catapult
 
About Speaker:

Part of the team that created the Catapult, he is also an Executive Director. He is responsible for finding, funding and transacting the business of the Catapult and has been a champion of the early integration of healthcare economics and reimbursement expertise into decision- making and clinical product design.  He began his career in international investment banking and has successfully managed a number of SMEs in the biotechnology sector.   His first degree was from Oxford University in Biology and he has an MBA (High Honors) from Chicago Booth. He is a non-executive director of two immune-oncology companies and periodically assists the UK Government and international organisations in the development of initiatives in healthcare innovation.

 
Abstract: The cell and gene therapy industry has made a remarkable transition over the last few years and companies are now preparing for a world where produ...Read More 

The cell and gene therapy industry has made a remarkable transition over the last few years and companies are now preparing for a world where products reach the market in increasing numbers: a world with competition.  Companies often do not have the resources to work beyond their immediate needs and so miss an opportunity to shape their future. The Cell and Gene Therapy Catapult has researched the industry’s view of its structure in the future. This creates an agenda for technological development which will give participants advantage.  This presentation will explore the opportunities that this view of the future creates.

 Read Less
1:55
Stem Cell Product Development and Commercialization
 
Hans Keirstead
Hans Keirstead
Chief Executive Officer
AIVITA BIOMEDICAL
About Speaker: Dr. Keirstead is an internationally known stem cell expert and has led therapy development for late stage cancers, immune disorders, motor neuron diseases, spinal cord injury and retinal diseases. He is the CEO of AIVITA Biomedical, an Irvine, CA-bas... Read Full Bio 
 
 
Hans Keirstead
Chief Executive Officer
AIVITA BIOMEDICAL
 
About Speaker:

Dr. Keirstead is an internationally known stem cell expert and has led therapy development for late stage cancers, immune disorders, motor neuron diseases, spinal cord injury and retinal diseases. He is the CEO of AIVITA Biomedical, an Irvine, CA-based company focused on the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. As Full Professor of Anatomy and Neurobiology at the University of California at Irvine he founded and directed the Sue and Bill Gross Stem Cell Research Center. He has been a long-time advisor to several governments on biomedical policy, and was a founding advisor of the California Stem Cell Initiative that resulted in a $3 billion stem cell fund (CIRM).

 
Abstract: AIVITA Biomedical has developed proprietary methods for the scalable production of differentiated human cells in high-...Read More 

AIVITA Biomedical has developed proprietary methods for the scalable production of differentiated human cells in high-purity from human stem cells. This has enabled the Company to generate virtually unlimited quantities of various cell types for therapeutic application. In addition, the Company has leveraged and adapted its proprietary cell culture techniques to explore new areas of commercial opportunity, including a novel skin care technology launched in February 2017.

The Company’s lead therapeutic candidate, an autologous dendritic-cell/tumor-cell cancer immunotherapy with platform applicability, represents more than a decade of cancer research combined with AIVITA’s proprietary manufacturing expertise. AIVITA has made successive advancements in the methods used to manufacture its patient-specific treatment, rendering it more economical to produce and thus more commercially viable.

AIVITA’s skin care technology was made possible through the Company’s expertise in stem cell growth and high-purity differentiation. Leveraging this expertise in cell culture, AIVITA can generate unstressed populations of human skin progenitor cells, from which it can capture the complete milieu of cell secretions directly relevant to the growth and maintenance of young human skin. The result is a technology which effectively mimics the environment in which young developing skin thrives.

Profits from the sale of AIVITA’s skin care products directly support its therapeutic development pipeline, including its upcoming Phase II trial in ovarian cancer. The introduction of AIVITA’s aesthetics technology as a revenue source allows the Company more freedom to pursue its long-term therapeutic development goals.

 Read Less
2:45
Product Delineation in Allogeneic Cytotherapies
 
Alain Vertès
Alain Vertès
Managing Director
NxR Biotechnologies GmbH
About Speaker: Dr Alain Vertès is a Sloan Fellow from London Business School and a microbiologist by training (University of Illinois at Urbana-Champaign, Institut Pasteur Paris, University of Lille Flandres-Artois). He is a strategy and business development consu... Read Full Bio 
 
 
Alain Vertès
Managing Director
NxR Biotechnologies GmbH
 
About Speaker:

Dr Alain Vertès is a Sloan Fellow from London Business School and a microbiologist by training (University of Illinois at Urbana-Champaign, Institut Pasteur Paris, University of Lille Flandres-Artois). He is a strategy and business development consultant and works to enable funding and partnering in biotechnology. Focusing on technology deployment and innovation commercialization, he has contributed to both white (industrial) and red (pharmaceutical) biotechnology, in different functions including research, manufacturing, contract research, and strategic alliances in pharmaceuticals (Lilly, Pfizer, Roche), biotechnology (Mesoblast), petrochemicals (Mitsubishi Chemical Corporation), public research, and consulting (Australian Strategic Policy Institute, NxR Biotechnologies GmbH). Particularly, he has long been associated with the Research Institute of Innovative Technology for the Earth in Kyoto, Japan, working on developing transformational biorefinery technologies and on deploying green technologies to the marketplace. In the pharmaceutical industry, he champions radical innovation for bringing to patients disease-modifying, paradigm-changing therapeutics such as siRNA, cell- or gene-based pharmaceuticals.

3:10
Mapping the European Landscape for Patenting Stem Cell Related Inventions
 
Aliki Nichogiannopoulou
Aliki Nichogiannopoulou
Director, Biotechnology
European Patent Office
About Speaker: Aliki Nichogiannopoulou was born in Athens, Greece. She studied biology and philosophy at the Albert-Ludwigs-University in Freiburg, Germany and did her diploma thesis in molecular immunology at the Max-Planck-Institute of Immunology in Freiburg. She... Read Full Bio 
 
 
Aliki Nichogiannopoulou
Director, Biotechnology
European Patent Office
 
About Speaker:

Aliki Nichogiannopoulou was born in Athens, Greece. She studied biology and philosophy at the Albert-Ludwigs-University in Freiburg, Germany and did her diploma thesis in molecular immunology at the Max-Planck-Institute of Immunology in Freiburg. She completed her Ph.D. thesis on adult, fetal and embryonic stem cells in the Department of Genetics at Harvard Medical School in Boston, and did her post-doctoral research on stem cells at Harvard Medical School at the Massachusetts General Hospital. She became a patent examiner at the European Patent Office in September 1998 and joined the Patent Law department in 2004. She was holding a joint appointment in the two departments until December 2009. She participated in all major stem cell cases at the European Patent Office and represented the President of the European Patent Office in front of the Office's Enlarged Board of Appeal in the Thomson case on human embryonic stem cells. She has represented the Office in patent law, scientific and ethical conferences and workshops and has lectured on the legal and ethical aspects of stem cell and DNA patenting at several occasions. In January 2010 she was appointed director in Biotechnology at the European Patent Office. Since January 2017 she holds a position in the European patent office’s International Cooperation department.

 
Abstract: The European Patent Office is the patent granting authority for Europe. Patents are granted under the European Patent ...Read More 

The European Patent Office is the patent granting authority for Europe. Patents are granted under the European Patent Convention which lays down in its Article 53(a) that no European patents shall be granted in respect of inventions the commercial exploitation of which would be contrary to “ordre public” or morality. An additional rule of this convention exemplifies as inventions offending morality, all uses of human embryos for industrial or commercial purposes. Human embryonic stem cells originate from human embryos and this very fact presents an obstacle to their patentability as well as to the patentability of inventions relating thereto.

In this talk you will find:

  • the exact nature of this hurdle
  • the current state of European jurisdiction on the issue
  • the current case law of the Boards of Appeal of the European Patent Office
  • the possibilities for overcoming this hurdle under the above frameworks
 Read Less
3:35
Conference Concludes