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Cell & Gene Therapy

2017-11-182018-07-302018-06-30
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2017 Agenda
Day 1 - Monday, September 11th, 2017
7:00
Registration & Continental Breakfast
8:15
Welcome & Opening Remarks by Dr. Denis Rodgerson, Director of Science, GTCbio
10:00
Morning Networking Break
Development of Novel Technologies
Moderator: Mahendra Rao, New York Stem Cell Foundation
10:45
Development of AAV Products for Rare Diseases
 
Sam  Wadsworth
Sam Wadsworth
Chief Scientific Officer
Dimension Therapeutics
About Speaker: Dr. Wadsworth is the Chief Scientific Officer of Dimension Therapeutics, Inc., a leader in discovering and developing new therapeutic products for people living with devastating rare and metabolic diseases associated with the liver, based on the most... Read Full Bio 
 
 
Sam Wadsworth
Chief Scientific Officer
Dimension Therapeutics
 
About Speaker:

Dr. Wadsworth is the Chief Scientific Officer of Dimension Therapeutics, Inc., a leader in discovering and developing new therapeutic products for people living with devastating rare and metabolic diseases associated with the liver, based on the most advanced mammalian adeno-associated virus (AAV) gene delivery technology.  Dr. Wadsworth brings extensive drug discovery and development experience in the gene therapy and rare disease area. In his more than 20 years of industry experience at Genzyme and later Sanofi, he oversaw the discovery and translational research on multiple rare disease and gene therapy programs. Most recently before joining Dimension, he served as the head of gene therapy research and early development at Sanofi Genzyme. Previously, he served as group vice president for translational research at Genzyme. Dr. Wadsworth holds a Ph.D. from the University of Chicago and a B.A. from Southern Illinois University.

11:10
Next Generation Cell Therapies – Will These Revolutionize Healthcare
 
Devyn Smith
Devyn Smith
Chief Operating Officer
Sigilon
About Speaker: Devyn recently joined Sigilon, a biotechnology company, as its Chief Operating Officer tasked with building out strategy and operational elements of the company as it builds a portfolio of potential new medicines.  Prior to Sigilon, Devyn was part ... Read Full Bio 
 
 
Devyn Smith
Chief Operating Officer
Sigilon
 
About Speaker:

Devyn recently joined Sigilon, a biotechnology company, as its Chief Operating Officer tasked with building out strategy and operational elements of the company as it builds a portfolio of potential new medicines.  Prior to Sigilon, Devyn was part of Pfizer's Medicinal Sciences Division of R&D as Head of Business Operations & Strategy focused on both day to day business operations of the division, as well insuring implementable strategies are developed.  Medicinal Sciences encompasses the groups focused on discovery, formulation, and early manufacturing of the large molecule, small molecule, cell and gene therapy programs in Pfizer R&D.  Prior to this Devyn was Head of Strategy for the Pharmatherapeutics Division of Pfizer R&D focused on developing and implementing core strategies in Winning by Design in Small Molecules as well as optimizing the ROI on novel technology inventions.  Before this role, Devyn was part of Pfizer's Neusentis Research Unit in the UK as Chief Operating Officer responsible for strategy, operations and implementing key strategies.  In addition, he supported the Pharmatherapeutics Global Clinical Research Organization as the Chief Operating Officer.  He joined Pfizer's Strategic Management Group in August 2009.

Prior to joining Pfizer, Devyn was a principal for The Frankel Group (a boutique management consulting firm in New York City and Cambridge).  In his consulting experience, Devyn led a wide range of projects, across multiple therapeutic areas and a host of technology platforms, including basic R&D tools, regenerative medicine, gene therapy, and macromolecules/biologic products. Client relationships have ranged from large pharma to small biotech companies. Regenerative Medicine has been an area of strong focus with over 30 engagements, several published papers, and numerous invited conference presentations. Prior to joining The Frankel Group, Devyn worked as a management consultant at Adventis Corporation focused on clients in information intensive companies. 

Devyn received his Ph.D. in Genetics from Harvard Medical School where his research culminated in 12 publications in leading journals such as Cell, Nature, and Development.  He also holds an MS in Biology from Idaho State University and a BS in Zoology from Brigham Young University.

 
Abstract: New modalities are rapidly approaching potential regulatory approval, with additional next gen modalities in the pipeline.  There are many que...Read More 

New modalities are rapidly approaching potential regulatory approval, with additional next gen modalities in the pipeline.  There are many questions that remain unanswered, but determining how we to frame and potentially answer these questions is important.  Some questions include:  How will these therapies change the way diseases are treated?  How will we deal with new pricing models? How will the healthcare system evolve?  A framework for consideration of these questions will be discussed.

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11:35
Automated Closed System Production of Dendritic Cells
 
Shashi  Murthy
Shashi Murthy
Professor of Chemical Engineering
Northeastern University
About Speaker: Shashi Murthy is a Professor of Chemical Engineering and the Founding Director of the Michael J. and Ann Sherman Center for Engineering Entrepreneurship Education at Northeastern University. The core expertise of his lab is in cell purification a... Read Full Bio 
 
 
Shashi Murthy
Professor of Chemical Engineering
Northeastern University
 
About Speaker:

Shashi Murthy is a Professor of Chemical Engineering and the Founding Director of the Michael J. and Ann Sherman Center for Engineering Entrepreneurship Education at Northeastern University. The core expertise of his lab is in cell purification and processing for therapeutic use. Prof. Murthy has co-authored over 70 publications and is an inventor on 7 issued or pending patents. He previously co-founded Quad Technologies which has products for releasable magnetic bead separation of cells and T cell activation. Prof. Murthy is the recipient of the U.S. National Science Foundation’s Faculty Early Career Development (CAREER) Award and the Søren Buus Award for Outstanding Research in Engineering at Northeastern University. Prof. Murthy received his Ph.D. from MIT and B.S. from the Johns Hopkins University and is a Fellow of the American Institute of Medical and Biological Engineering (AIMBE).

 
Abstract: ...Read More 

DCs play an important role in emerging cell-based cancer immunotherapies ranging from antigen-pulsed autologous dendritic cell therapies to dendritic cell-stimulated autologous T cell therapies. For these and other research and discovery applications, DCs are typically obtained by the conversion of blood-derived monocytes using well-established protocols. In basic research and pre-clinical settings, these protocols are carried out manually, beginning with either the total peripheral blood mononuclear cell (PBMC) population or magnetic bead-enriched monocytes. The conversion process involves the transformation of plastic-adherent monocytes into non-adherent, large DCs. When carried out manually, this process can have high variability in yield and significant operator dependence. Such challenge can persist even in clinical manufacturing. This presentation will describe a new automated closed system, MicroDEN, for dendritic cell generation that overcomes these challenges. This system utilizes plastic adherence in exactly the same manner as manual culture but also includes medium perfusion in order to achieve the transformation of monocytes into DCs. Comparability between the automated system and manually generated DCs is demonstrated via phenotypic analysis and functional assays.

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12:00
Rapid, Efficient, Scale up/Scale out for any Cell Therapy Production Process
 
Kevin Murray
Kevin Murray
Vice President, Global Sales
BioSpherix Medical
About Speaker: Sales Manager for BioSpherix, Ltd. BS BioChemistry, MBA Finance & Marketing, 20+ Years experience in the Pharmaceutical/Biotech/Medical Research Industry.... Read Full Bio 
 
 
Kevin Murray
Vice President, Global Sales
BioSpherix Medical
 
About Speaker:

Sales Manager for BioSpherix, Ltd. BS BioChemistry, MBA Finance & Marketing, 20+ Years experience in the Pharmaceutical/Biotech/Medical Research Industry.

 
Abstract: ...Read More 

In order for cell therapies to be commercially successful and efficient, cost effective manufacturing must be utilized. Cleanrooms are simply not a viable option for the production of cell therapies. They are monolithic, time consuming to get up and running, extremely costly to operate, are not flexible in design, and sizing them for future scaling purposes is inefficient. “Rapid, Efficient, Scale up/Scale out for any Cell Therapy Production Process” is possible with the BioSpherix Medical platform.

• Scale around production bottlenecks with ease
• Flexible platform allows for efficient adaptation towards automation
• Distributed production brings your product closer to the patient
• Dramatically reduce production cost
• Remove contamination risk

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12:25
Sponsored Lunch Workshop
Lunch Workshop Presentation

12:25
Immunotherapy, Gene Editing, and the Lonza Nucleofector Technology
 
Gregory  Alberts
Gregory Alberts
Global Subject Matter Expert
Lonza
About Speaker: Dr. Gregory Alberts is currently the Global Subject Matter Expert for Lonza, and has worked at Lonza (and previously Amaxa) since 2003. He was the first field scientist hired by Amaxa in North America, and he has considerable experience in Nucleofect... Read Full Bio 
 
 
Gregory Alberts
Global Subject Matter Expert
Lonza
 
About Speaker:

Dr. Gregory Alberts is currently the Global Subject Matter Expert for Lonza, and has worked at Lonza (and previously Amaxa) since 2003. He was the first field scientist hired by Amaxa in North America, and he has considerable experience in Nucleofection, primary cells (including stem cells and iPSC generation), and is well versed in the use of CRISPR and other genome-editing technologies with Nucleofection. He received a Ph.D. from the George Washington University in 2003 in Molecular Biology, and an M.S. in Bacterial Genetics from the University of Illinois-Chicago in 1986.

On a practical note, Dr. Alberts worked at the American Red Cross Holland Laboratory for 14 years in basic vascular and cancer research, worked for three years at Oncor, Inc. in molecular cancer diagnostics, and spent four years working on bacterial genetics and DNA uptake in Streptococcus pneumoniae at University of Illinois at Chicago.

 
Abstract: Immunotherapy is an umbrella term that represents new hope in the fight against cancer and other diseases, by exploiting the body’s own immun...Read More 

Immunotherapy is an umbrella term that represents new hope in the fight against cancer and other diseases, by exploiting the body’s own immune system to fight disease, and was recognized by Science Magazine as the 2013 “Scientific Breakthrough of the Year”. The body’s immune system is an extremely powerful tool that can attack disease in fundamentally different ways than most conventional therapies, and with the advent of new scientific breakthroughs that can help the immune system specifically target diseases like cancer, there are myriad ways in which this new idea can be exploited, some of which will be explored in the course of this presentation.

The ability to modify the cells of the immune system is a key part of Immunotherapy. Lonza has the Amaxa Nucleofection platform, which is superb at the transfection of primary cells, and especially clinically relevant immune cells such as human T cells, CD34+ cells, mesenchymal stem cells, and iPSCs. Nucleofection can transfect these cell types very easily and reproducibly, with virtually any substrate, including genome-editing substrates such as CRISPR.Cas9 DNA, mRNA, and protein (RNP), and with high transfection efficiencies and viabilities. The Nucleofector platform is comprehensive and scalable, which makes it ideal for Immunotherapy research. The Nucleofector platform consists of the benchtop 4D Nucleofector, the 96-well Shuttle, the 384-well HT Nucleofector System, and the new large volume LV Nucleofector system.

The LV Nucleofector is the latest addition to the Nucleofector platform, and can transfect anywhere from 10 million cells up to 2 billion cells, with the same performance of the other Nucleofector devices. Because of the ability of Nucleofection to transfect primary cells so well, it is proven choice for iPSC generation and genome modification applications like CRISPR, ZFN, or TALENs, and is poised to play a comprehensive role in Immunotherapy.  

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Translational Studies & Lessons Learned
Moderator: Khalid Shah, Harvard Medical School
1:55
Post-transplant Immunotherapy Against Leukemia Without Graft-versus-host Disease
 
Denis-Claude Roy
Denis-Claude Roy
Professor, Director Cellular Therapy Laboratory
University of Montreal
About Speaker: Denis-Claude Roy, M.D., FRCPC, Hôpital Maisonneuve-Rosemont, Professor of Medicine, Université de Montréal, Director of Research – East of Montreal, and Scientific director, Center of Excellence for Cell Therapy, Montréal, Canada.   Deni... Read Full Bio 
 
 
Denis-Claude Roy
Professor, Director Cellular Therapy Laboratory
University of Montreal
 
About Speaker:

Denis-Claude Roy, M.D., FRCPC, Hôpital Maisonneuve-Rosemont, Professor of Medicine, Université de Montréal, Director of Research – East of Montreal, and Scientific director, Center of Excellence for Cell Therapy, Montréal, Canada.

 

Denis Claude Roy received his M.D. degree in 1982 from the University of Montreal (UofM).  After completing an Internal Medicine residency and training in Hematology at the University of Montreal, Dr. Roy did a research fellowship at Harvard University, in the Division of Tumor Immunology, Dana-Farber Cancer Institute, Boston.  In 1990, Dr. Roy joined the Division of Hematology and Bone Marrow Transplantation at the Maisonneuve-Rosemont Hospital, University of Montreal, as Director of the Cell Therapy Laboratory and Clinical Program. He is currently a Full Professor of Medicine at the UofM.  His research interests focus on the immunobiology of stem cell transplantation, and particularly at the treatment of cell grafts to foster immunotolerance and develop immune therapies against cancer. He has chaired 15 Phase I-II clinical trials at the national and international level. He has published more than 100 original articles and book chapters in prestigious journals such as Cell, Science, PLoS Medicine, Nature Medicine and Blood. Dr. Roy is Co-Director of the ThéCell FRSQ Network. He is also CEO of CellCAN Regenerative Medicine and Cell Therapy Network (Network of Centres of Excellence), and CSO of the Centre for Commercialization of Cancer Immunotherapy (C3i). He is currently Director of Research for East-of-Montreal-CIUSSS, and Scientific Director of the Center of Excellence in Cellular Therapy (CETC), Hopital Maisonneuve-Rosemont.

 

 
Abstract: Cellular immunotherapy is a particularly potent anti-cancer treatment. The ability of immune cells to fight cancer forms the basis of several appro...Read More 

Cellular immunotherapy is a particularly potent anti-cancer treatment. The ability of immune cells to fight cancer forms the basis of several approaches to treat patients with refractory hematologic malignancies. It is also recognized as a prominent mechanism whereby stem cell transplantation cures high-risk leukemia and other cancers. The administration of donor cells in the context of stem cell transplantation is however associated an important risk of developing graft-versus-host disease (GVHD). This is mediated by donor T cells recognizing host antigens as foreign.

Haplo-identical stem cell transplantation uses donors that are only partially (half) matched and therefore at high risk of GVHD. We have developed a strategy to selective photodeplete (SPD) alloreactive T cells present in the donor cell graft to eliminate GVHD-causing cells. Importantly, this SPD strategy preserves T cells with the ability to respond to infectious agents and leukemia cells. The T cell product photodepleted of anti-host reactive cells has been termed “ATIR”. A phase I dose-ranging study has confirmed the ability of ATIR to generate anti-infection activity and low relapse rates, suggesting promotion of the graft-versus-leukemia (GVL) effect. The most favorable results observed in a very high-risk patient category transplanted in this Phase I study have been confirmed in a Phase II clinical trial of 23 patients treated at a single ATIR cell dose of 2 million CD3+ cells/kg. In both studies, no patients encountered severe (Grade III-IV) GVHD although no GVHD immunoprophylaxis was given to any patient. These exciting results confirm the clinical potential of this novel immunotherapeutic strategy and prompted the development of a Phase III clinical trial.

We have also developed a novel T cell immunotherapy approach that can be used in the context of HLA-matched stem cell transplantation. This strategy enables the expansion of T cells with the ability to recognize minor histocompatibility antigens found on leukemia cells. A clinical trial has currently started to evaluate this novel treatment in patients. Laboratory and clinical results of these studies will be presented.

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2:20
Stem Cell Based Receptor Targeted Therapies for Cancer: From Bench to Bedside
 
Khalid  	 Shah
Khalid Shah
Vice Chair of Research, Director Center for Stem Cell Therapeutics and Imaging
Harvard Medical School
About Speaker: Dr. Shah is an Associate Professor at Harvard Medical School and the Director of the Center for Stem Cell Therapeutics and Imaging at BWH. He is also the Vice Chair of Research for the Department of Neurosurgery at BWH and a Principal Faculty at Harv... Read Full Bio 
 
 
Khalid Shah
Vice Chair of Research, Director Center for Stem Cell Therapeutics and Imaging
Harvard Medical School
 
About Speaker:

Dr. Shah is an Associate Professor at Harvard Medical School and the Director of the Center for Stem Cell Therapeutics and Imaging at BWH. He is also the Vice Chair of Research for the Department of Neurosurgery at BWH and a Principal Faculty at Harvard Stem Cell Institute in Boston. 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. 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. In an effort to translate the exciting therapies develped in his laboratory into clinics, he has recently founded biotech company, AMASA Technologies Inc. whose main objective is the clinical translation of therapeutic stem cells in cancer patients.

2:45
Use of T-allo10 Cell Infusion Combined with Mismatched Related or Mismatched Unrelated Donor Hematopoietic Stem Cell Transplantation (HSCT) for Hematologic Malignancies.
 
Rajni  Agarwal
Rajni Agarwal
Associate Professor and Medical Director, Division of Pediatric Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics
Stanford University
About Speaker: I trained extensively in India as a pediatric Hematologist-Oncologist. Due to a strong interest in pursuing translational research in the field I chose to come to the USA. During my stay at Children's Hospital Medical Center (CHMC) in Cincinnati, Ohi... Read Full Bio 
 
 
Rajni Agarwal
Associate Professor and Medical Director, Division of Pediatric Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics
Stanford University
 
About Speaker:

I trained extensively in India as a pediatric Hematologist-Oncologist. Due to a strong interest in pursuing translational research in the field I chose to come to the USA. During my stay at Children's Hospital Medical Center (CHMC) in Cincinnati, Ohio I developed a strong interest in the field of stem cell biology and transplantation. My experience in this field also included extensive training and expertise obtained at the hospital for sick children, Toronto, Canada and National Institute of Health (NIH), USA. At the hospital for sick children I developed stem cell assays and invivo models of human hematopoiesis that were critical in understanding of stem cell biology and its clinical applications. At the NIH I spent two years with Drs. Nienhuis and Cynthia Dunbar and developed gene transfer assays in Hematopoietic cells. At the NIH, I also worked on developing the mammalian models for in vivo gene transfer in hematopoietic cells. During this time we also published our work on chronic myeloid leukemia addressing the role of interaction of stromal cells with hematopoietic cells in the bone marrow. This publication defined conditions to favor the growth of benign hematopoietic cells in patients with chronic myeloid leukemia.
At CHMC, Cincinnati, I established the stem cell biology laboratory to further investigate the field of Hematopoietic stem cells specifically involving the umbilical cord blood. I started the clinical Umbilical cord blood transplant program at CHMC. Through my research efforts we were able to develop a sterile system for collection and use of cord blood cells. This endeavor later contributed in establishing methods to collect and store cord blood for clinical use. In the laboratory we were able to set up the assays to identify and collect highly purified hematopoietic cells from the cord blood. The engraftment and expansion potential of the cord blood derived hematopoietic cells was studied in the immune deficient mice. Also these models were then used to develop assays for gene transfer in Fanconi Anemia.

Currently, at Stanford my focus is to develop clinical research protocols to reduce toxicity from high doses of chemotherapy and radiation therapy, cellular therapies to reduce graft vs host disease, Graft manipulation to reduce complications from graft vs. host disease in patients who receive mismatched donor stem cells and develop cellular therapies to treat viral infections post transplant.
Throughout my academic career, I have maintained my interest and commitment in providing not only excellent patient care but to provide clinical research data which can be utilized to improve patient care at the end.

 
Abstract: T regulatory cells comprise different subsets of lymphocytes that are indispensable for induction and maintenance of immunological tolerance. CD4+ ...Read More 

T regulatory cells comprise different subsets of lymphocytes that are indispensable for induction and maintenance of immunological tolerance. CD4+ IL-10-producing T regulatory type 1 (Tr1) cells are identified by their unique cytokine production profile (IL-10+TGF-+IL-4-IL-17-), the co-expression of CD49b, LAG3 and CD226 and the ability to inhibit effector T cell responses in vitro and in vivo. Tr1 cells suppress T cell responses primarily via the secretion of IL-10 and TGF and the specific killing of myeloid antigen presenting cells. Highly suppressive CD49b+ LAG3+ Tr1 cells can be isolated from IL-10 polarized T cell lines or IL-10 anergized T cell cultures generated in vitro from CD4+ cells of healthy donors. Furthermore, CD49+LAG3+ Tr1 cells can be tracked in the peripheral blood of patients who developed tolerance after allogeneic hematopoietic stem cell transplant (HSCT).

Due to their suppressive properties, Treg cells have been extensively researched for their use as a cellular therapy for graft-versus-host disease, the main side effect post-HSCT, and in limiting immune responses to allograft after transplantation. The first proof-of-principle clinical trial of cell therapy with alloantigen specific IL-10 anergized T cells, containing approximately 5% of Tr1 cells, demonstrated feasibility and safety in allogeneic HSCT from haploidentical donors (Bacchetta R. et al, Frontiers Immunol 2014). We recently developed a new highly reproducible method to produce a cell product which contains a higher proportion of donor derived Tr1 cells (T- allo 10 cells) with specific suppressive activity against the host allogeneic cells.  The method for in vitro generation of T-allo10 cells  has scaled up in GMP conditions at the Stanford LCGM laboratory, which has alsoh is also performeding stability studies and analytical testing.  We will start in 1Q 2017 aA phase I/II trial using escalating doses of T-allo10 cells in pediatric and young adult patients after mismatched related and unrelated HSCT for hematologic malignancies is currently open for accrual. In addition to safety and feasibility we will assess the incidence of Graft vs Host Disease (GVHD) in the mismatched donor transplants for hematologic malignancies.

The use of Tr1 cells as cellular therapeutic to suppress GvHD and rejection after stem cell or organ transplantation represents an innovative approach to improve transplant outcomes.

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3:10
When Should you Use IPSC Derived Products
 
Mahendra  Rao
Mahendra Rao
Vice President for Regenerative Medicine
New York Stem Cell Foundation
About Speaker: Mahendra Rao  received his MD from Bombay University in India and his PhD in Developmental Neurobiology from the California Institute of Technology. Mahendra Rao is widely known for his research involving human embryonic stem cells (hESCs), iPSC, an... Read Full Bio 
 
 
Mahendra Rao
Vice President for Regenerative Medicine
New York Stem Cell Foundation
 
About Speaker:

Mahendra Rao  received his MD from Bombay University in India and his PhD in Developmental Neurobiology from the California Institute of Technology. Mahendra Rao is widely known for his research involving human embryonic stem cells (hESCs), iPSC, and other somatic stem cells. Dr. Rao   has an extensive  academic  background  with faculty positions at several  institutions including  the National Insttitutes of Health, Johns Hopkins University School of Medicine, The National Centre for Biological Sciences in Bangalore, India, and the University of Utah School of Medicine.  Dr. Rao has published more than 300 papers on stem cell research and is the co-founder of a neural stem cell company Q therapeutics  and its subsidiary Neuro Q.

Dr. Rao serves on several editorial boards, review panels scientific advisory boards and company boards. He continues to work with the ISCT, ISSCR  and FDA on consent  and other regulatory authorities. Dr Rao is currently the Vice president of Research in Regenerative Medicine at Neuro Q and provides consulting service to several companies focused on regenerative medicine. He currently  serves  on the Board of CESCA, XCell and Stempeutics  and as a consultant  for the New York Stem Cell foundation. He continues to maintain an active research program in neural development and in evaluating cell based screening and therapy to treat disorders of the nervous system.

 

 
Abstract: Regenerative Medicine is based on the assumption that cells  can  either provide trophic  support, or can provide functional replace...Read More 

Regenerative Medicine is based on the assumption that cells  can  either provide trophic  support, or can provide functional replacement or can be engineered to  and modified to construct organs and organoids.  In each of these cases one is faced with choices as to which cell is the right cell to use. I will present data on how such choices are being made and the consequences of these choices. I will use case histories and examples from the literature and our own personal experience with making these choices.

 

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3:35
Afternoon Networking Break
Round Table Discussions
Chairpersons Shown Below
4:00
Round Table 1: Pros & Cons Between Autologous & Allogeneic Approaches for Cell Therapy
 
Marcie  Glicksman
Marcie Glicksman
Chief Scientific Officer
ORIG3N
About Speaker: Marcie Glicksman, Ph.D. is the Chief Scientific Officer at ORIG3N. ORIG3N combines genetics and regenerative medicine by leading the development of allogeneic, iPSC (induced pluripotent stem cell)-derived cell therapies by leveraging the world’s la... Read Full Bio 
 
 
Marcie Glicksman
Chief Scientific Officer
ORIG3N
 
About Speaker:

Marcie Glicksman, Ph.D. is the Chief Scientific Officer at ORIG3N. ORIG3N combines genetics and regenerative medicine by leading the development of allogeneic, iPSC (induced pluripotent stem cell)-derived cell therapies by leveraging the world’s largest, uniformly consented, HLA-matched cell bank. ORIG3N also offers a broad range of affordable DNA tests that help people learn more about their genes, and the role they play in health and wellness. Dr. Glicksman received a bachelor’s degree from Brown University and a Ph.D. degree in Neuroscience from Washington University.  Dr. Glicksman has over 75 publications and 12 issued patents. Dr. Glicksman has been in the field of drug discovery for more than 20 years. She worked in the bio-pharma-ceutical industry for 13 years and then was on the faculty for ten years at Harvard Medical School and Brigham and Women’s Hospital and Co-Directed the Laboratory for Drug Discovery in Neurodegeneration (LDDN). In her career, she has led multiple advanced therapeutic programs including drug candidates that were tested in the clinic. She is on the science advisory board for the Alzheimer’s Drug Discovery Foundation (ADDF) and the California Institute for Regenerative Medicine (CIRM), and reviews grants for NIH, Department of Defense, the Michael J Fox Foundation, Alzheimer’s Association, and Rett Foundation.

4:00
Round Table 2: Ethical Issues in Gene Therapy
 
Devyn Smith
Devyn Smith
Chief Operating Officer
Sigilon
About Speaker: Devyn recently joined Sigilon, a biotechnology company, as its Chief Operating Officer tasked with building out strategy and operational elements of the company as it builds a portfolio of potential new medicines.  Prior to Sigilon, Devyn was part ... Read Full Bio 
 
 
Devyn Smith
Chief Operating Officer
Sigilon
 
About Speaker:

Devyn recently joined Sigilon, a biotechnology company, as its Chief Operating Officer tasked with building out strategy and operational elements of the company as it builds a portfolio of potential new medicines.  Prior to Sigilon, Devyn was part of Pfizer's Medicinal Sciences Division of R&D as Head of Business Operations & Strategy focused on both day to day business operations of the division, as well insuring implementable strategies are developed.  Medicinal Sciences encompasses the groups focused on discovery, formulation, and early manufacturing of the large molecule, small molecule, cell and gene therapy programs in Pfizer R&D.  Prior to this Devyn was Head of Strategy for the Pharmatherapeutics Division of Pfizer R&D focused on developing and implementing core strategies in Winning by Design in Small Molecules as well as optimizing the ROI on novel technology inventions.  Before this role, Devyn was part of Pfizer's Neusentis Research Unit in the UK as Chief Operating Officer responsible for strategy, operations and implementing key strategies.  In addition, he supported the Pharmatherapeutics Global Clinical Research Organization as the Chief Operating Officer.  He joined Pfizer's Strategic Management Group in August 2009.

Prior to joining Pfizer, Devyn was a principal for The Frankel Group (a boutique management consulting firm in New York City and Cambridge).  In his consulting experience, Devyn led a wide range of projects, across multiple therapeutic areas and a host of technology platforms, including basic R&D tools, regenerative medicine, gene therapy, and macromolecules/biologic products. Client relationships have ranged from large pharma to small biotech companies. Regenerative Medicine has been an area of strong focus with over 30 engagements, several published papers, and numerous invited conference presentations. Prior to joining The Frankel Group, Devyn worked as a management consultant at Adventis Corporation focused on clients in information intensive companies. 

Devyn received his Ph.D. in Genetics from Harvard Medical School where his research culminated in 12 publications in leading journals such as Cell, Nature, and Development.  He also holds an MS in Biology from Idaho State University and a BS in Zoology from Brigham Young University.

4:00
Round Table 3: Potency Assays for CAR/TCR Therapies
 
Joseph  Melenhorst
Joseph Melenhorst
Director of Product Development & Correlative Sciences (PDCS), Center for Cellular Immunotherapies (CCI); Adjunct Associate Professor of Pathology and Laboratory Medicine
University of Pennsylvania
About Speaker: Dr. J. Joseph Melenhorst is the Director of Product Development & Correlative Sciences (PDCS) at the Center for Cellular Immunotherapies (CCI), and Adjunct Associate Professor of Pathology and Laboratory Medicine, University of Pennsylvania (UPen... Read Full Bio 
 
 
Joseph Melenhorst
Director of Product Development & Correlative Sciences (PDCS), Center for Cellular Immunotherapies (CCI); Adjunct Associate Professor of Pathology and Laboratory Medicine
University of Pennsylvania
 
About Speaker:

Dr. J. Joseph Melenhorst is the Director of Product Development & Correlative Sciences (PDCS) at the Center for Cellular Immunotherapies (CCI), and Adjunct Associate Professor of Pathology and Laboratory Medicine, University of Pennsylvania (UPenn). After he obtained is MSc degree at the University of Nijmegen, Netherlands, and PhD degree at the University of Leiden, Netherlands, he did his post-doctoral research at the National Institutes of Health in Bethesda, Maryland, studying the biology of bone marrow failure syndromes, leukemia and graft-versus host immunology, and adoptive cell therapy.

 

Dr. Melenhorst was recruited to the University of Pennsylvania in 2012. The focus of his laboratory is to understand and improve the efficacy and safety of cellular immunotherapy through biomarker, mechanistic, and product development studies. His laboratory has uncovered key biomarkers of a chimeric antigen receptor (CAR) T cell therapy-associated toxicity, cytokine release syndrome (CRS), and developed predictive monitoring tools for CRS in leukemia 1. He furthermore identified the critical parameters determining success in CAR T cell therapies in leukemia 2 (and manuscripts under revision).

 

Lastly, his efforts include the development of a universal, streamlined, cost-effective and potent manufacturing process for T cell engineering and some of his insight has already been translated into clinical practice.

5:15
Networking Reception & Poster Session
Day - 2 Tuesday, September 12th, 2017
7:15
Breakfast with Mentors from Academia & Industry
Industry Partnering with Academia & CROs to Advance Research
Moderator: Christopher-Paul Milne, Tufts Center for the Study of Drug Development
8:30
Bench to Bedsides: Achieving Commercially Viable Scale out for Cellular Therapies
 
Ian Gaudet
Ian Gaudet
Director of Process Development
Miltenyi Biotec
About Speaker: Dr. Ian Gaudet studied Bioengineering at the University of California, San Diego, and received his doctorate in Biomedical Engineering jointly from Rutgers University and the University of Medicine and Dentistry of New Jersey, with work focused o... Read Full Bio 
 
 
Ian Gaudet
Director of Process Development
Miltenyi Biotec
 
About Speaker:

Dr. Ian Gaudet studied Bioengineering at the University of California, San Diego, and received his doctorate in Biomedical Engineering jointly from Rutgers University and the University of Medicine and Dentistry of New Jersey, with work focused on novel biomaterials for regenerative medicine applications. Following his Ph.D., Dr. Gaudet worked for Progenitor Cell Therapy (PCT) where he was the Sr. Engineer and then Director of Innovation and Engineering, leading a scientific and engineering team in support of design, development, and implementation of clinical cell therapy manufacturing processes, as well as cell therapy commercialization strategy consulting. Dr. Gaudet led multiple projects from tech transfer, through development, and into clinical manufacturing, including multiple gene-modified hematopoietic stem cell and T cell therapies. He joined Miltenyi Biotec in 2016 and is currently the Director of Process Development at Miltenyi’s contract development and manufacturing facility in Sunnyvale, CA, where he focuses on development and translation of novel cell therapy processes into clinical manufacturing and beyond.

 
Abstract: ...Read More 

The clinical promise and initial success of cellular therapies for addressing devastating illnesses and fulfilling unmet medical needs is clear. However, it’s also clear that despite clinical efficacy, cell-based therapy may not be adopted widely if reimbursement concerns continue to make insurers (and therefore physicians) hesitant to support this 4th pillar of healthcare. A major factor in this issue is the high cost of goods currently required to manufacture these complex, living medicines. Production requires a large up-front investment, and therefore financial risk, from both the manufacturer and the payer. Not surprisingly, the best way to mitigate this risk is to reduce the cost of goods required per dose, which will enable more flexibility in the reimbursement strategy. Currently, the cost of goods for production of cellular therapies is dominated by labor due to the complex nature and frequently academic genesis of these types of processes. Automation of the entire production process, including the physical needle-to-needle cell journey as well as the flow of information, will be required to reduce the onerous costs required to create each dose of cell therapy with the paradigm seen in current clinical trials. To this end, Miltenyi Biotec is leading the field in supplying automated tools for cell therapy manufacturing and analysis, as well as developing systems for integrating these tools to enable throughput of product characterization and release much more efficiently, and thus more cost-efficient as well.

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8:55
Industry-Academia Partnering in the Adoptive T-Cell Therapy Business: The UPenn-Novartis Approach
 
Joseph  Melenhorst
Joseph Melenhorst
Director of Product Development & Correlative Sciences (PDCS), Center for Cellular Immunotherapies (CCI); Adjunct Associate Professor of Pathology and Laboratory Medicine
University of Pennsylvania
About Speaker: Dr. J. Joseph Melenhorst is the Director of Product Development & Correlative Sciences (PDCS) at the Center for Cellular Immunotherapies (CCI), and Adjunct Associate Professor of Pathology and Laboratory Medicine, University of Pennsylvania (UPen... Read Full Bio 
 
 
Joseph Melenhorst
Director of Product Development & Correlative Sciences (PDCS), Center for Cellular Immunotherapies (CCI); Adjunct Associate Professor of Pathology and Laboratory Medicine
University of Pennsylvania
 
About Speaker:

Dr. J. Joseph Melenhorst is the Director of Product Development & Correlative Sciences (PDCS) at the Center for Cellular Immunotherapies (CCI), and Adjunct Associate Professor of Pathology and Laboratory Medicine, University of Pennsylvania (UPenn). After he obtained is MSc degree at the University of Nijmegen, Netherlands, and PhD degree at the University of Leiden, Netherlands, he did his post-doctoral research at the National Institutes of Health in Bethesda, Maryland, studying the biology of bone marrow failure syndromes, leukemia and graft-versus host immunology, and adoptive cell therapy.

 

Dr. Melenhorst was recruited to the University of Pennsylvania in 2012. The focus of his laboratory is to understand and improve the efficacy and safety of cellular immunotherapy through biomarker, mechanistic, and product development studies. His laboratory has uncovered key biomarkers of a chimeric antigen receptor (CAR) T cell therapy-associated toxicity, cytokine release syndrome (CRS), and developed predictive monitoring tools for CRS in leukemia 1. He furthermore identified the critical parameters determining success in CAR T cell therapies in leukemia 2 (and manuscripts under revision).

 

Lastly, his efforts include the development of a universal, streamlined, cost-effective and potent manufacturing process for T cell engineering and some of his insight has already been translated into clinical practice.

 
Abstract: Ever since the inception of the alliance between the University of Pennsylvania (UPenn) and Novartis to expand use of personalized chimeric antigen...Read More 

Ever since the inception of the alliance between the University of Pennsylvania (UPenn) and Novartis to expand use of personalized chimeric antigen receptor (CAR)-based T cell therapy for cancer patients, joined activities have accelerated the global roll-out of our personalized therapy and the development of new targeting receptors for liquid and solid tumors. In my talk I will highlight the successful cell manufacturing technology transfer from UPenn to the Novartis Morris Plains manufacturing facility and provide examples of novel CAR development to advance cancer therapies.

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Panel: Industry Partnering with Academia & CROs to Advance Research
Moderator: Denis Rodgerson, GTCbio
9:20
Joseph  Melenhorst
Joseph Melenhorst
Director of Product Development & Correlative Sciences (PDCS), Center for Cellular Immunotherapies (CCI); Adjunct Associate Professor of Pathology and Laboratory Medicine
University of Pennsylvania
About Speaker: Dr. J. Joseph Melenhorst is the Director of Product Development & Correlative Sciences (PDCS) at the Center for Cellular Immunotherapies (CCI), and Adjunct Associate Professor of Pathology and Laboratory Medicine, University of Pennsylvania (UPen... Read Full Bio 
 
 
Joseph Melenhorst
Director of Product Development & Correlative Sciences (PDCS), Center for Cellular Immunotherapies (CCI); Adjunct Associate Professor of Pathology and Laboratory Medicine
University of Pennsylvania
 
About Speaker:

Dr. J. Joseph Melenhorst is the Director of Product Development & Correlative Sciences (PDCS) at the Center for Cellular Immunotherapies (CCI), and Adjunct Associate Professor of Pathology and Laboratory Medicine, University of Pennsylvania (UPenn). After he obtained is MSc degree at the University of Nijmegen, Netherlands, and PhD degree at the University of Leiden, Netherlands, he did his post-doctoral research at the National Institutes of Health in Bethesda, Maryland, studying the biology of bone marrow failure syndromes, leukemia and graft-versus host immunology, and adoptive cell therapy.

 

Dr. Melenhorst was recruited to the University of Pennsylvania in 2012. The focus of his laboratory is to understand and improve the efficacy and safety of cellular immunotherapy through biomarker, mechanistic, and product development studies. His laboratory has uncovered key biomarkers of a chimeric antigen receptor (CAR) T cell therapy-associated toxicity, cytokine release syndrome (CRS), and developed predictive monitoring tools for CRS in leukemia 1. He furthermore identified the critical parameters determining success in CAR T cell therapies in leukemia 2 (and manuscripts under revision).

 

Lastly, his efforts include the development of a universal, streamlined, cost-effective and potent manufacturing process for T cell engineering and some of his insight has already been translated into clinical practice.

9:20
Ian Gaudet
Ian Gaudet
Director of Process Development
Miltenyi Biotec
About Speaker: Dr. Ian Gaudet studied Bioengineering at the University of California, San Diego, and received his doctorate in Biomedical Engineering jointly from Rutgers University and the University of Medicine and Dentistry of New Jersey, with work focused o... Read Full Bio 
 
 
Ian Gaudet
Director of Process Development
Miltenyi Biotec
 
About Speaker:

Dr. Ian Gaudet studied Bioengineering at the University of California, San Diego, and received his doctorate in Biomedical Engineering jointly from Rutgers University and the University of Medicine and Dentistry of New Jersey, with work focused on novel biomaterials for regenerative medicine applications. Following his Ph.D., Dr. Gaudet worked for Progenitor Cell Therapy (PCT) where he was the Sr. Engineer and then Director of Innovation and Engineering, leading a scientific and engineering team in support of design, development, and implementation of clinical cell therapy manufacturing processes, as well as cell therapy commercialization strategy consulting. Dr. Gaudet led multiple projects from tech transfer, through development, and into clinical manufacturing, including multiple gene-modified hematopoietic stem cell and T cell therapies. He joined Miltenyi Biotec in 2016 and is currently the Director of Process Development at Miltenyi’s contract development and manufacturing facility in Sunnyvale, CA, where he focuses on development and translation of novel cell therapy processes into clinical manufacturing and beyond.

9:20
Marcie  Glicksman
Marcie Glicksman
Chief Scientific Officer
ORIG3N
About Speaker: Marcie Glicksman, Ph.D. is the Chief Scientific Officer at ORIG3N. ORIG3N combines genetics and regenerative medicine by leading the development of allogeneic, iPSC (induced pluripotent stem cell)-derived cell therapies by leveraging the world’s la... Read Full Bio 
 
 
Marcie Glicksman
Chief Scientific Officer
ORIG3N
 
About Speaker:

Marcie Glicksman, Ph.D. is the Chief Scientific Officer at ORIG3N. ORIG3N combines genetics and regenerative medicine by leading the development of allogeneic, iPSC (induced pluripotent stem cell)-derived cell therapies by leveraging the world’s largest, uniformly consented, HLA-matched cell bank. ORIG3N also offers a broad range of affordable DNA tests that help people learn more about their genes, and the role they play in health and wellness. Dr. Glicksman received a bachelor’s degree from Brown University and a Ph.D. degree in Neuroscience from Washington University.  Dr. Glicksman has over 75 publications and 12 issued patents. Dr. Glicksman has been in the field of drug discovery for more than 20 years. She worked in the bio-pharma-ceutical industry for 13 years and then was on the faculty for ten years at Harvard Medical School and Brigham and Women’s Hospital and Co-Directed the Laboratory for Drug Discovery in Neurodegeneration (LDDN). In her career, she has led multiple advanced therapeutic programs including drug candidates that were tested in the clinic. She is on the science advisory board for the Alzheimer’s Drug Discovery Foundation (ADDF) and the California Institute for Regenerative Medicine (CIRM), and reviews grants for NIH, Department of Defense, the Michael J Fox Foundation, Alzheimer’s Association, and Rett Foundation.

10:10
Morning Networking Break
Regulatory Challenges
Moderator: Cliff Mintz, Bioinsights
10:40
Are You BREXIT Ready?
 
Peadar Mac Gabhann
Peadar Mac Gabhann
Chief Executive Officer
Biostór Ireland
About Speaker: Peadar Mac Gabhann is a graduate of the National University of Ireland, University College Dublin, M.Sc. in Industrial Microbiology. He carried out post graduate research at the Netherlands Cancer Institute (NKI) Amsterdam, Biogen at ETH Zurich and t... Read Full Bio 
 
 
Peadar Mac Gabhann
Chief Executive Officer
Biostór Ireland
 
About Speaker:

Peadar Mac Gabhann is a graduate of the National University of Ireland, University College Dublin, M.Sc. in Industrial Microbiology. He carried out post graduate research at the Netherlands Cancer Institute (NKI) Amsterdam, Biogen at ETH Zurich and the Faculty of Medicine at Kyoto University Japan. He joined Schering Plough Corporation (Merck) and was a key member of staff in the start-up of the first world-wide Biopharma facility in Cork for the commercial production of Interferon in 1983. As Director and board member of Schering Plough Japan, he led the company’s research and Asian business development operations. Peadar has more than 30 years’ experience in the international pharmaceutical industry. In the past 10 years he co-founded and directed two Life Science start-ups and participated in several international EU projects: He has presented a position paper on “Biobanks - Key Resources for Advancement of Biotechnology & Human Health” to the expert group of the EU Parliament on the Future of Medicine. He is national expert representing Ireland on the working group to establish a new ISO TC276 standard in Biobanking & Bioprocessing. He currently consults with Jacobs Engineering on the design and construction of major Biopharma facilities for Bristol Myers Squibb, Novo Nordisk & Shire.He lectures part-time in Pharmaceutical Business at Griffith College Dublin as well as running a certified Tissue Establishment in Wexford, Biostór Ireland.

 

 
Abstract: ...Read More 

The talk will focus on the latest BREXIT developments that impact the Biopharma and Cell Therapy Industry. It will provide up-to-date details under the following: Regulatory, Trade Restrictions, Import and Export, Clinical Trials, Research. (As BREXIT is an evolving process and negotiations between the EU and the UK are only just getting underway this month it is impossible at this stage to provide an accurate brief)

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Panel: Funding & Regulatory Challenges
Moderator: Peadar Mac Gabhann, Biostór Ireland
11:05
Christopher-Paul  Milne
Christopher-Paul Milne
Director of Research
Tufts Center for the Study of Drug Development
About Speaker: Dr. Christopher Milne joined the Center for the Study of Drug Development at Tufts University (Tufts CSDD) in 1998 as a Senior Research Fellow and has published over 100 book chapters, CSDD reports, white papers, and journal articles. His researc... Read Full Bio 
 
 
Christopher-Paul Milne
Director of Research
Tufts Center for the Study of Drug Development
 
About Speaker:

Dr. Christopher Milne joined the Center for the Study of Drug Development at Tufts University (Tufts CSDD) in 1998 as a Senior Research Fellow and has published over 100 book chapters, CSDD reports, white papers, and journal articles. His research interests include: academic-industry collaborations; global disease, demographic and market access factors; and, FDA incentive programs. He is currently Director of Research at Tufts CSDD, Research Associate Professor at Tufts University School of Medicine, Innogen Center Associate (University of Edinburgh), and editorial board member of Therapeutic Innovation & Regulatory Science and Pharma Focus Asia. Dr. Milne received a BA from Fordham University, an MPH from The Johns Hopkins University, and holds doctoral degrees in veterinary medicine and law, respectively from the UNPHU and UNH School of Law.

11:05
Cliff  Mintz
Cliff Mintz
Chairman & Chief Executive Officer
Bioinsights
About Speaker: Clifford S. Mintz, PhD has an extensive background in biopharmaceutical drug development, strategic planning and commercialization of biosimilar drugs. He is the founder of BioInsights a biopharmaceutical consulting and education/training f... Read Full Bio 
 
 
Cliff Mintz
Chairman & Chief Executive Officer
Bioinsights
 
About Speaker:

Clifford S. Mintz, PhD has an extensive background in biopharmaceutical drug development, strategic planning and commercialization of biosimilar drugs.

He is the founder of BioInsights a biopharmaceutical consulting and education/training firm

Cliff received a B.S. in microbiology/animal science from Cornell University, a Ph.D. in Bacteriology from the University of Wisconsin-Madison and performed his post-doctoral studies at Oregon Health Sciences University and the College of Physicians & Surgeons, Columbia University

11:05
Greg  Dombal
Greg Dombal
Chief Operating Officer
Halloran Consulting Group
About Speaker: Greg joined Halloran Consulting Group in 2007.  He focuses on helping emerging companies navigate challenges in product development while establishing appropriate clinical, regulatory, and compliance structures. Greg represents clients to worldwi... Read Full Bio 
 
 
Greg Dombal
Chief Operating Officer
Halloran Consulting Group
 
About Speaker:

Greg joined Halloran Consulting Group in 2007.  He focuses on helping emerging companies navigate challenges in product development while establishing appropriate clinical, regulatory, and compliance structures. Greg represents clients to worldwide regulatory agencies and routinely functions as an on-demand Head of Regulatory, Clinical, or Development.

Greg pairs over 25 years’ experience in worldwide regulatory affairs, quality assurance and clinical development with a willingness to challenge conventional, conservative approaches and find the best solution for each company and their products.  Before joining Halloran, Greg was responsible for the regulatory, quality assurance, and clinical groups at ArQule. At its peak, the programs he was responsible for encompassed 17 clinical studies with annual budgets in excess of $25 million.

Over his career, Greg has successfully filed over 45 clinical trial applications (IND, IDE or equivalent), attended over 75 regulatory agency meetings, presented to multiple Advisory Committees and helped companies respond to Warning Letters, 522 Orders, and Clinical Hold notices.  He has obtained Orphan Drug designation and/or Fast Track designation for fifteen individual products, successfully negotiated multiple Special Protocol Agreements, and has extensive involvement with NDA/MAA submissions resulting in multiple product approvals.

11:05
Andrew  Collard
Andrew Collard
Engagement Manager
Back Bay Life Science Advisors
About Speaker: Andy Collard is an Engagement Manager at Back Bay Life Science Advisors, a healthcare-focused strategy consulting firm based in Boston. At Back Bay, Andy has led several of the firm’s projects working with market leading gene therapy and gene editi... Read Full Bio 
 
 
Andrew Collard
Engagement Manager
Back Bay Life Science Advisors
 
About Speaker:

Andy Collard is an Engagement Manager at Back Bay Life Science Advisors, a healthcare-focused strategy consulting firm based in Boston. At Back Bay, Andy has led several of the firm’s projects working with market leading gene therapy and gene editing companies, advising on indication prioritization, enabling technologies, and articulating companies’ value proposition for potential investors. In addition to these projects, Back Bay has worked on several buy-side diligence projects evaluating orphan disease companies and programs, including gene therapy assets. Andy received his MBA with a focus in healthcare from Yale School of Management. Prior to Back Bay, he worked at Myriant Technologies, engineering new bacteria and yeast strains to produce renewable bio-based chemicals.

12:05
Lunch Provided by GTCbio
Keynote Panel Discussion
Moderator: Denis Rodgerson, GTCbio
2:20
Richard Gatti
Richard Gatti
Distinguished Professor in the Departments of Pathology & Laboratory Medicine and Human Genetics
David Geffen UCLA School of Medicine
About Speaker: Richard Gatti is a molecular biologist and geneticist who joined the UCLA School of Medicine faculty in 1974 as full professor. After a pediatric residency at Children???s Memorial Hospital in Chicago (1962-65), and serving as Captain, U.S. Army in E... Read Full Bio 
 
 
Richard Gatti
Distinguished Professor in the Departments of Pathology & Laboratory Medicine and Human Genetics
David Geffen UCLA School of Medicine
 
About Speaker:

Richard Gatti is a molecular biologist and geneticist who joined the UCLA School of Medicine faculty in 1974 as full professor. After a pediatric residency at Children???s Memorial Hospital in Chicago (1962-65), and serving as Captain, U.S. Army in Eritrea, Ethiopia (1966-68), he did post-doctoral fellowships with Robert A. Good, MD, PhD, at the University of Minnesota (1968-72) in basic and clinical immunology, and George Klein, MD, at the Karolinska Institute in Stockholm, Sweden (1972-75) in tumor biology. He served as Director of Pediatric Hematology, Oncology and Immunology at Cedars-Sinai Medical Center (1974-1980). He joined the Department of Pathology & Laboratory Medicine at UCLA in 1981. He founded the Molecular Diagnostics Laboratory (1987) and is co-director. He is also a member of the Department of Human Genetics (2005- _. He is Scientific Director of the Ataxia-Telangiectasia Medical Research Foundation (1984- ) as well as the Neuromuscular Diseases Foundation (2006- ). He was a recipient of a USPHS Research Career Development Award (1972-75) and has been funded by NIH or the Department of Energy since 1967. He has co-authored over 330 scientific articles. He pioneered the field of hematopoietic stem cell transplantation and other treatments for primary immunodeficiencies. His current research focuses on the molecular genetics, diagnostics, radiobiology, and treatment of children with ataxia-telangiectasia, a progressive neurological disease of children.

2:20
Philip  Gregory
Philip Gregory
Chief Scientific Officer
Bluebird Bio
About Speaker: Philip D. Gregory, D. Phil., has served as Chief Scientific Officer of bluebird bio since June 2015. Prior to joining bluebird Philip was at Sangamo BioSciences, Inc., where he held multiple leadership positions, most recently serving as Chief S... Read Full Bio 
 
 
Philip Gregory
Chief Scientific Officer
Bluebird Bio
 
About Speaker:

Philip D. Gregory, D. Phil., has served as Chief Scientific Officer of bluebird bio since June 2015. Prior to joining bluebird Philip was at Sangamo BioSciences, Inc., where he held multiple leadership positions, most recently serving as Chief Scientific Officer and Senior Vice President, Research. Philip has led pioneering research in gene therapy and genome editing and was named by Thompson Reuters as one of “The World’s Most Influential Scientific Minds 2014” and “Most Cited Researchers 2015”. He served as a member of the Scientific Advisory Board of the Keystone Symposia from 2009 to 2015, and the ASGCT Advisory Council from 2016. Before joining the biotechnology industry Philip was at the University of Munich, Germany, where he studied the role of chromatin structure in gene regulation. Philip earned a D. Phil. in biochemistry from the University of Oxford (Keble College) and holds a B.Sc. in microbiology from the University of Sheffield.

3:05
Conference Concludes