Regenerative Medicine: Transitioning Therapeutics from Cells to the Clinic

Regenerative Medicine: Transitioning Therapeutics from Cells to the Clinic

Monday, February 22, 2016

The New York Academy of Sciences

The field of regenerative medicine is burgeoning with academic and industry groups, actively developing cell-based therapeutic products. These therapies aim to enhance or correct cell function, often utilizing genetic modification or gene editing approaches. Preclinical and early stage clinical trials have demonstrated that cell-based therapies can provide local and systemic clinical benefit by driving production and secretion of biologically active mediators such as cytokines and growth factors, reversing disease progression and promoting healing. Notably, cell-based therapies are already commercially available for cartilage injury and severe burn injuries, and clinical trials have shown promising results in diabetes, kidney disease, heart disease, stroke, cancer, spinal cord injury, and graft versus host disease (GVHD).

To advance the development of cell-based therapies, next generation approaches for scaled manufacturing and formulation of cell-based therapeutic products are being implemented, enabling distribution demands in compliance with regulatory guidelines. Moreover, as numerous prospective therapeutics are targeted to clinical indications where there are no approved therapies, adaptive clinical trial strategies along with novel outcome endpoints for registration must be contemplated. These manufacturing and clinical endeavors require focused interactions between academia and industry to discover and translate key innovations in these areas. The symposium will unite key representatives from both academia and industry to discuss the challenges and important new developments critical for the advancement of the regenerative medicine field.

*Reception to follow.

Call for Poster Abstracts

Abstract submissions are invited for a poster session. For complete submission instructions, please send an email to RegMed@nyas.org with the words "Abstract Information" in the subject line. The deadline for abstract submission is February 5, 2016.

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Member (Student / Postdoc / Resident / Fellow)$25
Nonmember (Academia)$105
Nonmember (Corporate)$160
Nonmember (Non-profit)$105
Nonmember (Student / Postdoc / Resident / Fellow)$70


This event will also be broadcast as a webinar; registration is required.

Please note: Transmission of presentations via the webinar is subject to individual consent by the speakers. Therefore, we cannot guarantee that every speaker's presentation will be broadcast in full via the webinar. To access all speakers' presentations in full, we invite you to attend the live event in New York City where possible.

Webinar Pricing

Member$30
Member (Student / Postdoc / Resident / Fellow)$15
Nonmember (Academia)$65
Nonmember (Corporate)$85
Nonmember (Non-profit)$65
Nonmember (Student / Postdoc / Resident / Fellow)$45

Agenda

* Presentation titles and times are subject to change.


Monday, February 22, 2016

9:00 AM

Opening Remarks
Sonya Dougal, PhD, New York Academy of Sciences
Jane S. Lebkowski, PhD, Asterias Biotherapeutics

9:15 AM

iPSC-based Cell Therapy: Issues and Solutions
Mahendra Rao, MD, PhD, Wake Forest University School of Medicine

9:55 AM

Engineering Genetic Cures: Genome Editing of Stem Cells
Edward O. Lanphier, Sangamo BioSciences Inc

10:35 AM

Networking Coffee Break

10:55 AM

Stem Cell Therapies for Neurodegenerative Diseases
Jonathan D. Glass, MD, Emory University School of Medicine

11:35 AM

Long-term Relapse-free Survival of Patients with Acute Myeloid Leukemia (AML) Receiving a Telomerase-engineered Dendritic Cell Immunotherapy
John F. DiPersio, MD, PhD, Washington University School of Medicine

12:15 PM

Networking Lunch and Poster Session

1:15 PM

Clinical Assessment of hESC-derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Sensorimotor-Complete Thoracic and Cervical Spinal Cord Injuries
Jane S. Lebkowski, PhD, Asterias Biotherapeutics

1:55 PM

Mechanism of Action of PLX - Placental-Derived Cellular Therapy
Racheli Ofir, PhD, Pluristem LTD

2:35 PM

Development of Stem-Cell Derived, Macroencapsulated Islet Replacement for Type 1 Diabetes
Thomas Schulz, PhD, ViaCyte, Inc.

3:15 PM

Networking Coffee Break

3:35 PM

Neural Stem Cell Treatment for Stroke Disability
John D. Sinden, PhD, ReNeuron Limited

4:15 PM

Overview of the Challenges Faced and Opportunities Available to Expedite Development for Advanced Therapies
Kathleen Tsokas, JD, Janssen Research & Development

4:55 PM

Closing Remarks and Poster Award Presentation
George Zavoico, PhD, JonesTrading Institutional Services LLC

5:00 PM

Networking Reception

6:00 PM

Close

Organizers

Jane S. Lebkowski, PhD

Asterias Biotherapeutics

Jane Lebkowski has been actively involved in the development of cell and gene therapies since 1986 and is currently Chief Scientific Officer and President of R&D at Asterias Biotherapeutics Inc, where she is responsible for all preclinical product development of Asterias’ products. From 1998 to 2012, Dr. Lebkowski was Senior Vice President of Regenerative Medicine and Chief Scientific Officer at Geron Corporation. Dr. Lebkowski led Geron’s human embryonic stem cell program, being responsible for all research, preclinical development, product development, manufacturing, and clinical development activities. Prior to Geron, Dr. Lebkowski was Vice President of Research and Development at Applied Immune Sciences.  Following the acquisition of Applied Immune Sciences by Rhone Poulenc Rorer (RPR, currently Sanofi), Dr. Lebkowski remained at RPR as Vice President of Discovery Research. Dr. Lebkowski received her Ph.D. in Biochemistry from Princeton University in 1982, and completed a postdoctoral fellowship at the Department of Genetics, Stanford University in 1986. Dr. Lebkowski has published over 70 peer reviewed papers and has 13 issued U.S. patents. Dr. Lebkowski has served as the co-chair of the Industrial Committee of the International Society for Stem Cell Research and serves on several scientific advisory boards and other professional committees.

George Zavoico, PhD

JonesTrading Institutional Services LLC

George Zavoico is a Senior Equity Analyst, Healthcare, at JonesTrading Institutional Services, a leading equity trading firm with a focus on block trading and a growing capital markets business.  He has over 11 years of experience as a life sciences analyst writing research on publicly traded equities. His principal focus is on biotechnology, biopharmaceutical, specialty pharmaceutical, and molecular diagnostics companies. He received The Financial Times/Starmine Award two years in a row for being among the top-ranked earnings estimators in the biotechnology sector. In 2009, Zavoico was hired as the first equity analyst at MLV & Co., a New York-based boutique investment bank and institutional broker-dealer at the time, where he helped establish its Healthcare research team. He returned to MLV in mid-2014 after serving for a brief period as a Senior Equity Analyst at H.C. Wainwright & Co. in early 2014, and then joined JonesTrading in early 2015. Previously, Zavoico was an equity research analyst in the healthcare sector at Westport Capital Markets and Cantor Fitzgerald. Prior to working as an analyst, Zavoico established his own consulting company serving the biotech and pharmaceutical industries, providing competitive intelligence and marketing research, due diligence services and guidance in regulatory affairs. Zavoico began his career as a senior research scientist at Bristol-Myers Squibb Co., moving on to management positions at Alexion Pharmaceuticals Inc. and T Cell Sciences Inc. (now Celldex Therapeutics Inc.). Zavoico has a bachelor's degree in biology from St. Lawrence University and a PhD in physiology from the University of Virginia. He held post-doctoral fellowships at the University of Connecticut School of Medicine and Harvard Medical School/Brigham & Women's Hospital. He has published more than 30 papers in peer-reviewed journals and has coauthored four book chapters.

Sonya Dougal, PhD

The New York Academy of Sciences

Caitlin McOmish, PhD

The New York Academy of Sciences

Speakers

John F. DiPersio, MD, PhD

Washington University School of Medicine

Dr. John F. DiPersio, Deputy Director, Alvin J. Siteman Cancer Center and Chief of the Division of Oncology at Washington University School of Medicine in St. Louis and the Virginia E. and Samuel J. Golman Professor of Medicine. Dr. DiPersio’s research focuses on fundamental and translational aspects of leukemia and stem cell biology. These studies include identification of genetic abnormalities in human leukemias, understanding processes involving stem cell and leukemia cell trafficking, and clinical and translational programs in both leukemia/myelodysplastic syndrome and stem cell transplantation. Dr. DiPersio is Chair of ASH Scientific Committee on Hematopoiesis, a member of the Board of Scientific Counselors (Clinical Science and Epidemiology) of the National Cancer Institute, and the 2013 recipient of the Daniel P. Schuster Distinguished Translational Investigator Award from Washington University, the 19th Annual AACR Joseph H. Burchenal Memorial Award for Outstanding Achievement in Clinical Cancer Research in 2014 and the 2014 recipient of the American Society of Hematology Mentor Award for Clinical Investigations..  He has authored or co-authored more than 300 publications and over 60 invited reviews and book chapters. Dr. DiPersio received his M.D. and Ph.D. from the University of Rochester and his B.A. in Biology from Williams College.  He completed an internship and residency at Parkland Memorial Hospital and The University of Texas Southwestern Medical Center in Dallas. After serving as chief resident at Parkland Memorial Hospital, Dr. DiPersio completed a fellowship in the Division of Hematology/Oncology at the University of California, Los Angeles (UCLA).

Jonathan D. Glass, MD

Emory University School of Medicine

Jonathan D. Glass is Professor of Neurology and Pathology, and the Director of the Emory ALS Center in the Department of Neurology at Emory University in Atlanta, Georgia. He is widely known for his research on the pathogenesis and prevention of nerve degeneration in neurological diseases, and for his work in human and experimental neuropathology. His laboratory currently focuses on the causes of neurodegeneration in cellular and animal models of motor neuron disease, including the development of novel therapeutic interventions to prevent the death of neurons, nerve fibers, and the neuromuscular junction. Dr. Glass is the Principle Investigator for the ongoing clinical trial of intraspinal stem cell transplantation for ALS (Neuralstem). Dr. Glass is an active clinician who has been cited each year since 2001 as one of "America's Top Doctors" (Castle Connelly) and since 2005 as one of the very few neurologists in "Atlanta's Top Doctors" (Atlanta Magazine). He is also a teacher and mentor to young physicians and served as the director of Emory's Neurology residency program from 2001-2006. Dr. Glass received his undergraduate degree from Middlebury College (Vermont) and his MD from the University of Vermont.  He trained in Neurology and Neuropathology at Johns Hopkins, where he was a faculty member until moving to Emory in 1996.

Edward O. Lanphier

Sangamo BioSciences, Inc.

Edward Lanphier is the founder of Sangamo BioSciences, and has served as president, chief executive officer and a member of the Board of Directors since the company’s inception in 1995.  Sangamo BioSciences, Inc. is a leader in therapeutic genome editing and gene regulation. Edward has over 35 years of experience in the pharmaceuticals and biotechnology industry including 25 years in gene therapy and modified-cell therapies. He is currently the Chairman of the Alliance for Regenerative Medicine, the principle industry advocacy organization in the gene and modified-cell therapy space.  He is also a member of the Industry Advisory Board for the California Institute for Regenerative Medicine, the Dean’s Advisory Board of the University of Michigan School Of Public Health, a director of The Biotechnology Institute, and the Board of Trustees of The Buck Institute for Research on Aging.

Jane S. Lebkowski, PhD

Asterias Biotherapeutics

Jane Lebkowski has been actively involved in the development of cell and gene therapies since 1986 and is currently Chief Scientific Officer and President of R&D at Asterias Biotherapeutics Inc, where she is responsible for all preclinical product development of Asterias’ products. From 1998 to 2012, Dr. Lebkowski was Senior Vice President of Regenerative Medicine and Chief Scientific Officer at Geron Corporation. Dr. Lebkowski led Geron’s human embryonic stem cell program, being responsible for all research, preclinical development, product development, manufacturing, and clinical development activities. Prior to Geron, Dr. Lebkowski was Vice President of Research and Development at Applied Immune Sciences.  Following the acquisition of Applied Immune Sciences by Rhone Poulenc Rorer (RPR, currently Sanofi), Dr. Lebkowski remained at RPR as Vice President of Discovery Research.  Dr. Lebkowski received her Ph.D. in Biochemistry from Princeton University in 1982, and completed a postdoctoral fellowship at the Department of Genetics, Stanford University in 1986. Dr. Lebkowski has published over 70 peer reviewed papers and has 13 issued U.S. patents. Dr. Lebkowski has served as the co-chair of the Industrial Committee of the International Society for Stem Cell Research and serves on several scientific advisory boards and other professional committees.

Racheli Ofir, PhD

Pluristem LTD

Dr. Ofir is the VP of Research and Intellectual Property of Pluristem Ltd. Racheli Ofir holds a PhD from the Technion, Israel Institute of Technology, and a Masters in Law from Haifa University. Dr. Ofir has a broad professional experience in the field of human cell therapy as leading the research team in elucidating the therapeutic effect as well as the Mechanism of Action, Immunogenecity and in-vivo safety of Placenta derived cellular therapy. Dr. Ofir is the co-author of numerous peer reviewed articles and a co-inventor on numerous patents and patent applications in the field of cellular therapy.

Mahendra Rao, MD, PhD

Wake Forest University School of Medicine

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 wisely known for his research involving human embryonic stem cells (hESCs), iPSC, and other somatic stem cells. Dr. Rao and has worked in the stem cell field for more than twenty years with stints in academia, government and regulatory affairs and industry. Dr Rao is currently on the faculty at Wake Forest University and is the Vice president of Research in Regenerative Medicine at Q therapeutics. He currently serves on the Board of CESCA, XCell and Q therapeutics and on the SAB’s and as a consultant of various stem cell companies including 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.

Thomas Schulz, PhD

ViaCyte, Inc.

Tom Schulz is the Director of ES Cell Technology & Scale Up of ViaCyte. He completed a PhD in Biochemistry and Molecular Biology from the University of Adelaide. From1997-2003 he held post-doctoral appointments at Tottori University, the NIH and the University of Georgia, prior to joining ViaCyte. Dr Schulz is the author and inventor of multiple articles and patents related to human embryonic stem cells and cellular therapy. He is a member of the International Society for Stem Cell Research.

John D. Sinden, PhD

ReNeuron Limited

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

Katherine Tsokas, JD

Janssen Research & Development

Kathy Tsokas, JD, is responsible leading the Advanced Therapy Global Regulatory Affairs influencing activities across Janssen Research & Development, and is the Global Regulatory Leader for Janssen’s lead cell therapy project. Kathy has 25 years of global regulatory experience in both small and large sized Pharma companies.  She has worked on products in various therapeutic areas and at all stages of development, from early through to filing, approval and commercialization. Kathy has been with J&J for 8 years.  She has held several roles during this time including Global Regulatory Lead for the Research and Early Development (RED) units.  Currently, her responsibilities include providing strategic regulatory oversight to advanced therapy projects in several therapeutic areas by ensuring regulatory strategies contribute to and support the development plans for the products and that all opportunities for collaboration internally and externally are utilized.  Importantly, Kathy leads efforts internally and externally to enhance awareness and connectivity to influence the development of processes and regulatory pathways that enable the global development and commercialization of safe and effective advanced therapeutics. Kathy participates in discussions with global Health Authorities through internal project work and external collaborations. In addition, she represents Global Regulatory Affairs on the J&J First in Human Committee. Kathy is the Chair for the Alliance for Regenerative Medicine (ARM) Regulatory Committee, and a member of the ARM Government Affairs, and the Science and Technology Committees.  She is a frequent speaker at advanced therapy conferences and is a collaborator on planning programs in advanced therapy. Kathy received her Bachelor of Science Biology from Temple University, Juris Doctorate from Widener University Law School, and is admitted to the practice of law in Pennsylvania and New Jersey.

Sponsors

Grant Support

This program is supported in part by an educational grant from Merck and Co., Inc.

Promotional Partners

Alliance for Regenerative Medicine

The Helen L. and Martin S. Kimmel Center for Stem Cell Biology

Nature

Wake Forest Institute for Regenerative Medicine

The Biochemical Pharmacology Discussion Group is proudly supported by

  • Boehringer Ingelheim
  • Pfizer

Abstracts

iPSC based Cell Therapy: Issues and Solutions
Mahendra Rao, PhD, Wake Forest University School of Medicine

The discovery of induced pluripotent stem cells (iPSCs) and concurrent development of protocols for their cell-type specific differentiation have revolutionized studies of diseases and raised the possibility that personalized medicine may be achievable. Realizing the full potential of iPSCs will require addressing the challenges inherent in obtaining appropriate cells for millions of individuals while meeting the regulatory requirements of delivering therapy and keeping costs affordable. Critical to making PSC based cell therapy widely accessible is determining which mode of cell collection, storage and distribution, will work. In this presentation I will present our results in generation of clinically compliant cells and the cost reduction strategies we have implemented to make such therapy feasible. I will discuss the issues related to developing an iPSC bank and how it could be constructed as a solution to delivering personalized medicine.

Engineering Genetic Cures: Genome Editing of Stem Cells
Edward O. Lanphier, Sangamo BioSciences, Inc.

Advances in human hematopoietic stem cell (hHSC) isolation, purification and re-engraftment, combined with the ability to engineer zinc finger DNA binding protein nucleases (ZFNs) that enable the targeted and permanent modification of any investigator chosen gene sequence, can create therapeutic outcomes capable of generating lifelong genetic cures. This approach is being applied in HIV (targeted disruption of the CCR5 gene in HSCs) and in beta-thalassemia and sickle cell disease (targeted disruption of BCL11A). However, the generality of the ZFN platform permits the targeted disruption and targeted gene correction of any therapeutically relevant gene, thus providing a platform for engineering genetic cures through genome editing of stem cells.

Stem Cell Therapies for Neurodegenerative Diseases
Jonathan D. Glass, MD, Emory University School of Medicine

There is growing interest in clinical experimentation for the use of stem cell therapy as a treatment of neurodegenerative diseases. Over the past decade stem cells from various sources have been transplanted into people with a variety of disorders ranging from childhood diseases of the brain to age-related disorders such as Parkinson’s disease, Alzheimer’s disease and amyotrophic lateral sclerosis (ALS). This presentation will provide an overview of worldwide efforts at developing stem cell therapies for neurodegenerative diseases, with a focus on ALS, including the presenter’s personal experience with phase 1 and 2 trials of intraspinal transplantation of human spinal cord-derived neural stem cells. Stem cell therapies for neurodegenerative diseases certainly hold promise, but we do not yet have a firm understanding of cell fates and mechanisms of action, both of which will be required in moving these kinds of treatments from promise to practice.

Long-term Relapse-free Survival of Patients with Acute Myeloid Leukemia (AML) Receiving a Telomerase-engineered Dendritic Cell Immunotherapy
John F. DiPersio, MD, PhD1

There are few treatment options for patients with intermediate and high risk AML. A Phase 2 clinical trial was conducted in subjects with AML to assess a dendritic cell immunotherapy (AST-VAC1) engineered to express a modified form of telomerase that is processed through the MHC Class I and II antigen presentation pathway. AML patients were enrolled if they were in complete remission with intermediate or high risk cytogenetics.  AST-VAC1 containing 1 x 107 cells was administered as 6 weekly followed by 6 biweekly intradermal injections.   Twenty one patients in complete remission (16 CR1 and 3 CR2) and or early relapse (2) received AST-VAC1.  The majority of adverse events were transient including headache, fatigue, and erythema. Of the 19 patients that were in CR, 58% (11/19) developed hTERT-specific T cell responses. Eleven of 19 patients (median follow-up 52 mos.) were in remission as of last follow-up; seven developed detectable cellular immune responses to hTERT. Of the 19 CR patients, 7 were ≥ 60 years old at the time of AST-VAC1 immunotherapy. Four of 7 patients ≥ 60 years old remained relapse free 52-59 months post AST-VAC1 immunotherapy with all four developing immune responses to hTERT. The three patients that received AST-VAC1 while in CR2 were in remission as of their last follow-up of 24, 50 and 59 months with two having hTERT immune responses. The results suggest that immunotherapy with AST-VAC1 is safe, can stimulate immune responses to telomerase, and may extend relapse-free survival even in patients with high risk AML.
 
Coauthors: H. Jean Khoury, MD, FACP2, Robert H. Collins Jr., MD3, William Blum, MD4, Patrick Stiff, MD5, Edward Wirth III, MD PhD6, Kevin Nishimoto PhD6, and Jane S Lebkowski, PhD6
1 Washington University School of Medicine, Siteman Cancer Center, Saint Louis, MO
2 Winship Cancer Institute, Emory University School of Medicine, Atlanta, GA
3 UT Southwestern Med. Ctr. at Dallas, Dallas, TX
4 The Ohio State University, Columbus, OH
5 Medicine, Loyola University, Maywood, IL
6 Asterias Biotherapeutics., Fremont, CA

Clinical Assessment of hESC-derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Sensorimotor-Complete Thoracic and Cervical Spinal Cord Injuries
Jane Lebkowski PhD1

Spinal cord injury (SCI) produces numerous life-changing clinical sequelae. The complex pathology in SCI restricts repair of the lesion by classic pharmacological drugs. AST-OPC1 are oligodendrocyte progenitors differentiated from human embryonic stem cells which promote remyelination of axons, produce neurotrophic factors and stimulate vascularization of the injury site. A phase 1 clinical trial assessing the safety of AST-OPC1 has been completed in 5 subjects with neurologically-complete T3-T11 thoracic SCI. All subjects were administered a low dose of 2 x 106 AST-OPC1 within 14 days of injury. Subjects received a low dose of tacrolimus which was tapered and discontinued by day 60. All 5 patients have been followed for over 4 years. There have been no serious adverse events related to AST-OPC1, tacrolimus, or the injection procedure. There were no unexpected changes in neurological function. A second Phase 1/2 clinical trial is underway in patients with sensorimotor complete C5-C7 cervical SCI patients. In this trial, 3 cohorts of patients are receiving escalating doses of 2-20 x 106 AST-OPC1 14-30 days post-injury. The trial is designed to examine safety of AST-OPC1 and the potential effects on upper extremity motor function including improvements in neurological level of motor function. Improvements in 2 or more levels of neurological function in the patient population would translate into clinically significant improvements in ability for self-care, independence and quality of life. After enrollment of 13 patients, it is intended to expand enrollment in the trial to 40 patients to provide further safety and activity data regarding AST-OPC1.
 
Coauthors: Richard Fessler MD2, David Chen MD3, Gary Steinberg MD, PhD4, Stephen McKenna MD5, David Apple MD6, Donald Peck Leslie MD7, Maria Schaefer1, Linda Jones8, Edward Wirth MD, PhD1
 
1 Asterias Biotherapeutics Inc 6300 Dumbarton Circle Fremont California 94555
2 Dept of Neurosurgery Rush University 1725 W Harrison St Suite 855 Chicago Illinois 60612
3 Rahabilitation Institute of Chicago 345 Superior Street Chicago Illinois 60611
4 Department of Surgery, Stanford School of Medicine, 300 Pasteur Drive Stanford California 94305
5 Dept of Physical Medicine and Rehabilitation Santa Clara Valley Medical Center 751 South Bascom Avenue San Jose California 95128
6 Shepherd Center Care Hospital 2020 Peachtree Road NW Atlanta Georgia 30309
7 Shepherd Center and Crawford Research Institute 2020 Peachtree Road NW Atlanta Georgia 30309
8 Craig H Neilson Foundation 16830 Ventura Blvd Suite 352 Encino California 91436
 

Mechanism of Action of PLX - Placental-Derived Cellular Therapy
Racheli Ofir, PhD, Pluristem LTD

PLX are 3D-expanded placenta-derived cells with biological properties including a profound capacity to induce therapeutic effect via secretion of a broad array of cytokines. Different cell populations possess different biological properties making them suitable for different therapeutic indications. PLX-PAD, developed for the treatment of Peripheral Artery Disease (PAD). PLX‑PAD intramuscular administration in mice with experimentally-induced hind limb ischemic damage significantly improved limb function, blood flow to the ischemic limb, and muscle regeneration. Pluristem has completed 3 clinical studies with PLX-PAD demonstrating efficacy and safety following single or repeated unmatched cell administration. PLX-R18 secrete a broad array of cytokines that contribute to the reconstitution of the hematopoietic system. PLX-R18 administered intramuscularly to a mouse model of Radiation induced Bone Marrow (BM) failure significantly improved survival, weight reduction and cell counts of the three hematopoietic lineages in the BM and blood as compared to control animals. Analysis of plasma in the irradiated treated animals detected the presence of critical, PLX-R18-derived (human) cytokines as well as alterations in the equivalent murine cytokines, suggesting a direct role of PLX-R18 secreted cytokines in animal recovery. Interestingly, human cytokines were detected only after irradiation and were not present in the plasma of sham non-irradiated animals treated with PLX‑R18. This may imply that the active in vivo cytokine secretion by PLX-R18 is a response to signals from the environment in the irradiated animals. The accumulated data demonstrates a critical role for cross talk between administered PLX cells and Host to facilitate a therapeutic effect.

Development of Stem-Cell Derived, Macroencapsulated Islet Replacement for Type 1 Diabetes
Thomas Shulz, PhD, ViaCyte, Inc.

ViaCyte Inc. is a clinical stage company developing a stem cell-based islet replacement therapy for treatment of patients with diabetes. The therapy is a combination product comprised of pancreatic endoderm cells encapsulated within a retrievable delivery device, ENCAPTRA® Drug Delivery System.  After implantation, encapsulated progenitor cells differentiate into glucose-responsive, insulin-secreting cells. The renewable starting material for cell product manufacturing is human embryonic stem cells that are directed to differentiate to the pancreatic endoderm cell product using scalable processes. The bio-stable delivery device is designed to fully contain cells and to protect cells from immune attack, with the goal of eliminating the need for immunosuppressant drugs.
 
Funding in part from California Institute for Regenerative Medicine; SP1-06513, DR1-01423, TR1-01215 and JDRF.

Neural Stem Cell Treatment for Stroke Disability
John D. Sinden, PhD1

Over 10 years ago, we isolated from a single fetal tissue sample the conditionally-immortalized human neural stem cell line, CTX from human first trimester cortex. Based on promising in vitro and in vivo data, we invested in full cGMP manufacture. The c-mycER technology enables substantial cell expansion, with our three-tier serial frozen cell banks (MCB, WCB, drug substance-DS) strategy. Drug product (DP) for patients is manufactured from frozen DS also to GMP and delivered to the clinic as either a fresh or, more recently, frozen product ready for implantation. Based on published preclinical studies we are developing the CTX DP primarily for stroke disability In chronic stroke patients the Ph I dose-escalation safety trial, PISCES, has recently completed in Scotland and a Ph II multi-center efficacy trial has recently started, evaluating recovery of paretic upper limb motor function in stroke patients up to 8 weeks post-infarct. Data from the Ph I trial will be presented along with plans for the future commercial development of a first line stem cell treatment for motor disability.
 
Coauthors: R. Paul Stroemer, PhD1, Kenneth Pollock, PhD1, Laurence T. Dunn, PhD M.B. B.Ch2, Dheeraj Kalladka, M.B. B.S.2 and Keith W. Muir MB BS2
1 ReNeuron Limited, Guildford, Surrey, United Kingdom
2 Insitute of Neuroscience and Psychology, University of Glasgow, Glasgow, United Kingdom.

Overview of the Challenges Faced and Opportunities Available to Expedite Development for Advanced Therapies
Katherine Tsokas, JD, Janssen Research & Development

Advanced therapies have the promise to meet unmet medical needs and bring value added therapies to patients. Sponsors and regulators are looking for opportunities to accelerate development and expedite marketing authorization of these novel therapies. However, development and review of advanced therapies has unique challenges. An understanding of the challenges, potential mitigations and opportunities for expedited regulatory pathways must be part of the regulatory strategy for these novel products. In addition, this understanding by the various industry stakeholders (e.g., academics, sponsors and regulators) is necessary for determining collectively our next steps in shaping the regulatory environment for advanced therapies.

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