Regenerative Medicine

Regenerative medicine hopes to protect, replace or engineer human cells to restore diseased tissues back to a healthy homeostasis. By using stem cells, engineered biomaterials and gene editing approaches, labs around the world are  fighting the rise of chronic and degenerative diseases. Cambridge Healthtech Institute’s Inaugural Regenerative Medicine symposium will focus on the latest tools, targets and platforms. 

Final Agenda

Tuesday, September 25

8:00 am Pre-Conference Symposia and Short Course Registration

8:00 Morning Coffee

Stem Cell Harvesting And Harnessing

9:00 Welcome Remarks

Mana Chandhok, Associate Conference Producer, Cambridge Healthtech Institute

9:05 Chairperson’s Opening Remarks

Evan Y. Snyder, MD, PhD, Professor and Director, Sanford Burnham Prebys Medical Discovery Institute

9:15 Drive Thru Stem Cells

Hoggatt_JonathanJonathan Hoggatt, PhD, Assistant Professor of Medicine, Harvard Medical School Cancer Center and Center for Transplantation Sciences, Massachusetts General Hospital, Harvard Stem Cell Institute

Bone marrow transplant is growing, and with emerging stem cell gene therapies, the field is on the verge of rapidly moving into more non-malignant transplants. The ability to easily acquire high quality stem cells from donors and patients will be key to wide utility and success of stem cell transplant. This talk will outline the challenges and discuss a novel method for stem cell harvesting.

9:45 Harnessing Endogenous Repair Mechanisms to Promote Tissue Regeneration: Cure from the Inside

Franti_MichaelMichael Franti, PhD, Director, Regenerative Medicine, Research Beyond Borders, Boehringer Ingelheim Pharmaceuticals, Inc.

Stem cells are immature precursor cells with the ability to maintain the undifferentiated state, to proliferate and to differentiate into specialized cells in response to injury. Despite the significant advances in stem cell biology, several issues have limited their application in cell-based therapy. For these reasons, researchers are focusing on the development of new therapeutic approaches to manipulate the endogenous stem cells as well as their tissue microenvironment to repair damaged tissues. Recent progress, new insights, and future challenges to identify drugs that induce activation of endogenous stem cells in vivo that may ultimately lead to innovative new medicines will be presented.

10:15 Commercializing Pluripotent Stem Cell Therapeutics for The Heart And Brain

Deans_RobertRobert Deans, PhD, Chief Technology Officer, BlueRock Therapeutics

BlueRock Therapeutics is an iPSC based cell therapeutics company developing allogeneic products for treatment of congestive heart failure and Parkinson’s disease.  BlueRock has employed gene editing tools to create master iPSC banks which do not express class I HLA, and express an inhibitor of innate immune clearance.  BlueRock is submitting an IND for Parkinson’s disease in 2018.

10:45 Late Breaking Presentation

11:00 Networking Coffee Break with Poster Viewing

Cns Therapies

11:30 Target Identification for CNS Repair Using iPSC Derived Neurons

Singh_BhagatBhagat Singh, PhD, FM Kirby Neurobiology Center, Boston Children’s Hospital, Harvard Medical School

Central nervous system (CNS) injuries cause permanent disability because of limited regrowth of injured CNS axons. We have developed a phenotypic neurite outgrowth assay using human iPSC derived motor and cortical neurons. We are performing a large scale unbiased phenotypic screen on known bioactive compounds to identify novel targets capable of enhancing axonal regeneration. This screening platform will open novel opportunities for development of therapeutics for CNS repair.

12:00 pm Using Stem Cells to Model Neurological Disease & Advance Personalized Medicine

Snyder_EvanEvan Y. Snyder, MD, PhD, Professor and Director, Sanford Burnham Prebys Medical Discovery Institute

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

12:30 The Development and Application of a Potency Assay to Evaluate Cell Therapies for Traumatic Brain Injury

Olson_ScottScott Olson, PhD, Assistant Professor, Department of Pediatric Surgery, UT Health

Cellular therapies for traumatic brain injury (TBI) have been difficult to translate. The field has experienced a number of promising preclinical and clinical trials that have later failed due to lack of efficacy and adverse events. We seek to advance the field using a collaborative development cycle, where real-world clinical use drives a seamless collaborative transition of work from preclinical research into cGMP manufacturing with both academic and industry applications.

1:00 Late Breaking Presentation

1:00 Pre-Conference Short Course Registration

1:15 Enjoy Lunch on Your Own

Aging, Small Molecule And Other Considerations

2:45 Chairperson’s Remarks

Michael Franti, PhD, Director, Regenerative Medicine, Research Beyond Borders, Boehringer Ingelheim Pharmaceuticals, Inc.

2:55 A Role for the Longevity Protein, Klotho, on Skeletal Muscle Regeneration

Ambrosio_FabrisiaFabrisia Ambrosio, PhD, PT, Associate Professor and Director of Rehabilitation, UPMC International, Physical Medicine & Rehabilitation, University of Pittsburgh

An age-related impairment of skeletal muscle regeneration is a major contributor to functional declines in an elderly population. Much of the healing defect has been attributed to an age-related decrease in muscle stem cell (MuSC) function. Fortunately, these age-related changes are reversible and studies show that rejuvenation of the microenvironment restores MuSC function in aged muscle. In her presentation, Dr. Ambrosio will discuss a potential role for Klotho in the maintenance of muscle regeneration over time.

3:25 Human Pluripotent Stem Cell-Based Disease Modeling and Drug Screening

Shuibing_Chen_20171212_318Shuibing Chen, PhD, Associate Professor of Chemical Biology in Surgery, Cornell University

We used human pluripotent stem cells derived pancreatic beta-like cells to explore the role of genetic and environmental factors in the progression of type 1 and type 2 diabetes. In addition, the platform was applied to identify a drug candidate that rescues gene-specific defects, which paves the way for precision therapy of metabolic diseases.

3:55 Small Molecules for Diabetes

Gourdie_RObertRobert G. Gourdie, PhD, Director and Professor, Center for Heart and Regenerative Medicine, Biomedical Engineering, and Emergency Medicine, Virginia Tech Carilion Research Institute

The gap junction protein Cx43 has key roles in wound healing, scarring, and regeneration. The Gourdie lab has developed a platform of drugs targeting Cx43 function. Following successful Phase I and Phase II clinical testing for indications in skin wound healing, including improving closure and resolution of diabetic foot ulcers and venous leg ulcers, the first of these compounds has begun pivotal Phase III testing on diabetic foot ulcers.

4:25 Immunology at the Intersection of Biomaterials and Regenerative Medicine

brown_BryanBryan N. Brown, PhD, Assistant Professor, McGowan Institute for Regenerative Medicine, University of Pittsburgh

Conventional approaches to biomaterial development focus upon inert immune reactions and long-lasting mechanical support. The emergence of regenerative medicine has driven advancements in biomaterial form and function. These advancements provide an impetus for re-examination of host-biomaterial interactions, including interface events, spatial and temporal patterns of remodeling, and downstream functional outcomes. A discussion of such issues is provided with a focus upon methods for modulating immune interactions at the host-biomaterial interface

4:55 Closing Comments

Michael Franti, PhD, Director, Regenerative Medicine, Research Beyond Borders, Boehringer Ingelheim Pharmaceuticals, Inc.

5:00 Close of Symposium

5:00 Pre-Conference Dinner Short Course Registration


Choose 2 Short Courses or 1 Symposium and 2 Conferences/ Training Seminars

September 25 Symposium: Regenerative Medicine

September 25 Short Course 9: CNS Translational Strategies

September 26-27 Conference: Target Identification and Validation - Part 1

September 27-28 Conference: Target Identification and Validation - Part 2

September 27 Short Course 18: Practical Phenotypic Screening