P R O G R A M

Connecticut’s Stem Cell and Regenerative Medicine Symposium

stemconn.org | stemconn2015@gmail.com

April 27, 2015Hartford Marriott Downtown

Hartford, Connecticut

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L E T T E R 1

Dear StemCONN participant,

Welcome to StemCONN 2015. We are thrilled to host our fifth StemCONN conference, in which we celebrate 10 years of accomplishments in stem cell and regenerative medicine research in Connecticut. During this time Connecticut has become an acknowledged world leader in this field. StemCONN 2015 highlights cutting-edge research advancements, and provides a forum for discussion among scientists, policy makers and our industry partners, in order to advance knowledge, stimulate new collaborations and foster clinical application and commercialization.

We are witnessing a very exciting time in stem cell research and regenerative medicine, as new discoveries in the laboratory lead to medical breakthroughs and clinical therapies never before imagined. Through skilled basic research, we are learning how stem cells function and how to use them to treat human injury and disease. We are also gaining new appreciation of the importance of the body’s own stem cells in maintaining organ health and promoting tissue repair and regeneration. The world-class science that will be shared at StemCONN 2015 embraces these themes.

StemCONN 2015 marks the 10th anniversary of the ground-breaking legislation that has propelled Connecticut to the forefront of stem cell and regenerative medicine research. Connecticut’s investment in bioscience initiatives continues to pay dividends for the state and its citizens — supporting major research advances, making possible state-of-the-art medical and research facilities, and creating opportunity and jobs. StemCONN 2015 demonstrates the success of bioscience as a vehicle for medical progress and economic growth by emphasizing the power of academia-industry partnerships and faculty entrepreneurship as a means to effectively move scientific discovery towards clinical application.

Science education is another important aspect of StemCONN’s mission. Nearly 75 scientific trainees, postdoctoral fellows and graduate students are presenting their latest research in our poster sessions today. This year, these trainees will have a special opportunity to meet with our invited speakers and one of them will be honored with the first Milton B Wallack Trainee Award for Excellence in Stem Cell and Regenerative Medicine Research. StemCONN 2015 also welcomes more than 100 college and high school students from the greater Hartford area and other parts of the state who are attending this conference through support from our educational sponsors.

We thank all of our conference sponsors for their generous financial support. Please refer to the sponsor list for the names of these contributors and, during the breaks, be sure to visit our Exhibitor Forum. Thanks also to my colleagues on the StemCONN 2015 organizing committee for their enduring commitment and contributions to planning this conference.

Have a great day!

Caroline Dealy, Ph.D.

Chair StemCONN 2015 Organizing Committee

On behalf of the University of Connecticut, including UConn Health, I am pleased to welcome you to StemCONN2015. As a founding member of the Connecticut research community and a nationally ranked research University, we are always striving for distinction in research and discovery. StemCONN enriches our efforts by creating a forum for collaboration, education and innovation. The size and strength of this meeting is due in large part to a state leadership that recognizes the powerful impact that bioscience research has for public health benefit and economic growth and makes major investments like Bioscience Connecticut.

The University of Connecticut is proud to be a major contributor to the State’s stem cell and regenerative medicine research efforts. Critical to this success is the State’s Stem Cell and Regenerative Medicine Initiative, which at the University of Connecticut and UConn Health alone has led to $40 million of funding supporting 75 investigators. These funds make possible discovery in areas of human health and disease including aging, memory, bone fracture and repair, osteoarthritis, heart attacks and cancer. Stem cell and regenerative medicine research projects in Connecticut support college and graduate students working toward health and science careers, some of whom now attend our state’s universities, medical or dental schools. These bright new stars will be the next generation to make, and to implement, life- saving discoveries in human medicine.

The University of Connecticut is committed to partnerships to promote medical advances and commercial opportunity. Through initiatives like its Technology Incubator Program, and its collaboration with Jackson Laboratories, the University of Connecticut encourages innovation and cooperation between academia and industry. These initiatives are important benchmarks for expansion and development of Connecticut’s bioscience industry, now leveraged with the creation of the Connecticut Bioscience Innovation Fund, which aims to accelerate the path between discovery and commercialization to facilitate new healthcare breakthroughs.

Sincerely,

Susan Herbst

President University of Connecticut

2 L E T T E R L E T T E R 3

Wesleyan University is pleased to be one of the sponsors of StemCONN 2015. Wesleyan has long had a deep commitment to interdisciplinary study, and the stem cell research of our life sciences faculty is a stellar example. In keeping with this year’s theme of stem cells and regenerative medicine, I take special pride in noting the work of Professors Laura Grabel, Gloster Aaron, and Janice Naegele, who have published several studies recently on the use of optogenetic approaches to test functional connectivity of stem cells grafted into the brain to treat temporal lobe epilepsy and ways to generate human inhibitory neurons for brain repair. They work closely with both graduate students and under-graduates — a model for how scientific research is conducted at Wesleyan.

Stem cell research is eagerly pursued by many of our faculty and students, and funding from the state of Connecticut is crucial in enabling those pursuits. That funding supports a productive and multifaceted stem cell research program at Wesleyan involving multiple laboratories, graduate students, postdoctoral fellows, and undergraduates. In supporting laboratory technicians and animal care staff, graduate stipends, and undergraduate summer research stipends, state funding is having a profound impact here on scientific research, on teaching, on training, and on outreach. Off campus, you see the impact in scientific publications and outreach educating the public on stem cell therapies for diseases. On campus, the research of Wesleyan’s scholar-teachers informs their teaching, and discoveries made in the laboratory quickly find their way into the classroom.

StemCONN provides the opportunity to further enrich perspectives on stem cell research through exchange, dialogue and collaboration. Those perspectives will be carried back by participants from Wesleyan to their the labs, their classrooms, and beyond.

Looking at the roster of speakers and sponsors, I see StemCONN as a model of how private and public institutions can work together in pursuing goals of profound importance, and I’m delighted Wesleyan is a part of it.

Michael S. Roth

President Wesleyan University

Welcome! I am delighted that over the course of this — the fifth biannual StemCONN — Yale scientists will join with colleagues from across Connecticut, throughout the Northeast, and around the country to examine this crucial area of research.

Few states’ educational and research facilities can compare to those of Connecticut: resources that would not exist without the state’s support of the biosciences and stem-cell research. Our community comprises both scientists and advocates of stem-cell research. Their efforts contribute not only to advancement of the field, but also to the fiscal health of the state by bringing in research funding and creating new jobs.

I am proud that the more than 70 investigators of the Yale Stem Cell Center are a part of this much larger regional community of researchers, clinicians, and students — all working together to advance understanding of stem-cell biology and to harness its potential to improve human health. On behalf of Yale University, thank you for participating in this important endeavor.

Sincerely,

Peter Salovey

President and Chris Argyris Professor of Psychology Yale University

4 T R I B U T E S C H E D U L E 5

Symposium Breakfast

7:30 – 8:30 Registration

Lobby Level

Continental Breakfast

Pre-Registered Symposium Attendees: The Capital Room StemConn Attendees: Marriott Foyer

7:45 – 8:30 Symposium Breakfast Lecture

The Capital Room; pre-registration required.

Ron Parchem, Ph.D.

University of California San Francisco, Eli and Edythe Broad Center for Regenerative Medicine

MicroRNA Regulation of Reprogramming and Development

Symposium

8:45 – 9:00 Welcome / Connecticut Stem Cell Update

Marriott Ballroom CDE

9:00 – 9:45 Robert Lanza, M.D.

Chief Scientific Officer, Ocata Therapeutics;

Professor, Institute for Regenerative Medicine, Wake Forest University School of Medicine.

Moving the First Pluripotent Stem Cell Therapies to the Clinic

9:45 – 10:15 Laura Niklason, M.D., Ph.D.

Professor of Anesthesiology and Biomedical Engineering, Yale University;

Founder, Humacyte.

Taking an Engineered Artery from the Bench into the Clinic

10:15 – 11:00 Coffee Break, Posters and Exhibitor Forum

Ballroom AB & Marriott Foyer

11:00 – 11:30 Craig Nelson, Ph.D.

Ballroom CDE

Associate Professor of Molecular and Cell Biology, Program in Genetics and Genomics, University of Connecticut;

Founder, Smpl Bio.

Deconstructing and Reconstructing Complex Systems with Single-Cell Analysis

11:30 – 12:00 Gloster B. Aaron, Ph.D.

Associate Professor of Neuroscience and Behavior, Wesleyan University

How Transplants Reduce Seizures: Brain Slice Electrophysiology and Optogenetic Stimulation of Transplanted Cells

Continued on page 6

Milton B. Wallack Trainee Award for Excellence in Stem Cell and Regenerative Medicine Research

We are delighted to announce that StemConn2015 is honoring excellence in research conducted by a predoctoral or postdoctoral trainee through a merit-based award that recognizes highly innovative and important stem cell research. This Trainee Award is presented

in honor of Dr. Milton B. Wallack, the founder of the CT Stem Cell Coalition, a longtime member of the CT Stem Cell Research Advisory Committee (SCRAC), and an ardent champion for stem cell research in Connecticut.

The naming of this award both acknowledges and honors Dr. Wallack for his unwavering support of stem cell research in the state of Connecticut. Thanks in large part to his efforts, Connecticut passed the Stem Cell Investment Act in 2005. This Act, supported by our state legislators, our former Governor Jodi Rell, and the people of Connecticut, committed $10 million to be allocated over 10 years and made Connecticut a major player in stem cell research. Success in this endeavor required countless hours; Dr. Wallack initiated the CT stem cell coalition with Paul Pescatello at CURE, lobbied to promote stem cell research, established meaningful relationships with members of the state legislature, and forged long-lasting connections between investigators throughout the state. Since the passage of the bill, Dr. Wallack has continued to work tirelessly, maintaining relationships with the legislature to ensure that funding for stem cell research in Connecticut continues through the next 4 years. He also helped to organize the StemConn meeting and has attended all of our tri-institutional retreats. He served consecutive terms as a devoted and hardworking member of the Stem Cell Research Advisory Committee (now known as the Regenerative Medicine Research Advisory Committee), wrote countless editorials to local newspapers in favor of federal and state stem cell funding, helped Yale and UConn with fundraising efforts, and served as a member of the state economic development committee. In addition to this extraordinary work in support of stem cell research, he has contributed significantly to the local Jewish community and worked to promote diabetes care, diabetes research, and educational efforts throughout Connecticut and nationwide.

Dr. Milton Wallack has served the Connecticut Stem Cell community with unflagging optimism, diligence, and hard work. Dr. Wallack has truly devoted his life to making the world a better place, and we remain deeply grateful for his exemplary commitment to our cause. His leadership is worthy not only of honor but also emulation.

The StemCONN2015 Organizing Committee

A B S T R A C T / B I O G R A P H Y 76 S C H E D U L E

Ron Parchem, Ph.D.

University of California San Francisco, Eli and Edythe Broad Center for Regenerative Medicine

MicroRNA Regulation of Reprogramming and Development

The ability to reprogram somatic cells into pluripotent stem cells has revolutionized the field of regenerative medicine. Like embryonic stem cells, these induced pluripotent stem cells (iPSCs) can be differentiated into various therapeutic cell types. Recent work has shown that microRNAs (miRNAs) have remarkable potential as therapeutic agents and biomarkers of disease; however, their functions are still poorly understood. Here, I will discuss the role of miRNAs in two opposing processes: (1) the acquisition of pluripotency during somatic cell reprogramming, and (2) the loss of pluripotency during embryonic development. Specifically, I will discuss the paths taken by cells during the reprogramming process by following the transcriptional activation of two pluripotent miRNA clusters, mir-290 and mir-302, in individual cells in vivo and in vitro using knock-in reporters. Our results demonstrate that cells can follow multiple paths during late stages of reprogramming, and that the trajectory of any individual cell is strongly influenced by the combination of factors introduced. Additionally, I will discuss the role of mir-302 during mouse embryonic development where it is essential for neural tube closure and embryonic viability. Our results suggest mir-302 is a regulator of neural differentiation and developmental timing. These studies demonstrate essential roles for pluripotent miRNAs in stem cell differentiation, embryonic development, and somatic cell reprogramming.

Ronald J Parchem, Ph.D. has been a postdoctoral fellow in the lab of Dr. Robert Blelloch at the Eli and Edythe Broad Center for Regenerative Medicine at the University of California San Francisco since 2009. Here, his work has focused on the most highly expressed microRNAs in embryonic stem cells which are critical regulators of pluripotency and reprogramming. Additional studies have showed that these same miRNAs are essential

for mouse embryonic development by regulating several developmental processes. Dr. Parchem earned his B.S. from the University of Illinois and his PhD from the University of California, Berkeley studying the evolution of development and body patterning. He has published on topics ranging from butterfly wing patterning, genomic organization of the amphipod, Parhyale hawaiensis, and somatic cell reprogramming.

Symposium (continued)

12:00 – 1:00 Lunch & Speaker-Trainee Lunch Groups

Box lunches are offered in the Capital Room and Exhibitor Forum area for conference participants who are not attending a Speaker-Trainee Lunch (below).

12:00 – 1:00 Speaker-Trainee Lunch

Conference Rooms 4, 5 and 7; for postdoctoral fellows & graduate students only; pre-registration required.

1:15 – 2:15 Commercialization & Translation Session:

Ballroom CDE

1:15 – 1:45 Frank McKeon, Ph.D.

Senior Group Leader, Genome Institute of Singapore;

Chief Scientist, Multiclonal Therapeutics.

Adult Stem Cells: Next Era of Regenerative Medicine and Disease Modeling?

1:45 – 2:15 Craig Crews, Ph.D.

Lewis B Cullman Professor of Molecular, Cellular and Developmental Biology; and Professor, Chemistry and Pharmacology, Yale University;

Founder, Arvinas.

Faculty Entrepreneurism: The What, How and Why

2:15 – 3:00 Carla Kim, Ph.D.

Associate Professor, Boston Children’s Hospital, Stem Cell Program;

Department of Genetics, Harvard Medical School.

Using Stem Cell Biology to Understand Lung Repair and Regeneration Mechanisms

3:00 – 3:45 Coffee Break, Posters and Exhibitor Forum

Ballroom AB & Marriott Foyer

3:45 – 4:10 Milton B. Wallack Trainee Award for Excellence in Stem Cell & Regenerative Medicine Research

Ballroom CDE

Arpita Biswas

Department of Molecular and Cell Biology, University of Connecticut

Stem Cell Dysfunction in Musculoskeletal Disease

4:10 – 4:55 David Kaplan, Ph.D.

Stern Family Endowed Professor of Engineering;

Professor & Chair of the Department of Biomedical Engineering, Tufts University.

Biopolymer Engineering for Stem Cell-Based Regenerative Medicine

4:55 – 5:00 Closing Remarks

Laura Niklason, M.D., Ph.D.

Professor of Anesthesiology and Biomedical Engineering, Yale University;

Founder, Humacyte.

Taking an Engineered Artery from the Bench into the Clinic

We have previously described a method for vascular regeneration that comprises seeding of vascular smooth muscle cells onto scaffolds that are cultured in a bioreactor in the laboratory. After culture, engineered blood vessels are treated to wash away the cells, leaving behind only the proteins that the cells secreted, such as collagens, which form a mechanically robust tubular tissue. Tissue engineered vascular grafts have been grown and tested in a set of preclinical models of arterial replacement, including coronary artery bypass grafting and vascular grafting for hemodialysis access. In each of these preclinical settings (swine, canine and primate) the engineered, non-cellular blood vessels were safe, well-tolerated and functioned as intended. The non-cellular vessels exhibited excellent mechanical properties prior to implant, comparable to those of native human vein and artery. Furthermore, the mechanical properties of the grafts strengthened after implantation, and no graft exhibited narrowing or dilatation. Based on the successful and robust preclinical data sets, regulatory bodies both in Europe and the United States have approved first-in-man testing of these bioengineered blood vessels for hemodialysis access grafting. To date, 60 grafts have been successfully implanted in patients as a vascular access graft for use in hemodialysis, with clinical follow-up ongoing. All implanted grafts have been suitable for hemodialysis access with excellent flow rates, with no evidence of structural degeneration or immunologic rejection. This emerging technology is poised to be a real alternative to conventional synthetic vascular prosthesis.

Laura Niklason, M.D., Ph.D., is a Professor of Anesthesia and Biomedical Engineering at Yale, and a leader in the engineering of arteries in vitro. Her research interests include vascular tissue engineering, adult stem cell differentiation into cardiovascular phenotypes, cardiac tissue engineering, biomimetic culture conditions for cardiovascular tissues, and vascular remodeling as it pertains to disease processes, including

vasospasm. Dr. Niklason received her PhD in Biophysics from University of Chicago, and her MD from the University of Michigan, where she also did her internship. She then went on to the Massachusetts General Hospital for residency training in Anesthesia, and a fellowship in Critical Care Medicine. During her time in Boston, Dr. Niklason was a post-doctoral researcher at MIT with Dr. Robert Langer, where she developed techniques for the tissue engineering of arteries. After becoming a faculty member at Duke University, she founded Humacyte, a biotechnology company designed to bring tissue engineered cardiovascular products to the clinic. Since 2006, Dr. Niklason has been at Yale University, where she is expanding her research program in tissue engineering of blood vessels and lung, as well as understanding the basic aspects of cellular aging. Dr. Niklason has received national and international recognition for her work including receipt of a Discover Magazine award for Technological Innovation, and selection as a US News & World Report Innovator and as a Fellow in the National Academy of Inventors.

8 A B S T R A C T / B I O G R A P H Y A B S T R A C T / B I O G R A P H Y 9

Robert Lanza, M.D.

Chief Scientific Officer, Ocata Therapeutics;

Professor, Institute for Regenerative Medicine, Wake Forest University School of Medicine.

Moving the First Pluripotent Stem Cell Therapies to the Clinic

Ever since their discovery over three decades ago, embryonic stem cells (ESCs) have been touted as the future of regenerative medicine, with a heavy burden of promise placed upon them to deliver an unprecedented number of cell-based therapies. In theory, their ability to undergo unlimited self-renewal and to generate any cell type in the body makes pluripotent stem cells (PSCs) like ESCs and induced pluripotent stem cells (iPSCs) an ideal starting material for treating a wide variety of diseases. Yet, in reality, potentially serious risks including the propensity to form tumors or trigger an immune response, and technical hurdles in directing their in vitro differ-entiation have thwarted efforts to bring PSC-based therapies to the clinic. After decades of work, clinical trials of PSC-derivatives are finally under-way in United States, Europe and Asia. Some of the recent clinical progress that has been made will be discussed, including several other potential PSC applications: the use of retinal pigment epithelium (RPE) and photo-receptor progenitors for the treatment of a variety of retinal degenerative diseases, the use of hemangioblasts for vascular restoration of organs and limbs, and to generate functional platelets and “universal donor” red blood cells for human transfusion, and the use of mesenchymal stem cells (MSCs) to treat immune-mediated and inflammatory diseases, among others. Preclinical progress using these cells to affect substantial rescue in animals will be presented, as well as an overview of our ongoing human embryonic stem cells trials evaluating the safety and tolerability of sub-retinal transplantation of hESC-derived RPE in patients with age-related macular degeneration and Stargardt’s macular dystrophy.

Robert Lanza, M.D. is Chief Scientific Officer at Ocata Therapeutics (formerly Advanced Cell Technology), and professor at the Institute for Regenerative Medicine at Wake Forest University School of Medicine. He has several hundred publications and inventions, and 30 scientific books: among them, “Essentials of Stem Cell Biology” and “Principles of Tissue Engineering” which are recognized as the definitive references

in the field. He is a former Fulbright Scholar, and studied as a student with immunologist Jonas Salk and Nobel laureates Gerald Edelman and Rodney Porter. He also worked closely (and co-authored a series of papers) with noted Harvard psychologist B.F. Skinner and heart transplant pioneer Christiaan Barnard. Dr. Lanza received his undergraduate and medical degrees from the University of Pennsylvania, where he was both a University Scholar and Benjamin Franklin Scholar. He has made numerous break-throughs in the field of stem cells and regenerative medicine. Lanza and his colleagues published the first-ever report of pluripotent stem cell use in humans. Among other achievements, Lanza and colleagues succeeded in differentiating pluripotent stem cells into RPE, and received FDA approval for clinical trials using them to treat degenerative eye diseases, including age-related macular degeneration (AMD), an untreatable eye disease that is a major cause of blindness. His company also received approval from the UK’s MHPRA to carry out the first-ever pluripotent stem cell trial in Europe. He has received numerous awards, including TIME Magazine’s 2014 TIME 100 list of the “100 Most Influential People in the World,” the 2013 Il Leone di San Marco award in Medicine, an NIH Director’s Award (2010) for “Translating Basic Science Discoveries into New and Better Treatments”; the 2010 “Movers and Shakers” Who Will Shape Biotech Over the Next 20 Years; and the 2005 Rave Award for Medicine.

Gloster B. Aaron, Ph.D.

Associate Professor of Neuroscience and Behavior, Wesleyan University

How Transplants Reduce Seizures: Brain Slice Electrophysiology and Optogenetic Stimulation of Transplanted Cells

There are many kinds of epilepsies. Temporal lobe epilepsy (TLE) is among the most common as well as most resistant to current therapies, and so there are personal and societal incentives for developing a greater range of options for treating this widespread neurological affliction. Our labs (Profs. Grabel, Naegele, and Aaron) have been pursuing the goal of healing damaged areas of the brain that are the source of seizures in TLE by providing newborn neurons to those areas. Ideally, these newborn neurons would replace dead neurons and repair broken neuronal circuits that are thought to be a cause of TLE. As a proof of principle, Janice R. Naegele and her colleagues at Wesleyan University recently demonstrated that the number of seizures in mice with TLE can be reduced by transplantation of neural progenitors harvested from areas of fetal mouse brains that produce a kind of neuron most often lost in TLE, ie, inhibitory interneurons. A loss of neuronal inhibition is a mechanism that promotes seizures, so increasing neuronal inhibition is a means of stopping and/or preventing seizures. My lab performed experiments used to determine the likely mechanisms by which this seizure reduction occurred. Our work was performed on brain slices made from two groups: mice with TLE vs. mice with TLE and transplants. In one set of experiments, we measured the amount of inhibitory currents in neurons near the transplants, and we found that mice with TLE and transplants had higher levels of inhibitory currents. To determine whether those inhibitory currents arose from the transplanted cells, transplants were given a protein that, when stimulated with blue light, caused the transplant cells to become very active (a technique called optogenetics). We then recorded cells near the trans-plants, stimulated the transplants, and showed that they produced large inhibitory events. Our results thus suggested that transplanted cells reduced seizures by increasing inhibition in the nearby neurons.

Gloster Aaron, Ph.D., is an Associate Professor of Biology, with a joint appointment in the Neuroscience and Behavior Program at Wesleyan University, CT since 2006. His lab studies seizure propagation across callosal circuits in addition to a series of collaborative stem cell projects with Profs. Janice Naegele and Laura Grabel. His work in these latter projects includes determining the fate of progenitor cells after

they are transplanted into the host brains. Dr. Aaron received his B.A. with High Honors in Neuroscience at Oberlin College, OH, worked as a technician at the Pennsylvania State Hershey Medical Center, earned a Ph.D. in Neuroscience at the University of Pennsylvania, and then worked as a postdoc at Columbia University under the mentorship of Dr. Rafael Yuste. He has published work on topics ranging from the rat somatosensory system in vivo, short term synaptic plasticity in cultured hippocampal cells in vitro, neuroinformatics studies in the rodent neocortex, and the mechanisms by which fetal stem cells may alleviate seizures in mice with temporal lobe epilepsy.

1 0 A B S T R A C T / B I O G R A P H Y A B S T R A C T / B I O G R A P H Y 1 1

Craig Nelson, Ph.D.

Associate Professor of Molecular and Cell Biology, Program in Genetics and Genomics, University of Connecticut;

Founder, Smpl Bio.

Deconstructing and Reconstructing Complex Systems with Single-Cell Analysis

Biological systems like animals, plants, organs, and tissues are complex mixtures of many cell types. In order to better understand these systems, and to use that understanding to improve health care, agriculture, and other areas touched by biotechnology, biologists have long worked to identify and characterize the cell types present in biological systems. Around 2006, with the help of a seed grant from the Connecticut Stem Cell Research Program, our lab began taking a very direct approach to this problem by measuring gene expression in one cell at a time. Recently, technological advances in microfluidics have vastly expanded our ability to run this kind of single-cell analysis, and our lab leverages these tools to understand complex biological systems such as stem cells, embryos, and organs. This work has provided an unprecedented level of resolution in our understanding of these systems and has laid the foundation for the comprehensive and systematic identification of all human cell types. In the mean-time, we have used the computational tools we developed to support our single cell research to found a new bioinformatics company, Smpl Bio, LLC. In its first year Smpl Bio has hired four UConn graduates to full-time positions, and secured a contract with the market leader in single cell genomics to provide our unique software solutions to single-cell biologists around the world.

Craig Nelson, Ph.D., is an Associate Professor in the Department of Molecular and Cell Biology at the University of Connecticut, and founder of Smpl Bio, a Connecticut bioinformatics startup. Dr. Nelson earned his BA in Biochemistry at Cornell University, and his Ph.D. in Genetics at Harvard University. Trained as an experimental embryologist during the rise of “Evo-Devo”, Dr. Nelson has published widely on many aspects

of embryology, developmental biology, computational biology, and gene family evolution. Since 2006, Dr. Nelson has been turning the focus of his research program and his company toward single cell analysis. While Dr. Nelson and his collaborators utilize single cell analysis and computational modeling to understand complex processes, such as somatic cell reprogramming and developmental decision making by embryonic progenitor cells and their niches, Smpl Bio translates this expertise into powerful and easy to use experimental design and analysis tools for single cell biologists.

1 2 A B S T R A C T / B I O G R A P H Y A B S T R A C T / B I O G R A P H Y 1 3

Craig Crews, Ph.D.

Lewis B Cullman Professor of Molecular, Cellular and Developmental Biology; and Professor, Chemistry and Pharmacology, Yale University;

Founder, Arvinas.

Faculty Entrepreneurism: The What, How and Why

Connecticut has a large untapped resource of potential biotech/biopharmas within its academic laboratories. To unlock this potential, three things are needed to translate that research into new ventures: 1) the infrastructure to generate the additional data needed to attract external investment, 2) a program to educate and guide our state’s academic researchers through these entrepreneurial endeavors, and 3) experienced leadership that has successfully bridged the worlds of academia, biopharma and venture capital. I will describe past and current efforts to assist Connecticut biomedical researchers in translating their research into research tools, drugs and biopharma ventures.

Dr. Crews is the Lewis B. Cullman Professor of Molecular, Cellular and Developmental Biology and holds joint appointments in the departments of Chemistry and Pharmacology at Yale University. He graduated from the U.Virginia with a B.A. in Chemistry and received his Ph.D. from Harvard University in Biochemistry. Dr. Crews has a foothold in both the academic and biotech arenas; on the faculty at Yale since

1995, his laboratory has pioneered the use of small molecules to control intracellular protein levels. In 2003, he co-founded Proteolix, Inc., whose proteasome inhibitor, Kyprolis™ recently received FDA approval for the treatment of multiple myeloma. Since Proteolix’s purchase by Onyx Pharmaceuticals in 2009, Dr. Crews has focused on a new drug development technology, which served as the founding IP for his latest New Haven-based biotech venture, Arvinas, Inc. Currently, Dr. Crews serves on several editorial boards and is Editor of Chemistry&Biology. In addition, he has received numerous awards and honors, including the 2013 CURE Entrepreneur of the Year Award and 2014 Ehrlich Award for Medicinal Chemistry.

Frank McKeon, Ph.D.

Senior Group Leader, Genome Institute of Singapore;

Chief Scientist, Multiclonal Therapeutics.

Adult Stem Cells: Next Era of Regenerative Medicine and Disease Modeling?

Shinya Yamanaka’s 2006 discovery of fibroblast “reprogramming” to embryonic stem cell-like iPSCs opened the possibility of personalized regenerative medicine for all organs. Nearly 10 years on in the “iPSC world”, biomedical researchers have grappled with the need to eliminate undifferentiated iPSCs that cause tumors, the requirement for complex guiding protocols to move iPSCs to specific lineages such as heart, brain, or liver, and the vexing observation that the final lineages lack the normal regenerative properties of these organs. Overlooked in the iPSC flurry is the fundamental work by Howard Green in cloning so-called “adult” stem cells of the epidermis and their phenomenal success in treating severe burn patients. Part of this oversight is due to the inability of the Green technology to capture stem cells of columnar epithelia, which include the gastrointestinal tract, liver, pancreas, kidney, and other organs central to human disease. Dr. Wa Xian, along with her co-workers and collaborators, has now cracked this problem and the emerging properties of her patient-specific, “ground state” stem cells are likely to initiate a sea-change in regenerative medicine. In particular, these stem cells can be rapidly cloned and expanded from endoscopic biopsies, display exquisitely precise lineage commitment identical to their origin, and possess intrinsic programs that enable complex differentiation. We will describe these properties, as well as how these stem cells can reveal fundamental features of chronic human diseases. Finally, we will present the adaption of this cloning technology to human cancers for personalized approaches to mitigating chemotherapy resistance.

Frank McKeon, Ph.D., is a Senior Investigator and Group Leader at the Genome Institute of Singapore, and founder of Multiclonal Therapeutics, a Connecticut startup company. Dr. McKeon is an internationally recognized authority and pioneer in the fields of cell biology, signal transduction, and p53-related genes. He has discovered fundamental mechanisms underlying T cell activation, chromosome

segregation, and stem cell regeneration. The p63 gene was discovered in his laboratory, as was the function of this gene in stem cells of the prostate, breast, and epidermis. He has demonstrated the value of p63 in the diagnosis of cancers of the prostate and breast and produced the monoclonal antibodies to p63 that are in world-wide use for cancer detection and stem cell analysis. Dr. McKeon obtained his B.S. in Biology at Pomona College and his Ph.D. in Biochemistry and Biophysics from the University of California, San Francisco. He was a Professor in Harvard University for many years before starting a laboratory with Dr. Wa Xian at the Genome Institute of Singapore and the Institute for Medical Biology in 2008. In 2012 he also joined the Jackson Laboratory for Genomic Medcine though has recently left to build Multiclonal Therapeutics along with his Singapore efforts. Multiclonal Therapeutics will focus on chronic inflammatory diseases of the airways and gastrointestinal tract, regenerative strategies for organ-specific diseases, and therapies directed at cancer stem cells and chemotherapy-resistant variants. He and Wa Xian were the first to demonstrate the remarkable potential of lung to regenerate and to clone the lung stem cell responsible for this process, and Dr. Xian’s discovery of methods to clone stem cells of columnar epithelia has enabled forays into multiple human diseases. They have three children and live in Connecticut and Singapore.

David Kaplan, Ph.D.

Stern Family Endowed Professor of Engineering;

Professor & Chair of the Department of Biomedical Engineering, Tufts University.

Biopolymer Engineering for Stem Cell-Based Regenerative Medicine

Materials found in Nature, such as silks and collagens, are very useful as biomaterials to help treat and repair problems in the human body. These proteins can be prepared in various formats, such as fibers, sponges and films, which in combination with cells including stem cells, can then be used to grow, treat and repair failing human tissues, develop new medical devices and provide important laboratory models to study new treatments for diseases.

David Kaplan, Ph.D., is the Stern Family Endowed Professor of Engineering at Tufts University. He is Professor and Chair of the Department of Biomedical Engineering and also holds faculty appointments in the School of Medicine, Department of Chemistry and the Department of Chemical and Biological Engineering. He received his Ph.D. from Syracuse University and the State University at Syracuse. Dr. Kaplan’s

research focus is on biopolymer engineering to understand structure-function relationships, with emphasis on studies related to self-assembly, biomaterials engineering and regenerative medicine. Since 2004 he has directed the NIH P41 Center on Tissue Engineering and has published over 600 peer reviewed papers. He is the editor-in-chief of ACS Biomaterials Science and Engineering and serves on many other editorial boards and programs for journals and universities. Discoveries made by Dr. Kaplan and his collaborators have launched four start-up companies based on the potential of silk as a biomaterial for medical applications and health care. These innovations have been featured on the front page of the New York Times, at TED 2011, on NPR and in The Economist. Dr. Kaplan has received numerous awards for his work including from the American Institute of Medical and Biological Engineering; the Society for Biomaterials, and the Tissue Engineering Society.

A B S T R A C T / B I O G R A P H Y 1 51 4 A B S T R A C T / B I O G R A P H Y

Carla Kim, Ph.D.

Associate Professor, Boston Children’s Hospital, Stem Cell Program;

Department of Genetics, Harvard Medical School.

Using Stem Cell Biology to Understand Lung Repair and Regeneration Mechanisms

Effective therapies are desperately needed for lung diseases, such as pulmonary fibrosis or emphysema, that have no cure and few treatment options. One hallmark shared by numerous lung diseases is extensive damage in multiple cell types, yet the precise way this damage occurs is poorly understood. Previous work from our laboratory has identified a stem cell population in the lung that has the capacity to repair the both the conducting airways and alveoli, where gas exchange takes place, known as the Bronchiolar Alveolar Stem Cell or BASC. Under healthy conditions the BASC is dormant. Upon injury, BASCs increase in number and contribute to regeneration of the damaged tissue. The signals that inform stem cells to repair damaged tissue are not known. We have recently discovered a new pathway, or set of molecules that work together, that controls how blood vessel cells communicate with lung stem cells to repair tissue damage. Further work to understand this pathway is underway, with the goal of facilitating the development of innovative targeted therapies for lung diseases.

Carla Kim, Ph.D., is an Associate Professor of Genetics and Pediatrics at Harvard School of Medicine, and a faculty member at Boston Children’s Hospital and the Dana Farber Harvard Cancer Center. She received her Ph.D. in Genetics from the University of Wisconsin, and conducted her postdoctoral studies at the Massachusetts Institute of Technology. The broad interest of Dr. Kim’s research program is to characterize

the biology of stem cells in normal lung tissue, and in lung disease. She was the first to isolate stem cells from the adult lung, and her work has made important connections between stem cell biology and lung biology that both advance our basic understanding of lung cell complexity and elucidate concepts relevant for human health. Numerous lung diseases such as cystic fibrosis or chronic obstructive pulmonary disease involve injured or depleted bronchiolar or alveolar epithelium. Bronchiolar and alveolar cells are also affected in adenocarcinoma, the most common form of lung cancer. Her long-term goal is to elucidate the role of stem cells in lung homeostasis as a prerequisite to the development of therapeutic strategies that can be used to prevent or attenuate lung disease. Dr. Kim has received numerous awards and honors including being named a March of Dimes Basil O’Connor Scholar and an American Cancer Society Scholar, and most recently received the Boston Children’s Hospital Research Award.

Symposium Organizers

Caroline Dealy, Ph.D. (Organizing Chair), Departments of Reconstructive Sciences, and Orthopaedic Surgery, Center for Regenerative Medicine and Skeletal Development, UConn Stem Cell Institute, University of Connecticut Health Center

Stormy Chamberlain, Ph.D., Department of Genetics and Genome Sciences, UConn Stem Cell Institute, University of Connecticut Health Center

Susan Froshauer, Ph.D., President and CEO, CURE — Connecticut United for Research Excellence

David Goldhamer, Ph.D., Department of Molecular and Cell Biology, UConn Stem Cell Institute, University of Connecticut

Diane Krause M.D., Ph.D., Departments of Cell Biology, Laboratory Medicine, and Pathology, Yale Stem Cell Center, Yale University

Janice Naegele, Ph.D., Departments of Biology, and Neuroscience and Behavior, Wesleyan University

In-Hyun Park, Ph.D., Department of Genetics, Yale Stem Cell Center, Yale University

Lawrence Rizzolo, Ph.D., Departments of Surgery, and Ophthalmology, Yale Stem Cell Center, Yale University

Milton Wallack, D.D.S., Connecticut Stem Cell Research Coalition; Connecticut Regenerative Medicine Research Advisory Committee

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