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JOSEPH MILETICH, MD, PHD

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Joseph Miletich, MD, PhD, is a former SVP, Research Sciences, at Merck. He has more than 20 years of combined experience at Merck and Amgen leading programs through Discovery Research, Preclinical Development and Translational Medicine/Early Development. At both companies, Dr. Miletich focused on building teams and investing in technologies, partnerships and acquisitions to enable capitalizing on new insights into biology regardless of therapeutic area or modality. Dr. Miletech currently serves as advisor and board director to several companies and firms. Most recently, Dr. Miletich was Merck Research Laboratories’ senior vice president of research sciences and is currently senior scientific advisor to Merck’s CEO, an advisor to Recursion, and a director to Flame Biosciences, among other roles. . Prior to his last 8+  years at Merck Research Laboratories, Dr. Miletich served as senior vice president of research and development at Amgen. 

Dr. Miletich received his M.D. and Ph.D. degrees from Washington University in St. Louis and pursued clinical training in internal medicine at the University of California in San Francisco. Subsequently he joined the faculty at Washington University, pursued subspecialty training in clinical hematology and in laboratory medicine, and developed a leading research effort in hemostasis and thrombosis. Over a 17 year career, he rose through the ranks to become Professor of Medicine and Pathology and Head of the Division of Laboratory Medicine at Washington University, in which capacity he was responsible for the combined laboratories of the Barnes-Jewish Hospital system, one of the nation’s largest clinical laboratories.

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Nicholas E. Johnson, MD, MSCI, FAAN

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Nicholas E. Johnson, MD, MSCI, FAAN is an associate professor, division chief of neuromuscular, and vice chair of research in the department of neurology at Virginia Commonwealth University with a focus in inherited neuromuscular disorders. He received his undergraduate degree in molecular and cellular biology and psychology at the University of Arizona. He then obtained his medical degree at the University of Arizona. He completed his neurology residency and combined fellowship in neuromuscular medicine and experimental therapeutics at the University of Rochester. His laboratory is focused on identifying the pathogenesis of myotonic dystrophy, the limb girdle muscular dystrophies, and facioscapulohumeral muscular dystrophy and identifying appropriate clinical endpoints for these conditions. Johnson conducts therapeutic trials in many other inherited nerve and muscle disorders.

Research Expertise

Translational laboratory focused on identifying genetic modifiers across muscular dystrophies, and developing clinical endpoints and patient reported outcomes for myotonic dystrophy, facioscapulohumeral muscular dystrophy, and the limb girdle muscular dystrophy. The laboratory uses cellular models to identify changes in RNA splicing in myotonic dystrophy.

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John W. Day, MD, PhD

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Since 2011 John has been Professor of Neurology, Pediatrics (Genetics) and Pathology at Stanford University, where he is Director of the Division of Neuromuscular Medicine. He received his MD from the University of Minnesota, and PhD in Neuroscience from Albert Einstein College of Medicine, where he studied neuronal electrophysiology, synaptic physiology and plasticity.  After completing neurology and neuromuscular training at UCSF, he was Professor of Neurology, Pediatrics and Genetics at the University of Minnesota, where he founded and directed the Paul and Sheila Wellstone Muscular Dystrophy Center. 

John has investigated the genetic causes and multisystemic effects of neuromuscular disorders and has more than 35 years of experience designing and directing clinical trials of novel therapeutics.  He spearheaded clinical research on spinal muscular atrophy (SMA) at Stanford, which resulted in the approval of antisense and AAV-gene replacement treatments, and led to nationwide newborn screening for SMA and institution of neonatal genetic treatment.

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Tina Duong PT, PHD

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Tina Duong is the Director of clinical outcomes research and development at Stanford University Neuromuscular division. Clinically, she is a physical therapist with 20 years of clinical experience in evaluating, treating and validating clinical outcomes for patients with neuromuscular disease. 

Her clinical and research interests are in developing, refining and understanding impacts of muscle imbalances and pathology on function and performance. Her work has contributed to improved understanding and characterization of disease phenotypes impacting clinical trial design and care management.

Currently, her research focuses on the effect of exercise and physical activity on human movement and disease physiology. She hopes to integrate technology and digital biomarkers as a complementary tool to performance based measures used in trials, in the clinic and remotely. She enjoys working on collaborative diverse inter-disciplinary teams, mentorship and creating patient focused solutions to improve the quality of life for individuals with neuromuscular disease.

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Peter Jackson, PhD

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Peter Jackson is a Professor in the Baxter Laboratory for Stem Cell Biology, Department of Microbiology & Immunology, at the Stanford University School of Medicine. Dr. Jackson returned to Stanford in 2013 after eight years as a Staff Scientist and Director at Genentech Inc. in S. San Francisco, California. At Genentech, he helped define and implement the development of therapeutics for cancer pathways including cell cycle checkpoints, stress pathways, and tumor metabolism. Before joining Genentech in 2005, he spent 10 years on the faculty at Stanford University School of Medicine. His laboratory has been involved in studies of cell cycle biochemistry, regulation of the cancer by kinases and phosphatases, the ubiquitin-proteasome system, and the discovery of a new physiological class of competitive (“pseudosubstrate”) E3 ubiquitin ligase inhibitors, exemplified by the APC/C regulator Emi1.

Since 2005, his lab has focused on signaling through the primary cilium, using proteomic approaches to define regulatory networks and new disease genes. More broadly, the lab has connected many proteins defective in human diseases and cancer to new complexes and pathways, with a view to discovering molecular signatures for diagnostics and therapeutic development. His research has earned him numerous visiting lectureships and honors, including awards from the Baxter Foundation, Howard Hughes Medical Institute, the American Cancer Society, the Pluto Society, and being a Stanford Hume Faculty Scholar and a Kirsch Scholar, and elected a Fellow of the American Association for the Advancement of Science (in 2008).

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Stanford School of Medicine

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Christopher Kirk, PhD

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Christopher Kirk, PhD is an independent director, scientific and strategic advisor to Juvena Therapeutics. He is the  co-founder, President and Chief Scientific Officer of Kezar Life Sciences, a publicly traded company developing first-in-class, small molecule therapies designed to inhibit multiple disease-driving cellular pathways by targeting key intracellular proteins. Dr. Kirk is an immunologist and tumor biologist by training. Prior to founding Kezar, he was the Vice President of Research at Onyx Pharmaceuticals where he played a key leadership role in the discovery and development of 2 proteasome inhibitors, including carfilzomib (KYPROLIS™), currently approved for the treatment of multiple myeloma. Dr. Kirk has authored multiple publications in several select journals including Cell, Nature Medicine, and Nature Reviews and is listed as an inventor on over 40 patents. He sits on the scientific advisory board at C4 Therapeutics, Avidity Biosciences, Rain Therapeutics, the Institute of Applied Clinical Science at the M.D. Anderson Cancer Center as well as the leadership council of the Life Sciences Institute of the University of Michigan.

Dr. Kirk received his B.S. in Biochemistry from the University of California, Davis, and his Ph.D. in Cellular and Molecular Biology from the University of Michigan.

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David Schaffer, PhD

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David Schaffer is a Professor of Chemical and Biomolecular Engineering, Bioengineering, and Neuroscience at UC Berkeley, where he also serves as the Director of the Berkeley Stem Cell Center. In addition, he is Co-Founder, Acting CSO, and Co-Chairman of 4D Molecular Therapeutics.

At UC Berkeley, Dr. Schaffer applies engineering principles to enhance stem cell and gene therapy approaches for neuroregeneration, work that includes novel approaches for molecular engineering and evolution of new viral vectors as well as new technologies to investigate and control stem cell fate decisions.

Professor Schaffer served as Dr. Yousef’s co-thesis advisor (2008-2013).

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UC Berkeley

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Irina Conboy, PhD

Irina Conboy is a Professor at the University of California, Berkeley in the Department of Bioengineering. Her discovery of the rejuvenating effects of young blood through parabiosis in a seminal paper published in Nature in 2005 paved the way for a thriving field of rejuvenation biology. The Conboy lab currently focuses on broad rejuvenation of tissue maintenance and repair, stem cell niche engineering, elucidating the mechanisms underlying muscle stem cell aging, directed organogenesis, and making CRISPR a therapeutic reality.

Professor Conboy served as Dr. Yousef’s co-thesis advisor (2008-2013).

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UC Berkeley

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Joe Wu, MD, PhD

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Joseph C. Wu, MD, PhD, is Director of the Stanford Cardiovascular Institute and Simon H. Stertzer Endowed Professor in the Departments of Medicine (Cardiology) and Radiology at Stanford School of Medicine. Dr. Wu received his medical degree from Yale. He completed his medicine internship, residency, and cardiology fellowship (STAR program) at UCLA. He obtained his PhD in the Department of Molecular & Medical Pharmacology with Dr. Sam Sanjiv Gambhir. His clinical interests include adult congenital heart disease and cardiovascular imaging.

His lab works on biological mechanisms of patient-specific and disease-specific induced pluripotent stem cells (iPSCs) with a focus on cardiovascular disease mechanisms and the implementation of precision medicine for prevention and treatment of cardiovascular patients. His lab uses a combination of genomics, stem cells, cellular & molecular biology, physiological testing, and molecular imaging technologies. Dr. Wu has published >400 manuscripts with H-index of 101 on Google scholar and recognition as top 1% of highly cited researchers in Web of Science (2018, 2019 & 2020). Among his trainees, >30 of them are principal investigators in the US or abroad.

Dr. Wu has received numerous awards, including National Institutes of Health (NIH) Director’s New Innovator Award, NIH Roadmap Transformative Award, American Heart Association (AHA) Innovative Research Award, Presidential Early Career Award for Scientists and Engineers (PECASE) given out by President Obama, AHA Established Investigator Award, Burroughs Wellcome Foundation Innovation in Regulatory Science Award, AHA Merit Award, and AHA Distinguished Scientist Award. Dr. Wu serves on the Scientific Advisory Board for the Keystone Symposia, FDA Cellular, Tissue, and Gene Therapies Advisory Committee, AHA National Board of Directors, and Chair of the AHA National Research Committee.

Dr. Wu is an elected member of American Society of Clinical Investigators (ASCI), Association of University Cardiologists (AUC), American Institute for Medical and Biological Engineering (AIMBE), American Association of Physicians (AAP), American Association for the Advancement of Science (AAAS), and National Academy of Medicine (NAM).

In addition, he has been scientific co-founder of several startups.

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