Management: The Mountaineers

Josh Mandel-Brehm
Josh Mandel-Brehm
Josh Mandel-Brehm is President & Chief Executive Officer of CAMP4 Therapeutics and holds a dual appointment as entrepreneur partner with Polaris Partners.
Mr. Mandel-Brehm previously held key business development and operations leadership roles at leading biotech companies. Most recently he served as part of the Business Development group at Biogen, where he led multiple strategic activities and corresponding transactions, which included expanding Biogen’s non-malignant hematology franchise and overseeing seminal investments to enter the ophthalmology field. Mr. Mandel-Brehm also played an integral role advancing Biogen’s gene therapy strategy, executing a series of external collaborations. Prior to Biogen, Mr. Mandel-Brehm held several roles of increasing responsibility at Genzyme as part of the business development group for the company’s rare disease business unit.
Mr. Mandel-Brehm earned a BA in Biology from Washington University in St. Louis and holds an MBA from the University of Michigan.

David Bumcrot, PhD
David Bumcrot, PhD
David Bumcrot, PhD, is Chief Scientific Officer and Senior Vice President of Research, where he oversees the Biology, Data Science and Discovery teams.
Prior to joining CAMP4, Dr. Bumcrot led groups at innovative biotech companies advancing novel technologies towards clinical development. He was one of the first employees at Alnylam pharmaceuticals where he spent ten years working to make RNA interference-based drugs a reality for multiple indications, including key contributions to a first-in-class systemically administered siRNA therapeutic tested in an oncology clinical trial. Dr. Bumcrot then moved on to Editas Medicine where his team established the company’s initial therapeutic programs utilizing groundbreaking CRISPR technology.
Dr. Bumcrot received his Bachelor of Science degree from Cornell University and a PhD in molecular biology from the University of Pennsylvania. He completed a post-doctoral fellowship at Harvard University.

Kelly Gold
Kelly Gold
Ms. Gold is the Chief Financial Officer at CAMP4 Therapeutics. She is responsible for guiding and executing the company’s strategy for establishing innovative strategic and technology-based partnerships that allow CAMP4 to realize the full value of the regRNA Actuating Platform. In addition, Ms. Gold leads all corporate finance and accounting, investor relations and strategic financial planning activities at CAMP4.
Ms. Gold joined CAMP4 in 2017 and has held roles of increasing responsibility leading the Business Development and Finance functions, most recently as Chief Business Officer and SVP, Finance. Prior to joining CAMP4, Ms. Gold held various roles in corporate finance and business planning at Biogen, where she provided financial leadership for the company’s late stage and marketed rare disease programs and developed long term strategic financial trajectories for the R&D organization. Prior to Biogen, Ms. Gold worked in the healthcare investment banking group at Deutsche Bank, advising on M&A, public and private equity offerings, leveraged finance transactions, and joint ventures to clients in the US, Latin America and Europe. She also worked in Deutsche Bank’s Latin American Investment Banking group in Mexico, executing dual-listed IPOs in the areas of financial services and oil and gas. Ms. Gold began her career as a mechanical engineer designing biocontainment systems for Biosafety Level 4 research facilities operated by the CDC, the NIH, and the US Army.
Ms. Gold holds Bachelor’s degrees in Biochemistry and Mechanical Engineering from Queen’s University in Ontario, as well as an MBA from the MIT Sloan School of Management.

Michelle Gates
Michelle Gates
Michelle Gates is Senior Vice President & Chief People Officer at CAMP4 Therapeutics. Ms. Gates is responsible for developing the people strategy aligned to the mission and vision of CAMP4. She also leads the HR function, providing key insights and expertise in building and sustaining CAMP4’s fast-growing team and culture.
Prior to joining CAMP4, Ms. Gates was Vice President of HR at Akamai Technologies. During Ms. Gates’ 16 year career at Akamai, she played a pivotal role in contributing to massive growth and global expansion of the organization, including several acquisitions. Ms. Gates was critical to the early development and continual transformation of the HR function at Akamai; where she led many company-wide programs, built centers of excellence, and hired and developed top talent. In her most recent role, as HR leader for Akamai’s Platform organization, and Enterprise Security division, Ms. Gates was instrumental in developing the enterprise security strategy, and building out the team globally. Ms. Gates is passionate about building and developing diverse and inclusive teams; and driving excellence through collaboration and ‘enterprise thinking’. Prior to Akamai, Ms. Gates held HR leadership roles at British Telecom, and Teradyne.
Ms. Gates holds a Bachelor of Arts in English and Spanish from Providence College, and has completed courses through Wharton’s Executive Education Program.
Ann Barbier, M.D., Ph.D.
Ann Barbier, M.D., Ph.D.
Prior to joining CAMP4, Dr. Barbier was Chief Medical Officer of Translate Bio, leading the development of an inhaled mRNA for the treatment of cystic fibrosis. Her prior experience includes positions at Agios, Shire, EnVivo, Johnson & Johnson and Aventis. During her career, Dr. Barbier has made significant contributions to several approved products such as Elaprase® (Hunter syndrome), Aubagio® (multiple sclerosis), Firazyr® (hereditary angioedema) and Pyrukynd® (pyruvate kinase deficiency) and has led several investigational new drug applications for new chemical entities. She is a member of the Board of Directors of Pieris Pharmaceuticals. Additionally, she has co-authored more than 50 peer-reviewed scientific articles, book chapters and invited reviews. Dr. Barbier received her M.D. and a Ph.D. in pharmacology from the University of Gent, Belgium, and a Master of Science from the Free University of Brussels, Belgium. She pursued a postdoctoral fellowship at the University of Tennessee in Memphis.
Caleb Moore
Caleb Moore
Caleb Moore is Vice President, Portfolio Strategy & Business Operations at CAMP4 Therapeutics. He is responsible for guiding CAMP4’s disease selection and program strategy based on biological rationale and downstream development and commercial considerations. He also oversees CAMP4’s operational activities, including laboratory operations, IT, and space planning.
Prior to joining CAMP4, Mr. Moore was Senior Director of Corporate Development at Acceleron Pharma, where he helped translate the company’s expertise in TGFβ biology into a focused therapeutic area strategy. He was instrumental in establishing Pulmonary as a pillar of Acceleron’s strategy based on the disease-modifying potential of its lead program in pulmonary arterial hypertension (PAH). Prior to Acceleron, Mr. Moore worked in Global New Product Planning at Cubist Pharmaceuticals, defining early commercial strategy for internal programs and external opportunities. He also held positions in Business Development & Strategy at Genzyme, steering multiple transactions that contributed to the growth of the rare disease and cardio-metabolic/renal businesses. Mr. Moore began his life sciences career at a boutique consulting and market research firm.
Mr. Moore earned a BA in Genetics, Cell & Developmental Biology from Dartmouth College and holds an MBA from the Tuck School of Business at Dartmouth.

Alfica Sehgal, PhD
Alfica Sehgal, PhD
Alfica Sehgal comes to us from Alnylam Pharmaceuticals, where she held multiple roles. Dr. Sehgal was the program leader for Alpha-1 antitrypsin deficiency related liver disease, driving the program from bench to Phase 1, as well as the program research lead for the fitusiran program for the treatment of hemophilia and rare bleeding disorders. She was also a member of the platform team working towards understanding delivery of siRNA in naked, conjugated and formulated forms to hepatocytes and extra-hepatic tissues. Dr. Sehgal was also part of the next wave team responsible for generating and triaging new pipeline programs.
Dr. Sehgal received her post-doctoral training at Johns Hopkins University and Yale University, studying lipid uptake, cholesterol and oxygen homeostasis in parasites, mammalian cells and mice. She received her doctoral degree from the Tata Institute of Fundamental Research (India) in naturally acquired immunity against malarial parasites. She has published more than 30 articles and book chapters in peer-reviewed journals and has authored 17 patents and PCT applications.

Yuting Liu, PhD
Yuting Liu, PhD
Yuting Liu joined CAMP4 Therapeutics in 2016 as the first computational biologist. Since then, he has been responsible for analyzing various types of next-generation sequencing data and supporting pipeline programs.
He received his PhD in Computational Biology from Shanghai Institute for Biology Science, Chinese Academy of Science, where he studied the post-transcriptional regulation in circadian clock and hibernation. His postdoctoral fellowship was conducted with Prof. Yi Zhang at Harvard Medical School, where he studied the epigenetic regulation in development and differentiation. He joined CAMP4 from Agios Pharmaceuticals where he studied the DNA methylation changes in the AML patients treated with IDH1 mutation inhibitor. He also worked on mining cancer vulnerability in TCGA, CCLE and Achilles screening database.
Dr. Liu has co-authored 17 peer-reviewed articles in the field of epigenomics and post-transcriptional gene regulation.

Board of Directors: Our Guides
Steven Holtzman
Steven Holtzman
Steve Holtzman is currently a Strategic Advisor for Decibel Therapeutics, where he formerly served as president and chief executive officer. Prior to Decibel, he served as Executive Vice President, Corporate Development at Biogen, Inc., where he led eight new drug approvals. Previously, Mr. Holtzman was the founder, chief executive officer and board chair of Infinity Pharmaceuticals, Inc., a cancer drug discovery and development company. He was also an early leader and the chief business officer of Millennium Pharmaceuticals (now Takeda Oncology), a pioneer in large-scale genetics and genomics, and was a founder, board member and executive vice president of DNX Corporation, the first transgenic animal company. He is chairman of the boards of Qihan Biotech and CAMP4, a board member of Molecular Partners, a strategic business advisor to Humatics and Compugen, a member of the board of the Berklee College of Music, and a senior fellow at the Belfer Center for Science and International Affairs at the Harvard Kennedy School. He was appointed by President Clinton to the National Bioethics Advisory Commission and also served as a member and vice chair of the board of the Hastings Center for the Life Sciences. He obtained his undergraduate degree in philosophy from Michigan State University and his graduate degree in philosophy from Oxford University, which he attended as a Rhodes Scholar.
Josh Mandel-Brehm
Josh Mandel-Brehm
Josh Mandel-Brehm is President & Chief Executive Officer of CAMP4 Therapeutics and holds a dual appointment as entrepreneur partner with Polaris Partners.
Mr. Mandel-Brehm previously held key business development and operations leadership roles at leading biotech companies. Most recently he served as part of the Business Development group at Biogen, where he led multiple strategic activities and corresponding transactions, which included expanding Biogen’s non-malignant hematology franchise and overseeing seminal investments to enter the ophthalmology field. Mr. Mandel-Brehm also played an integral role advancing Biogen’s gene therapy strategy, executing a series of external collaborations. Prior to Biogen, Mr. Mandel-Brehm held several roles of increasing responsibility at Genzyme as part of the business development group for the company’s rare disease business unit.
Mr. Mandel-Brehm earned a BA in Biology from Washington University in St. Louis and holds an MBA from the University of Michigan.
Jorge Conde
Jorge Conde
Jorge Conde is a General Partner at Andreessen Horowitz where he leads investments at the cross section of biology, computer science and engineering.
Prior to joining a16z, Mr. Conde served as Chief Strategy Officer for Syros (NASDAQ: SYRS), which is advancing a new wave of medicines that control expression of disease-driving genes to treat cancer and other diseases. He previously served as the company’s Chief Financial Officer and Chief Product Officer, leading the platform strategy for Syros’ novel gene regulation technology. Mr. Conde also cofounded Knome, a human genome interpretation company acquired by Tute Genomics in 2015. Earlier in his career, Mr. Conde worked in marketing and operations at MedImmune and as a biotechnology investment banker at Morgan Stanley.
Mr. Conde holds an MBA from Harvard Business School, an MS from the Harvard-MIT Division of Health Sciences and Technology, and a BA in Biology from Johns Hopkins University.
Mr. Condewas named one of the top 35 young innovators in the world by the MIT Technology Review and is a Henry Crown Fellow of the Aspen Institute and a member of the Aspen Global Leadership Network. He previously served on the board of the Museum of Science, Boston.
Shaan Gandhi, MD, DPhil
Shaan Gandhi, MD, DPhil
Michael Higgins
Michael Higgins
Michael Higgins currently serves as Chief Executive Officer and Executive Chairman of Kindex Pharmaceuticals. An experienced biotechnology executive, Mr. Higgins has held leadership roles in finance, operations and business development with both large multi-national organizations and small entrepreneurial firms. Previously, he served as Chief Operating Officer and Chief Financial Officer (CFO) at Ironwood Pharmaceuticals from 2003 through 2014. In 2014, Mr. Higgins was named Boston Business Journal’s CFO of the Year. Mr. Higgins worked at Genzyme Corporation from 1997 through 2003 in a variety of leadership roles, including Vice President of Corporate Finance and Vice President of Business Development. While at Genzyme, he was involved with multiple business units, including cell therapy, gene therapy and orphan diseases. Mr. Higgins also served as Chief Financial Officer at Procept, Inc. from 1992 to 1997. He began his pharmaceutical career as a sales representative for Schering-Plough Corporation.
Mr. Higgins currently serves as Chairman of Voyager Therapeutics’ Board of Directors. He also serves on the boards of several additional public and privately held companies, including Genocea Biosciences, Inc., Pulmatrix, Inc., CAMP4, Sea Pharmaceuticals and Private Equity Access Fund, II.
Mr. Higgins holds a Bachelor of Science degree from Cornell University and a Master of Business Administration from the Amos Tuck School of Business at Dartmouth College.
Amir Nashat, ScD
Amir Nashat, ScD
Amir Nashat is a managing partner in Polaris’ Boston office. He joined Polaris in 2002 and focuses on investments in healthcare.
Dr. Nashat currently represents Polaris as a Director of AgBiome, CAMP4, Dewpoint Therapeutics, Fate Therapeutics (NASDAQ: FATE), Freenome, Metacrine Therapeutics, Morphic Therapeutic (NASDAQ: MORF), Scholar Rock (NASDAQ: SRRK), Selecta Biosciences (NASDAQ: SELB), Syros Pharmaceuticals (NASDAQ: SYRS) and TARIS Biomedical.
Additionally, Dr. Nashat has served as a director of Adnexus Therapeutics (Bristol Myers Squibb), Athenix Corporation (Bayer), aTyr Pharmaceuticals (NASDAQ: LIFE), Avila Therapeutics (Celgene), Living Proof (Unilever), Pervasis Therapeutics (Shire Pharmaceuticals), Promedior Pharmaceuticals, Receptos (Celgene), and Sun Catalytix (Lockheed Martin). At Dewpoint, Living Proof, Sun Catalytix, Olivo Labs, and Jnana, Dr. Nashat has served as the company’s initial CEO. Dr. Nashat also serves on the Partners Innovation Fund, the Investment Advisory Committee for The Engine at MIT, and helped launch the MIT Sandbox Innovation Fund as its active president.
He previously served on the board of the New England Venture Capital Association. He has been named to the Forbes Midas List of “Top 100 Venture Capitalists.”
Prior to joining Polaris, Dr. Nashat completed his ScD as a Hertz Fellow in Chemical Engineering at the Massachusetts Institute of Technology with a minor in Biology under the guidance of Dr. Robert Langer. Dr. Nashat also earned both his MS and BS in Materials Science and Mechanical Engineering at the University of California, Berkeley.
Paula Ragan, PhD
Paula Ragan, PhD
Dr. Ragan is a founder, as well as the Chief Executive Officer & President of X4. She has more than 19 years of experience building companies in the biotechnology industry. Most recently, Dr. Ragan consulted as Chief Business Officer at Lysosomal Therapeutics Inc (LTI), an Atlas Venture portfolio company, where she led the company’s business development activities. Prior to LTI, Dr. Ragan held leadership roles in corporate development and operations at Genzyme/Sanofi where she led strategic partnering efforts for Genzyme’s Rare Disease business and headed the supply chain planning for Genzyme’s flagship commercial products. Other professional roles include business roles at Hydra Biosciences, Oscient Corporation and Celera Corporation. Dr. Ragan received her BS in Mechanical Engineering from Tufts University and her PhD from MIT. She completed post-doctoral studies at Harvard Medical School.
Andy Schwab
Andy Schwab
Ravi I. Thadhani, MD, MPH
Dean of the Faculty at MGB for Harvard Medical School
Ravi I. Thadhani, MD, MPH
Dean of the Faculty at MGB for Harvard Medical School
As the Chief Academic Officer of Mass General Brigham (MGB) and Dean of the Faculty at MGB for Harvard Medical School, Dr. Thadhani manages a ~ $2B research enterprise. He oversees several key system-wide departments at MGB, including Human Subjects Affairs, the Clinical Trials Office, Research Management, the MGB Biobank, and Graduate Medical Education across MGB. Dr. Thadhani managed a research laboratory for ~25 years, with a focus on kidney disease and developing diagnostics and therapeutics for women with complications in pregnancy. He has published over 300 manuscripts and has been inducted into several honor societies. He is a standing member of FDA’s Cardio-Renal Division Advisory Panel. Dr. Thadhani has received several distinguished awards including the Harold Amos Faculty Diversity Award from Harvard Medical School, the Alumni Award of Merit from the Harvard T.H. Chan School of Public Health, and the John P. Peters Award from the American Society of Nephrology. Dr. Thadhani received a bachelor’s degree from the University of Notre Dame, a medical degree from the University of Pennsylvania, a master’s in public health from the Harvard T.H. Chan School of Public Health, and an online business certificate from the Stanford Graduate School of Business.
Richard Young, PhD
Professor of Biology, Massachusetts Institute of Technology
Richard Young, PhD
Professor of Biology, Massachusetts Institute of Technology
Richard Young studies gene regulatory circuitry in health and disease. His research accomplishments range from the development of genome-wide analysis technologies to discovery of the core regulatory circuitry of human embryonic stem cells. Dr. Young received his PhD in Molecular Biophysics and Biochemistry at Yale University, conducted postdoctoral research at Stanford University and joined Whitehead Institute and MIT in 1984. He has served as an advisor to the National Institutes of Health, the World Health Organization, the Vatican and numerous scientific societies and journals. Dr. Young has founded and advised companies in the biotechnology and pharmaceutical industry and is currently a member of the Board of Directors of CAMP4, Syros Pharmaceuticals and Omega Therapeutics. His honors include Membership in the National Academy of Sciences, the Chiron Corporation Biotechnology Research Award, Yale’s Wilbur Cross Medal, and in 2006 Scientific American recognized him as one of the top 50 leaders in science, technology and business.

Founders: Our Cartographers
Richard Young, PhD
Professor of Biology, Massachusetts Institute of Technology
Richard Young, PhD
Professor of Biology, Massachusetts Institute of Technology
Richard Young studies gene regulatory circuitry in health and disease. His research accomplishments range from the development of genome-wide analysis technologies to discovery of the core regulatory circuitry of human embryonic stem cells. Dr. Young received his PhD in Molecular Biophysics and Biochemistry at Yale University, conducted postdoctoral research at Stanford University and joined Whitehead Institute and MIT in 1984. He has served as an advisor to the National Institutes of Health, the World Health Organization, the Vatican and numerous scientific societies and journals. Dr. Young has founded and advised companies in the biotechnology and pharmaceutical industry and is currently a member of the Board of Directors of CAMP4, Syros Pharmaceuticals and Omega Therapeutics. His honors include Membership in the National Academy of Sciences, the Chiron Corporation Biotechnology Research Award, Yale’s Wilbur Cross Medal, and in 2006 Scientific American recognized him as one of the top 50 leaders in science, technology and business.
Leonard Zon, MD
Leonard Zon, MD
Grousbeck Professor of Pediatrics, Harvard Medical School
Investigator, Howard Hughes Medical Institute
Dr. Zon is the Grousbeck Professor of Pediatric Medicine at Harvard Medical School, Investigator at Howard Hughes Medical Institute, and Director of the Stem Cell Program at Boston Children’s Hospital. Dr. Zon received his BS in chemistry and natural sciences from Muhlenberg College (1979) and his MD from Jefferson Medical College (1983). He subsequently did an internal medicine residency at New England Deaconess Hospital (1986) and a fellowship in medical oncology at Dana-Farber Cancer Institute (1989). His postdoctoral research was in Stuart Orkin’s laboratory (1990). Dr. Zon is internationally-recognized for his pioneering work in stem cell biology and cancer genetics. He has been the preeminent figure in establishing zebrafish as an invaluable genetic model for the study of the blood and hematopoietic development. He is founder and former president of the International Society for Stem Cell Research and chair of the Executive Committee of the Harvard Stem Cell Institute. In 2005, he completed a term as President of the American Society for Clinical Investigation. That same year, Dr. Zon was elected to the Institute of Medicine of the National Academies. In 2008, Dr. Zon was elected to the American Academy of Arts & Sciences. In 2010, Dr. Zon was awarded the E. Donnall Thomas Lecture and Prize from American Society of Hematology. In 2013, Dr. Zon received the ISEH Donald Metcalf Lecture Award. Other recent awards include the 2014 Boston Children’s Hospital Post-Doctoral Association Mentoring Award and the National Cancer Institute’s Alfred G. Knudson Award (2015).
Scientific Advisory Board: Our Navigators
Michael Hayden, CM OB CMB ChB PhD FRCP(C) FRSC
Michael Hayden, CM OB CMB ChB PhD FRCP(C) FRSC
Director of the Translational Laboratory in Genetic Medicine
Dr. Michael Hayden is a Killam Professor at the University of British Columbia. From 2012 to 2017, he served as the President of Global R&D and Chief Scientific Officer at Teva Pharmaceutical Industries Ltd. Dr. Hayden built and transformed research and development at Teva to become a highly productive research engine and led the approval of 35 new products.
Dr. Hayden has founded five biotechnology companies and has been the recipient of numerous prestigious honors and awards including being inducted into the Canadian Medical Hall of Fame, receiving the July 2012 Diamond Jubilee Medal, on behalf of HRH Queen Elisabeth II and the Margolese National Brain Disorder Prize, awarded to Canadians who have made outstanding contributions to the treatment, amelioration, or cure of brain diseases. He’s also received the Canada Gairdner Wightman award for his outstanding leadership in medicine and medical science as a physician-scientist. Dr. Hayden was awarded the Order of Canada, the Order of British Columbia, named Canada’s Health Researcher of the Year by Canadian Institutes of Health Research, and has received the Prix Galien for his contribution to Canadian pharmaceutical research. Most recently, Dr. Hayden was named one of the 50 Canadians born in the 20th century who have changed the world.
Karen Adelman, PhD
Karen Adelman, PhD
Dr. Adelman earned her PhD in 1999 from Universite de Paris VI, working at the Institut Pasteur under a fellowship from the National Science Foundation. She then joined the laboratory of John Lis, PhD, at Cornell University for her post-doctoral training. In 2005, she established her own laboratory at the National Institute of Environmental Health Sciences (NIH), and was promoted to Senior Investigator in 2011. In 2016, she joined the Harvard Medical School faculty as a Professor in the Department of Biological Chemistry and Molecular Pharmacology.
Jason Buenrostro, PhD
Jason Buenrostro, PhD
Jason Buenrostro is an Assistant Professor at Harvard University in the department of Stem Cell and Regenerative Biology. At Harvard, Dr. Buenrostro is developing new approaches for understanding gene regulation across cell fate decisions. Dr. Buenrostro earned a B.S. in general engineering and a B.S. in biology at Santa Clara University. He did his doctoral work at the Stanford University Department of Genetics with William Greenleaf and Howard Chang. At Stanford, he developed new high-throughput genomics tools including ATAC-seq and single-cell ATAC-seq.
Wolfram Goessling, MD, PhD
Wolfram Goessling, MD, PhD
Robert H. Ebert Associate Professor of Medicine, Harvard Medical School
Advisory Dean, Irving M. London Society
Associate Physician, Department of Medicine, Brigham and Women’s Hospital
Dr. Wolfram Goessling is a physician-scientist with an active laboratory within the Division of Genetics at Brigham and Women’s Hospital. He is board certified in both oncology and gastroenterology. He is the Co-Director of the Harvard-MIT Division of Health Sciences and Technology and Advisory Dean of the Irving M. London Society.
His clinical practice focuses on the treatment of patients with liver disease and gastrointestinal cancers. His laboratory seeks to understand the signals that indicate organ injury and regulate regenerative and malignant growth using vertebrate and invitro models to elucidate the mechanisms of liver development, regeneration and cancer.
Rudolf Jaenisch, MD
Rudolf Jaenisch, MD
Professor of Biology, Massachusetts Institute of Technology
Dr. Rudolf Jaenisch’s laboratory’s expertise is in epigenetics, reprogramming and stem cells. He began his research career as a pioneer making transgenic mice, some of which have produced important advances in understanding cancer, neurological and connective tissue diseases, and developmental abnormalities. These methods have been used to explore basic questions such as the role of DNA modification, genomic imprinting, X chromosome inactivation, nuclear cloning, and, most recently, the nature of stem cells. Dr. Jaenisch’s laboratory is known for its expertise in cloning mice and in studying the many factors that contribute to the success and failure of that process.
Dr. Jaenisch and his colleagues have gained important insights into therapeutic cloning and have rescued mice having a genetic defect through therapeutic cloning and gene therapy. In addition, using mice as a model and a technique called “altered nuclear transfer,” they have demonstrated that it is possible to procure embryonic stem cells without harming a viable embryo. More recently the lab has demonstrated that somatic cells can be reprogrammed in vitro to pluripotent ES-like cells and that these cells are suitable to correct both genetic and induced defects in mice by transplantation therapy. Using this technique for turning skin cells into stem cells, the lab has been able to cure mice of sickle cell anemia — the first direct proof that these easily obtained cells can reverse an inherited disease.
Dr. Jaenisch’s group comprises a team of scientists who are experts in deriving and manipulating iPS cells, and generating and differentiating neurons from them. Their interest in neurodegenerative diseases such as Parkinson’s, Alzheimers, and Synucleinopathy has also created powerful synergies with other laboratories, sharing their expertise to understand and solve these devastating diseases. Dr. Jaenisch has mentored more than 30 former predoctoral fellows and over 60 postdoctoral researchers, including current full professors at Harvard, Stanford and UCLA.
Dr. Jaenisch earned his MD from the University of Munich, Germany, and completed doctoral studies at the Max Planck Institute of Biochemistry in Munich, and Princeton University.
Benjamin Neale, PhD
Benjamin Neale, PhD
Associate Professor, Medicine, Harvard Medical School
Institute Member, Broad Institute
Dr. Benjamin Neale is an associate professor in the Analytic and Translational Genetics Unit at Massachusetts General Hospital (MGH), associate professor in medicine at Harvard Medical School (HMS), and an institute member at the Broad Institute. Dr. Neale is strongly committed to gaining insights into the genetics of common, complex human diseases. Dr. Neale and Mark Daly, both of whom are associated with the Broad Institute and MGH, lead the ADHD Initiative, a collaborative effort that focuses on genomic studies of attention deficit hyperactivity disorder (ADHD).
Dr. Neale’s research and training have focused heavily on statistical methodology. He has analyzed genetic data from large-scale studies of patients with ADHD, autism, age-related macular degeneration, type 2 diabetes, and metabolic disorders. Neale also analyzed data from the first ADHD genome-wide association study (GWAS) meta-analysis, which combined the results of four studies to boost statistical power. Neale contributed to the development of software tools such as PLINK, one of the most frequently used packages for GWAS analysis. In addition to his roles at both the Broad Institute and MGH, Dr. Neale is the head of the ADHD psychiatric genetics GWAS analysis committee and an active member of the broader Psychiatric GWAS Consortium analysis committee, which is charged with analyzing all psychiatric data from these large-scale genome-wide association studies. Dr. Neale also led the design of the exome chip, a genotyping array that captures rare coding variation in a cost-effective manner. To date, over 1.5 million exome chips have been sold.
Dr. Neale studied at the University of Chicago and Virginia Commonwealth University, earning a B.Sc. in genetics. He went on to earn his PhD in human genetics from King’s College in London, UK. Neale completed his postdoctoral training in Daly’s laboratory at MGH. In addition to many local research collaborations, he also serves as advisor and analyst to international genetic research consortia on psychiatric diseases.
Jay Shendure, MD, PhD
Jay Shendure, MD, PhD
Professor of Genome Sciences, University of Washington
Director, Allen Discovery Center for Cell Lineage Tracing
Scientific Director, Brotman Baty Institute for Precision Medicine
Dr. Shendure is an Investigator of the Howard Hughes Medical Institute, Professor of Genome Sciences at the University of Washington, Director of the Allen Discovery Center for Cell Lineage Tracing, and Scientific Director of the Brotman Baty Institute for Precision Medicine. His 2005 doctoral thesis with George Church included one of the first successful reductions to practice of next generation DNA sequencing. Dr. Shendure’s research group in Seattle pioneered exome sequencing and its earliest applications to gene discovery for Mendelian disorders and autism; cell-free DNA diagnostics for cancer and reproductive medicine; massively parallel reporter assays, saturation genome editing; whole organism lineage tracing, and massively parallel molecular profiling of single cells. Dr. Shendure is the recipient of the 2012 Curt Stern Award from the American Society of Human Genetics, the 2013 FEDERAprijs, a 2013 NIH Director’s Pioneer Award, the 2014 HudsonAlpha Life Sciences Prize, the 2018 Richard and Carol Hertzberg Prize for Technology Innovation, and the 2019 Richard Lounsbery Award from the National Academy of Sciences. He serves or has served as an advisor to the NIH Director, the US Precision Medicine Initiative, the National Human Genome Research Institute, the Chan-Zuckerberg Initiative and the Allen Institutes for Cell Science and Immunology. He received his MD and PhD degrees from Harvard Medical School in 2007.
Claes Wahlestedt, MD, PhD
Claes Wahlestedt, MD, PhD
Dr. Wahlestedt, M.D., Ph.D. is Leonard M. Miller Professor and Director of the Center for Therapeutic Innovation at the University of Miami where he also serves as Associate Dean for Therapeutic Innovation. A native of Sweden, Dr. Wahlestedt obtained his MD and PhD degrees from Lund University. Prior to joining the University of Miami, Dr. Wahlestedt was a founding professor and a director at The Scripps Research Institute’s Florida campus. Before that he was an endowed professor, founding genome center director and department chair at the Karolinska Institute in Stockholm. He has also been a faculty member at Cornell University Medical College and at McGill University. At different stages in his career, he has directed large R&D organizations in the pharmaceutical industry for Astra-Zeneca and Pharmacia/Pfizer. In recent years, he has co-founded several biotechnology companies. Dr. Wahlestedt is a drug discovery as well as a genome/transcriptome researcher with over 300 publications. He has extensive experience across different therapeutic areas with various therapeutic modalities, in particular small molecules and oligonucleotides.
Joanna Wysocka, PhD
Joanna Wysocka, PhD
Joanna Wysocka is a Lorry Lokey Professor in the Department of Chemical and Systems Biology and the Department of Developmental Biology at Stanford University, a Member of the Stanford Institute for Stem Cell Biology and Regenerative Medicine and an HHMI Investigator.
Dr. Wysocka has done her PhD work at the Cold Spring Harbor Laboratory and, after graduating in 2003, postdoctoral training at the Rockefeller University. She became a faculty member at Stanford in 2006. Dr. Wysocka’s research is focused on understanding gene regulatory mechanisms in human development, disease and evolution. Her lab is employing a broad combination of genomic, genetic, biochemical, biophysical, single-cell and embryological approaches in a number of cellular and organismal models to investigate functions of the non-coding parts of the genome, understand regulatory mechanisms underlying stem cell function, cellular plasticity and differentiation, investigate how quantitative changes in gene expression dictate differences in human traits, and study craniofacial development and variation. Dr. Wysocka is a recipient of numerous awards, including the Searle Scholar Award, W.M. Keck Foundation Distinguished Young Scholar Award, ISSCR Outstanding Young Investigator Award, and Vilcek Prize for Creative Promise. She was elected to the American Academy of Arts and Sciences in 2018 and as an EMBO international member in 2019.
Al Sandrock, Jr., M.D., Ph.D.
Al Sandrock, Jr., M.D., Ph.D.
Dr. Sandrock is recognized as an outstanding industry leader who brought numerous transformational therapies for the treatment of neurological diseases to patients. Dr. Sandrock spent 23 years at Biogen where he identified and developed novel therapies for a variety of serious diseases. While at Biogen, Dr. Sandrock served in positions of increasing responsibility, culminating in his service as Executive Vice President, Research and Development. He also served as Chief Medical Officer and held a seat on the Biogen Executive Committee. Over the course of his tenure, he led the discovery and development, and regulatory approval of numerous medicines including: ADUHELM, PLEGRIDY, SPINRAZA, TECFIDERA, and TYSABRI. Dr. Sandrock earned a B.A. in human biology from Stanford University, an M.D. from Harvard Medical School, and a Ph.D. in neurobiology from Harvard University. He completed an internship in medicine, a residency and chief residency in neurology, and a clinical fellowship in Neuromuscular Disease and Clinical Neurophysiology (electromyography) at Massachusetts General Hospital.