executive leadership team
Sandy Macrae, M.B., Ch.B., Ph.D.
Chief Executive Officer
Sandy Macrae, M.B., Ch.B., Ph.D., has served as Sangamo’s President and Chief Executive Officer and as a member of the Board of Directors since June 2016. He has twenty years of experience in the pharmaceutical industry most recently serving as the Global Medical Officer of Takeda Pharmaceuticals, from 2012 to March 2016, where he established and led the Global Medical Office, which encompasses medical affairs, regulatory affairs, pharmacovigilance, outcomes research and epidemiology, quantitative sciences and knowledge and informatics. From 2001 to 2012, Dr. Macrae held roles of increasing responsibility at GlaxoSmithKline, including Senior Vice President, Emerging Markets Research and Development (R&D), from 2009 to 2012. In that position, he provided expertise and resources to create a first-of-its-kind group to expand GSK’s global reach by providing R&D strategies, clinical development and regulatory resources to enter emerging markets and Asia-Pacific. From 2007 to 2008, he was Vice President, Business Development. In that position, he was responsible for scientific assessment and business development project leadership for the neurology, psychiatry, cardiovascular and metabolic therapeutic areas. Earlier in his career, he worked for SmithKline Beecham, where he was responsible for clinical development in the therapeutic areas of neurology and gastroenterology.
Dr. Macrae received his B.S. in pharmacology and his M.B., Ch.B. with honors from Glasgow University. He is a member of the Royal College of Physicians. Dr. Macrae also earned his Ph.D. in molecular genomics at King’s College, Cambridge.
Executive Vice President, Corporate Strategy
Stéphane Boissel has served as Executive Vice President of Corporate Strategy since joining Sangamo in October 2018. Prior to joining Sangamo, he served as CEO of TxCell SA, a CAR-Treg company based in Valbonne, France, which Sangamo acquired in 2018. Mr. Boissel is an experienced biotech professional who brings over 25 years of leadership experience across corporate finance, strategy and business development. Prior to his appointment as CEO of TxCell in April 2015, he served as CEO of Genclis, a molecular diagnostics company. From 2002 to 2010, he served as CFO then EVP and CFO of Innate Pharma SA, and from 2010 to 2014 he served as EVP and CFO of Trangene SA. Earlier in his career, Mr. Boissel worked in investment banking for Lazard, where he focused on principal investment in France, Singapore and Hong Kong.
Mr. Boissel completed his undergraduate work in management and finance at the University of Lyon and Paris-Dauphine in France and received his M.B.A. from the University of Chicago.
Adrian Woolfson, B.M., B.Ch., Ph.D.
Executive Vice President, Research & Development
Adrian Woolfson, BM, BCh, PhD, has served as Sangamo’s Executive Vice President of Research & Development since January 2019. Dr. Woolfson has over a decade of biopharmaceutical industry experience in drug discovery, medical affairs and early and late stage clinical development. Most recently, he served as Chief Medical Officer at Nouscom AG, a genetic cancer vaccine biotechnology company based in Basel, Switzerland, where he led the development of the company’s off-the-shelf and personalized neoantigen vaccine and oncolytic virus strategy. Prior to Nouscom, Dr. Woolfson served as Global Clinical Leader, Early and Late Stage Immuno-Oncology/Hematology at Pfizer Inc. in New York, and was responsible for defining Pfizer’s hematology immuno-oncology strategy and building its immuno-oncology hematological malignancies portfolio. Prior to that he was the Global Lead for Pfizer’s SMO inhibitor glasdegib, which received FDA approval in 2018. From 2007 to 2013, Dr. Woolfson held roles of increasing responsibility at Bristol-Myers Squibb, including Global Medical Lead for a first-in-human CDC7 inhibitor and selective JAK2 inhibitor.
Dr. Woolfson completed his post-graduate training in internal medicine at Addenbrooke’s Hospital Cambridge in the UK. He holds a BM BCh degree in Clinical Medicine from Oxford University and completed his PhD in molecular immunology at Cambridge University. He was the Charles and Katherine Darwin Research Fellow at Darwin College Cambridge, and a Wellcome Trust Clinical Research Fellow. He completed his doctoral and post-doctoral work in the laboratory of Nobel Prize winner and inventor of monoclonal antibodies Dr César Milstein in the Division of Protein and Nucleic Acid Chemistry at the MRC Laboratory of Molecular Biology in Cambridge, UK.
Executive Vice President and Chief Financial Officer
Sung Lee has served as Sangamo’s Executive Vice President and Chief Financial Officer since October 2019. Prior to joining Sangamo he spent nearly 14 years at Gilead Sciences where he most recently served as Senior Vice President leading the Financial Planning & Analysis (FP&A) and Investor Relations functions. He managed the global planning and budgeting process, drove process improvement, and managed all aspects of communications to the investor community. Prior to that role, he held roles of increasing responsibility at Gilead, including Head of Europe Finance and Head of R&D FP&A.
Mr. Lee received a B.A. in Economics from University of California, Irvine, and a M.B.T. from University of Southern California.
Gary H. Loeb
Executive Vice President and General Counsel
Gary H. Loeb has served as Sangamo’s Executive Vice President and General Counsel since July 2019. He is responsible for all legal matters for Sangamo. Mr. Loeb has over 20 years of experience in pharmaceutical compliance, legal transactions and operations, and executive leadership. Prior to joining Sangamo, he served as General Counsel, Corporate Secretary, and Chief Compliance Officer at Achaogen, an anti-infectives company. As a member of the Executive Team, he built Achaogen’s legal and compliance functions and supported its first product launch. Before Achaogen, Mr. Loeb was General Counsel for Counsyl, a genetic screening company, and Amyris, Inc., a synthetic biology company. Mr. Loeb began his in-house career at Genentech, where he was Vice President of Intellectual Property.
Mr. Loeb received his B.S. and B.A. degrees from Stanford University and his J.D. from Columbia Law School.
R. Andrew Ramelmeier, Ph.D.
Executive Vice President, Technical Operations
R. Andrew “Andy” Ramelmeier, PhD, currently serves as Executive Vice President, Technical Operations and is responsible for Technical Operations at Sangamo, including manufacturing, quality supply chain, and process and analytical development. Dr. Ramelmeier has 25 years of experience in the biopharmaceutical industry, developing and transferring biological processes, designing and building manufacturing facilities, and directing contract manufacturers as well as internal manufacturing operations. Prior to joining Sangamo in January 2018, he served as Senior Vice President, Technical Operations at Portola Pharmaceuticals, Inc., where he was responsible for tech transfer, bulk and drug product manufacturing, technical support and supply chain of Portola’s pipeline products. From 2006 to 2014, Dr. Ramelmeier served as Vice President, Manufacturing, Process Sciences and Facilities at BioMarin, overseeing multiple commercial biologics products, clinical pipeline, and facilities in Novato, CA, and Shanbally, Ireland. Earlier in his career, he held roles of increasing responsibility at Johnson & Johnson and Merck.
Prior to joining industry, Dr. Ramelmeier conducted post-doctoral work in Germany. He received a B.S. in Chemical Engineering from Johns Hopkins and his Ph.D. in Chemical Engineering from the University of California, Berkeley.
Edward Rebar, Ph.D.
Senior Vice President and Chief Technology Officer
Edward Rebar, Ph.D., has served as Senior Vice President and Chief Technology Officer since July 2018. Dr. Rebar joined Sangamo in 1998, and over the last 20 years has led the development of the Company's zinc finger protein technology platform. He has authored over 60 publications relating to the development of customized DNA binding proteins and nucleases for genome editing. Prior to joining Sangamo, he was a post-doctoral fellow at the University of California, Berkeley.
Dr. Rebar earned his B.S. in Biochemistry from Rutgers University and his Ph.D. in Biophysics and Structural Biology from MIT.
Bettina Cockroft, M.D.
Senior Vice President and Chief Medical Officer
Bettina Cockroft, M.D., M.B.A., has served as Senior Vice President and Chief Medical Officer since September 2019 and oversees all clinical development activities and operations. She has over 20 years of experience in the biopharmaceutical industry and has worked across multiple therapeutic areas and led programs in several countries. Prior to joining Sangamo, Dr. Cockroft served on the senior leadership team at Cytokinetics, Inc., where she was responsible for clinical development of fast skeletal muscle troponin activators in diseases such as Amyotrophic Lateral Sclerosis and Spinal Muscular Atrophy. Before that, Dr. Cockroft served as Chief Medical Officer of Auris Medical AG, where she led and grew the clinical development team responsible for two Phase 3 programs. Dr. Cockroft also held roles of increasing responsibility at Merck Serono S.A., Novartis Consumer Health and Menarini Ricerche earlier in her career.
Dr. Cockroft earned her M.B.A. at MIT Sloan School of Management and her M.D. from the University of Genova.
Jason Fontenot, PH.D.
Senior Vice President, Cell Therapy
Jason Fontenot, Ph.D., has served as Sangamo’s Senior Vice President of Cell Therapy since March 2019. Dr. Fontenot oversees Sangamo's engineered cell therapy portfolio, including a proprietary chimeric antigen receptor regulatory T cells (CAR-Tregs) pipeline, a partnership with Sanofi focused on ex vivo genome editing programs in hemoglobinopathies, and a partnership with Kite, a Gilead Company, to develop autologous and allogeneic CAR-T cell therapies in oncology. Dr. Fontenot has an extensive background in cell therapy and drug development, having previously served as Chief Scientific Officer at Immusoft, Head of Exploratory Research at Juno Therapeutics, and for nearly a decade as a group leader in the immunology department at Biogen.
Dr. Fontenot has expertise in lymphocyte biology, cell engineering and immune-mediated disease. His seminal work on regulatory T cells and FOXP3 conducted with Alexander Rudensky and colleagues transformed the study of immune regulation and produced some of the most highly cited immunology publications of the last 20 years.
Dr. Fontenot conducted post-doctoral work at Rockefeller University in New York City. He received his Ph.D. in Immunology from the University of Washington in Seattle working in the laboratory of Alexander Rudensky.
Nathalie Dubois-Stringfellow, Ph.D.
Senior Vice President, Product Development and Management
Nathalie Dubois-Stringfellow, Ph.D., currently serves as Senior Vice President, Product Development and Management. She oversees the development and execution of project team strategy for Sangamo’s ZFP therapeutic programs in hemophilia, lysosomal storage disorders, hemoglobinopathies, HIV and Huntington’s disease. She has over 20 years of experience implementing and managing preclinical and clinical development of biologic therapies in oncology, as well as immune, infectious and genetic diseases. Dr. Dubois-Stringfellow joined Sangamo in January 2011 as Senior Director, Project Management. Prior to joining Sangamo, she held various positions in Discovery Research, Preclinical Research, Project Management, Clinical Development, and Portfolio Management at Chiron Corp., Bayer Corp., Signature Biosciences, Inc. and most recently XOMA LLC, where she served as Senior Director, Preclinical Portfolio and Alliance Management.
Dr. Dubois-Stringfellow was a post-doctoral fellow at the University of North Carolina, Chapel Hill, where she studied angiogenesis and tumorigenesis in transgenic mice and cell culture systems. She received her M.S. in Genetics and Immunology, and her Ph.D. in Human Genetics from the Université Pierre et Marie Curie in Paris, France.
Senior Vice President, Corporate Communications and Investor Relations
McDavid Stilwell currently serves as Senior Vice President, Corporate Communications and Investor Relations. He has over 20 years of relevant experience across various business functions, including communications, investor relations, business development and financial analysis. Prior to joining Sangamo in 2016, Mr. Stilwell served as Vice President, Corporate Communications, Investor Relations & Business Development at Orexigen Therapeutics, Inc. From 2005 to 2012 he served as Director, Business Development, Corporate Communications and Financial Analysis at GTx, Inc., a development stage biopharmaceutical company. Earlier, Mr. Stilwell worked for five years as a Senior Investment Analyst for Shadwell Capital LLC, a hedge fund. Mr. Stilwell began his career as a journalist, first as a newspaper reporter and later as an executive editor of an investor newsletter covering financial services industry mergers and acquisitions.
Mr. Stilwell received a B.A. in Liberal Arts from St. John’s College, Annapolis, Maryland, and a M.B.A. from Harvard Business School.
Senior Vice President, General Manager of Europe
Duncan McKay currently serves as Sangamo’s Senior Vice President and General Manager of Europe. He has over 30 years of experience in the pharmaceutical industry across R&D and commercial strategy, business development, risk management, compliance and communications. Throughout his career Mr. McKay obtained regulatory approval for over 10 new medicines for patients in major markets. Prior to joining Sangamo in 2018, he held various leadership positions at GlaxoSmithKline (GSK), from 2000 to 2015, in Emerging Markets R&D, Oncology R&D and Clinical Operations of Europe. While at GSK, Mr. McKay developed a high value portfolio of patented and non-patented medicines and created access for several million new patients, played an integral role in the design and creation of Oncology R&D within the organization and successfully led pivotal registration clinical studies for three new oncology medicines. Earlier in his career, he worked in clinical development at Beecham Research Laboratories and SmithKline Beecham, legacy companies of GSK.
Mr. McKay received a B.S. in Biochemistry from the University of Surrey and his M.B.A. from Henley Business School in the United Kingdom.
Vice President, Human Resources
Julie Person has served as Sangamo’s Vice President of Human Resources since March 2019. She is responsible for leading Sangamo’s Human Resources function, including talent management and organizational development, recruitment, and HR processes and programs. Ms. Person brings more than 20 years of global experience in human resources across the biotech and pharma industries. Prior to joining Sangamo, Ms. Person served as Vice President of Talent Management and Organizational Development at Shire (now Takeda). From 2015-2019, Ms. Person served in HR leadership roles at Baxalta/Shire/Takeda. Before Takeda, she worked in roles of increasing responsibility at Blue Shield of California and McKesson Corporation.
Ms. Person received her B.A. in Communications from Saint Mary’s College of California.
Board of Directors
H. Stewart Parker
Chairwoman of the Board
H. Stewart Parker has been a member of our Board of Directors since June 2014. Ms. Parker has over 30 years of experience in the biotechnology industry. She served as the chief executive officer of The Infectious Disease Research Institute (IDRI), a not-for-profit global health research institute from March 2011 to December 2013. In 1992, Ms. Parker founded Targeted Genetics Corporation, a publicly traded Seattle-based biopharmaceutical company formed to develop gene-based treatments for acquired and inherited diseases that became a world leader in AAV gene therapy. She held the position of President and CEO and was a member of its board of directors from the company's inception until November 2008. Prior to founding Targeted Genetics, Ms. Parker served in various capacities at Immunex from August 1981 through December 1991, most recently as vice president, corporate development. From February 1991 to January 1993, Ms. Parker served as president and a director of Receptech Corporation, a company formed by Immunex in 1989 to accelerate the development of soluble cytokine receptor products. She has served on the board of directors and the executive committee of BIO, the primary trade organization for the biotechnology industry. She currently serves as a member of the board of directors for several for-profit and non-profit companies including C3 Jian Inc, Oncogenex Technologies Inc and IDRI and on the advisory boards of the University of Washington Foster School of Business and College of Arts & Sciences. Ms. Parker received her B.A. and M.B.A. from the University of Washington.
Executive Vice President and CBO of Horizon Pharma, plc
Robert F. Carey has served as a member of Sangamo's Board of Directors since June 2016. Mr. Carey currently serves as Executive Vice President and Chief Business Officer of Horizon Pharma, plc, a biopharmaceutical company with nine commercialized medicines though its orphan, rheumatology and primary care units. He also serves as a member of the Board of Directors of Argos Therapeutics, Inc., a biopharmaceutical company focused on the development and commercialization of personalized immuntherapies for the treatment of cancer and infectious diseases. Before joining Horizon Pharma, he served as managing director and head of the healthcare investment banking group at JMP Securities LLC from March 2013 to March 2014. Prior to that, Mr. Carey served as a managing director in the healthcare groups at Dresdner Kleinwort Wasserstein and Vector Securities International, Inc. He has also held roles at Shearson Lehman Hutton and Ernst & Whinney. Mr. Carey received a B.S. in Accounting from the Univesity of Notre Dame.
Stephen Dilly, M.B.B.S., Ph.D.
Former CEO of Aimmune Therapeutics
Stephen G. Dilly, M.B.B.S., Ph.D., has served as a member of our Board of Directors since March 2010. From May 2014 – June 2018, he served as CEO of Aimmune Therapeutics. Prior to that, from December 2011 to December 2012 Dr. Dilly was CEO of PhotoThera, Inc., a medical device company with a product in Phase III clinical trials for the treatment of ischemic stroke. From 2006 to 2011, Dr. Dilly served as President and Chief Executive Officer and a member of the Board of Directors of APT Pharmaceuticals, Inc. From 2003 to 2006, he served as Chief Medical Officer and Senior Vice President of Development of Chiron BioPharma, a biotechnology company which was later acquired by Novartis International AG. From 1998 to 2003, he held various management positions at Genentech Inc., including Vice President of Development Sciences from 2002 to 2003. From 1988 to 1998, Dr. Dilly held various management positions in drug development with SmithKline Beecham in the U.K. During his career, Dr. Dilly has been closely associated with the development and launch of marketed drugs for many therapeutic areas, including Kytril, Paxil, Kredex, Requip, TNKase, Xolair, Avastin, Raptiva, Tarceva, Lucentis and Cubicin. Dr. Dilly currently serves as a member of the Advisory Board of Physic Ventures and the National Board of Advisors of the UC Davis Health System. In 1982, Dr. Dilly earned his M.B.B.S., the equivalent of an M.D. in the U.S., from the University of London in the U.K. and received his Ph.D. in Cardiac Physiology from University of London in 1988.
Sandy Macrae, M.B., Ch.B., Ph.D.
Chief Executive Officer
Sandy Macrae, M.B., Ch.B., Ph.D., has served as Sangamo’s President and Chief Executive Officer and as a member of the Board of Directors since June 2016. He has twenty years of experience in the pharmaceutical industry most recently serving as the Global Medical Officer of Takeda Pharmaceuticals, from 2012 to March 2016, where he established and led the Global Medical Office, which encompasses medical affairs, regulatory affairs, pharmacovigilance, outcomes research and epidemiology, quantitative sciences and knowledge and informatics. From 2001 to 2012, Dr. Macrae held roles of increasing responsibility at GlaxoSmithKline, including Senior Vice President, Emerging Markets Research and Development (R&D), from 2009 to 2012. In that position, he provided expertise and resources to create a first-of-its-kind group to expand GSK’s global reach by providing R&D strategies, clinical development and regulatory resources to enter emerging markets and Asia-Pacific. From 2007 to 2008, he was Vice President, Business Development. In that position, he was responsible for scientific assessment and business development project leadership for the neurology, psychiatry, cardiovascular and metabolic therapeutic areas. Earlier in his career, he worked for SmithKline Beecham, where he was responsible for clinical development in the therapeutic areas of neurology and gastroenterology. Dr. Macrae received his B.Sc. in pharmacology and his M.B., Ch.B. with honors from Glasgow University. He is a member of the Royal College of Physicians. Dr. Macrae also earned his Ph.D. in molecular genomics at King’s College, Cambridge.
Managing Partner of Life Sciences Advisory
Saira Ramasastry has served as a member of our Board of Directors since June 2012. Since April 2009 she has served as Managing Partner of Life Sciences Advisory, LLC, a company that she founded to provide strategic advice and business development solutions for life science companies. From 1999 to 2009, Ms. Ramasastry was an investment banker with Merrill Lynch & Company, Inc. where she helped establish the biotechnology practice and was responsible for origination of mergers and acquisitions (M&A), strategic and capital markets transactions. Prior to joining Merrill Lynch she served as a financial analyst in the M&A group at Wasserstein Perella & Co., an investment banking firm. Ms. Ramasastry currently serves on the Industry Advisory Board of the Michael J. Fox Foundation for Parkinson's Research, the board of directors of Pain Therapeutics and Repros Therapeutics, and lead business advisor for the European Prevention of Alzheimer's Dementia (EPAD) consortium. She received a B.A. in Economics with Honors and Distinction and an M.S. in Management Science and Engineering from Stanford University as well as an M. Phil. in Management Studies from the University of Cambridge, where she is a guest lecturer for the Bioscience Enterprise Programme and serves on the Cambridge Judge Business School Advisory Council.
Karen Smith, M.D., Ph.D., M.B.A.
Formerly Executive Vice President, Global Head R&D, and CMO of Jazz Pharmaceuticals
Karen Smith, M.D., Ph.D., M.B.A., has served as a member of Sangamo’s Board of Directors since June 2018. Dr. Smith has over 20 years of biopharmaceutical industry experience in the United States, Europe, Canada and Asia. She has overseen more than 50 clinical trials and more than 20 regulatory approvals leading to global product launches of small molecules, biologics and devices. Most recently, she served as Executive Vice President, Global Head R&D, and Chief Medical Officer of Jazz Pharmaceuticals, Inc. Prior to joining Jazz in 2015, Dr. Smith served as Senior Vice President, Global Medical Affairs and Global Dermatology Head for Allergan plc. From 2008 to 2011, she held leadership positions in External Medical Relations and Global Development at AstraZeneca, and from 2002 to 2008 she held various senior management and clinical roles with Bristol-Myers Squibb, including Head of U.S. Clinical Operations. Dr. Smith currently is a member of the Board of Directors of Acceleron Pharma and a Board Advisor for the Australian Philanthropic 'Eliminate Cancer' Initiative.
Dr. Smith received a B.App.Sc. in Biotechnology and a B.Sc. in Haematology from Curtin University of Technology, an M.D. from the University of Warwick, her Ph.D. in Oncology Molecular Genetics from the University of Western Australia, an M.B.A. from the University of New England in Australia, and a Master of Laws (L.L.M.) in Health Law from the University of Salford.
Joseph S. Zakrzewski
Formerly Chairman and CEO of Amarin Pharmaceuticals
Joseph S. Zakrzewski has served as a member of Sangamo's Board of Directors since June 2017. Mr. Zakrzewski has over 25 years of experience in the biopharmaceutical industry with senior leadership experience in R&D, supply chain and manufacturing operations, business development and commercialization. From 2010 through 2013, he was Chairman and CEO of Amarin Pharmaceuticals, where he led the development and commercialization of the company's first product, Vascepa. Mr. Zakrzewski previously served as a Venture Partner with Orbimed Advisors, a venture capital firm, and as Chairman, President and CEO of Xcellerex, a privately held company focused on the commercialization of its proprietary manufacturing technology for biotherapeutics and vaccines. Earlier, he served as COO of Reliant Pharmaceuticals before its acquisition by GlaxoSmithKline in 2007 and held various executive positions at Eli Lilly & Company in the areas of R&D, manufacturing, finance and business development. Mr. Zakrzewski currently serves as a member of the board of directors of various public and private companies, including Acceleron Pharma, Amarin Pharmaceuticals Insulet Corporation and Onxeo SA where he serves as Chairman. Mr. Zakrzewski received a B.S. in Chemical Engineering and an M.S. in Biochemical Engineering from Drexel University, and an M.B.A. in Finance from Indiana University.
Scientific Advisory Board
Roger Kornberg, Ph.D.
Winzer Professor in Medicine, Structural Biology Department, Stanford University Medical School and Chair of the Sangamo Scientific Advisory Board
Roger Kornberg is the Winzer Professor in Medicine in the Department of Structural Biology at Stanford University. In his doctoral research, he demonstrated the diffusional motions of lipids in membranes, termed flip-flop and lateral diffusion. He was a postdoctoral fellow and member of the scientific staff at the Laboratory of Molecular Biology in Cambridge, England from 1972-1975, where he discovered the nucleosome, the basic unit of DNA coiling in chromosomes. He moved to his present position in 1978, where his research has focused on the mechanism and regulation of eukaryotic gene transcription. Notable findings include the demonstration of the role of nucleosomes in transcriptional regulation, the establishment of a yeast RNA polymerase II transcription system and the isolation of all the proteins involved, the discovery of the mediator of transcriptional regulation, the development of two-dimensional protein crystallization and its application to transcription proteins, and the atomic structure determination of an RNA polymerase II transcribing complex. Kornberg was elected to the National Academy of Sciences in 1993. He has received many awards, including the Welch Prize (2001), highest award in chemistry in the United States, the Leopold Mayer Prize (2002), highest award in biomedical sciences of the French Academy of Sciences, and the Nobel Prize in Chemistry (unshared, 2006). Kornberg received his B.A. from Harvard College and his Ph.D. in Chemistry from Stanford University. His closest collaborator has been his wife, Dr. Yahli Lorch. They have three children, Guy, Maya, and Gil.
Dr. Mark Chee, Ph.D.
President, Encodia Inc., CEO and CSO Prognosys Biosciences
Mark Chee, Ph.D., is the President of Encodia and an internationally recognized expert in genomics technologies. He serves as President of Encodia and CEO and CSO of Prognosys Biosciences. Previously, he co-founded Illumina, and was Director of Genetics Research at Affymetrix. He also serves as a Scientific Advisor to Complete Genomics and Omniome and was a member of the National Human Genome Research Institute Advisory Council and the Human Cancer Genome Project External Scientific Committee. Dr. Chee is an inventor on over 100 issued US patents. He received his B.Sc. in Biochemistry from the University of New South Wales and his Ph.D. in Molecular Biology from the University of Cambridge.
Jef Boeke, Ph.D., D.Sc.
Director, Institute for Systems Genetics, NYU Langone Health
Jef D. Boeke, Ph.D., D.Sc., founded and directs the Institute for Systems Genetics at NYU Langone Health. From 1985-2014 Dr. Boeke was on the faculty at Johns Hopkins University School of Medicine. Dr. Boeke is known for his foundational work on mechanistic and genomic aspects of retrotransposition. His lab develops new technologies in genetics, genomics and synthetic biology. He elucidated a major form of mobile DNA, based on reverse transcription of RNA. He coined the term “retrotransposition” to describe this process, common to virtually all eukaryotic genomes and now studied by a worldwide scientific community. His systems-level studies helped elucidate intricate molecular mechanisms involved in retrotransposition in yeasts, mice and humans.
In the area of synthetic biology, Jef Boeke is using yeast as a platform for exploring the construction of fully synthetic chromosomes for practical and theoretical studies. He leads an international team to synthesize an engineered version of the yeast genome, Sc2.0, the first synthetic eukaryotic genome, and a consortium to explore the design and synthesis of even larger genomes. In 2018, he launched the “dark matter project” designed to better understand the “instruction manuals” that specify how human genes are expressed, using big DNA technology.
Megan Levings, Ph.D.
Professor, Department of Surgery and School of Biomedical Engineering, University of British Columbia, Lead, Childhood Diseases Theme, BC Children’s Hospital Research Institute
Dr. Megan Levings has been in the University of British Columbia Department of Surgery since 2003, when she was recruited as a Canada Research Chair in Transplantation. In 2011, she joined the BC Children’s Hospital Research Institute where she now heads the Childhood Diseases Research Theme. Dr. Levings’ scientific career started with summer research positions in a fruit fly genetics lab at Simon Fraser University. She then did her graduate training in the genetics program with Dr. John Schrader at UBC, during which time she studied cytokine receptors and signaling pathways. In 1999 she joined Dr. Maria Grazia Roncarolo's lab in Milan, Italy, undertaking postdoctoral training in the then emerging area of immune regulation. She was among the first groups to show that a special kind of white blood cell, known as a T regulatory cell, could be used as a cellular therapy to stop harmful immune responses. She continues this line of research at UBC, and now leads a vibrant group of trainees and staff who are researching how to use T regulatory cells to replace conventional immunosuppression in the context of transplantation and autoimmunity. She is internationally recognized in the field of human immunology and currently chairs the Federation of Clinical Immunology Societies Centers' of Excellence and is a member of the NIH-funded Immune Tolerance Network steering committee.
Stuart Schreiber, Ph.D.
Morris Loeb Professor of Chemistry and Chemical Biology, Harvard University
Stuart L. Schreiber, Ph.D. is a co-Founder of the Broad Institute, the Morris Loeb Professor of Chemistry and Chemical Biology at Harvard University, and is a member of the National Academy of Sciences, National Academy of Medicine and American Academy of Arts and Sciences. His research has been recognized through numerous awards, most recently being the Wolf Prize in Chemistry.
Dr. Schreiber’s research integrates chemical biology and human biology to advance the discovery of novel therapeutics. He is known for his use of small molecules to explore biology and medicine, and for his role in the development of the field of chemical biology. His lab discovered mTOR and illuminated the nutrient-response signaling network, histone deacetylase (HDAC) and the role of chromatin marks in gene expression, and with Gerry Crabtree he mapped the first membrane to nuclease signaling pathway (calcium–calcineurin–NFAT). His work demonstrated for the first time that drugs can result from: 1) the targeting of protein kinases (sirolimus/mTOR) and protein phosphatases (sandimmune/calcineurin); 2) gene regulation by chromatin-modifying enzymes (vorinostat/HDAC), 3) small-molecule dimerizers that activate cellular processes by enforced proximity (GVH Disease); and 4) targeting of the proteasome (bortezomib/proteasome). These efforts accelerated the development of many additional widely used drugs.
Schreiber’s development of diversity-oriented synthesis has led to the discovery of many promising agents, including a novel mechanism of action anti-malarial agent being developed in collaboration with the pharmaceutical company Eisai. His most recent discovery revealed a novel cell state responsible for the ability of cancers to resist a wide range of therapies, and a means to target the cancer therapy-resistant state.
Schreiber extended chemical biology principles to medicine by founding several biotech companies, including Vertex Pharmaceuticals (fosamprenavir/Lexiva; telaprevir/Incivek; ivacaftor/Kalydeco), ARIAD Pharmaceuticals (ponatinib/Iclusig; brigatinib/Alunbrig; ridaforolimus; AP1903), Infinity Pharmaceuticals (reta¬spimycin; duvelisib), Forma Therapeutics, H3 Biomedicine, Jnana Therapeutics, Kojin Therapeutics, oNeir Therapeutics, and Kisbee Therapeutics.
Samuel Aparicio, BM., B.Ch., Ph.D., FRCPath FRSC
Senior Scientific Director of Cancer Genomics and Co-Chair, Enabling Innovation Working Group, New York Genome Center
Dr. Aparicio is the Nan & Lorraine Robertson Chair in Breast Cancer Research, holds the Canada Research Chair (Tier 1) in Molecular Oncology, and is a Fellow of the Royal Society of Canada. He is also Head of the Department of Molecular Oncology at BC Cancer and a Professor in the Department of Pathology and Laboratory Medicine at the University of British Columbia. Dr. Aparicio's research program encompasses the fields of cancer genomics, cancer evolution, single cell biology, mouse genetic models, high throughput screens, small molecule chemical probe development, and translational breast cancer research.
Dr. Aparicio graduated in medical and natural sciences from Cambridge University (UK), clinical medicine from Oxford, and subsequently in internal medicine and pathology. After doctoral work with Sydney Brenner in Cambridge, he held a Wellcome Trust Career Development Fellowship at the Wellcome/CRUK Developmental Biology Institute. From 2000-2005 he was a senior investigator in the Department of Oncology, Cambridge. He was a co-leader of the international consortium that sequenced the genome of the pufferfish Fugu rubripes in 2002. He moved to Vancouver in 2005 where he has been leading genomics research in cancer. He was a co-founder of Paradigm Therapeutics (eventually acquired by Takeda Pharma) and currently Contextual Genomics Ltd. His contributions to academic research have been widely published in scientific and clinical journals such as Nature, Science, Cell and The New England Journal of Medicine. He is the recipient of numerous awards from academic as well as industrial institutions.
Michael Grunstein, Ph.D.
Distinguished Professor, Biological Chemistry, UCLA - Department of Biological Chemistry
Michael Grunstein’s accomplishments include the rapid identification of bacterial colonies that carry eukaryotic genes of interest. This protocol “Colony Hybridization” was published in 1975 and the paper is one of the most highly referenced papers in the recombinant DNA cloning field. After completing his post-doctoral work, first with Larry Kedes and then with David Hogness at Stanford, Grunstein moved to UCLA where his group was at the vanguard of the chromatin field. His discoveries since 1980 include the finding that yeast histones and their post-translational modifications regulate gene activity. It was recognized by a number of prizes in Genetics, the most recent being the Gruber and the Lasker awards.
Robert J. Desnick, Ph.D., M.D. D.Sc. (Hon)
Dean for Genetics and Genomic Medicine & Professor & Chair Emeritus, Icahn School of Medicine at Mount Sinai
Dr. Robert J. Desnick is Dean for Genetics and Genomic Medicine and Professor and Chairman Emeritus of the Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai. In 1977, he joined the Mount Sinai faculty as the Arthur J. and Nellie Z. Cohen Professor of Pediatrics and Genetics. In 1993, he became the first Chairman of the Department of Genetics and Genomic Sciences at Mount Sinai, and in 2011, he was appointed Dean for Genetics and Genomic Medicine. His research interests include inherited metabolic diseases and their treatment. His basic laboratory research efforts led to FDA- & EMA-approval of enzyme replacement therapy (ERT) for Fabry disease (Fabrazyme) and on-going ERT clinical trials for Niemann-Pick B disease with Sanofi-Genzyme. He was co-scientific founder of Amicus Therapeutics, which develops oral pharmacologic chaperone therapies for various diseases including Fabry disease (EMA- & FDA-approved). He was Principal Investigator for Clinuvel Pharmaceuticals’ multisite clinical trials of afamelanotide, a “first-in-class” synthetic peptide of α-melanocyte stimulating hormone for the treatment of the Erythropoietic Porphyrias (EMA- & FDA-approved). He is a co-inventor of RNAi therapy for the Acute Hepatic Porphyrias (NDA pending) and is currently using gene editing/gene therapy for the treatment of inherited diseases. He served as the Chairman of the Scientific Advisory Committee (SAC) of Synageva Biopharma and as SAC Chair for Kiniksa Pharmaceuticals. He has published over 790 research papers and chapters, including ten edited books. He is a Fellow of the American Association for the Advancement of Science and an elected member of the National Academy of Medicine.
Dr. Desnick received his B.A., M.D., and Ph.D. degrees from the University of Minnesota.
Douglas Higgs, FRS
Director, MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford, John Radcliffe Hospital
Professor Douglas Higgs is one of the UK’s most prominent hematologists at national and international levels. His excellent research has been recognized by his election as a Fellow of the Royal Society, a Fellow of the Academy of Medical Sciences, and a member of the European Molecular Biology Organization. Despite extensive administrative duties he continues to run an outstanding research group who regularly publish their work in the highest profile scientific and medical journals. He was recently awarded the Royal Society’s Buchannan Medal for Medical Research. Over the past ten years he has built one of the UK’s leading academic departments of clinical hematology with an excellent record of clinical research in malignant and non-malignant hematology, which is now recognized as one of the leading UK centers of excellence for training in academic hematology. He has recently widened his contribution to medicine, having been appointed as the Director of the MRC Weatherall Institute of Molecular Medicine (University of Oxford). Over the past 5 years he has raised over £45M in competitive grants for medical research.
Frank Walsh, Ph.D., B.Sc.
Founder and CEO, Ossianix
Frank S. Walsh is the founder and CEO of Ossianix, a Biotechnology company that develops single domain VNAR antibodies for biologics drug delivery to the brain. Frank was previously head of Discovery Research worldwide at Wyeth Pharmaceuticals in Collegeville, PA (2002-2009). Prior to joining Wyeth, he was SVP at GlaxoSmithKline UK and Head of the Neurology CEDD since 2000. From 1997-2000 he was VP of Neuroscience Research at Smith Kline Beecham UK. Before joining the pharmaceutical industry, Frank had a distinguished academic career in Neuroscience as the Sir William Dunn Professor of Experimental Pathology at London’s UMDS, Guy’s Hospital and Institute of Neurology, Queen Square.
Frank received his Ph.D. from University College London and BSc from University of Strathclyde in Glasgow. His postdoctoral work was with Marshall Nirenberg at the NIH, Bethesda, USA. He has been awarded Honorary degrees from the Universities of Bologna and Perugia in Italy and Dundee and Strathclyde in Scotland. He is a fellow of the Royal Society of Edinburgh, the Academy of Medical Sciences in London, and King’s College London.
Steven W. Pipe, M.D.
Professor of Pediatrics and Pathology, Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases, Pediatric Medical Director, Hemophilia and Coagulation Disorders Program, Director Special Coagulation Laboratory, University of Michigan
Dr. Steven Pipe is a Professor and the Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology at the University of Michigan, Ann Arbor, Michigan, USA. He is the medical director of the Pediatric Hemophilia and Coagulation Disorders Program and medical director of the Special Coagulation Laboratory. His clinical interests include bleeding and thrombotic disorders and congenital vascular anomalies. Dr. Pipe also directs a basic research lab investigating coagulation factor VIII and the molecular mechanisms of hemophilia A. He has been actively involved in clinical trials with novel therapeutics for hemophilia including gene therapy. He was the 2015 recipient of the Leadership in Research Award from the National Hemophilia Foundation. He has served on the Board of Directors for the Hemostasis and Thrombosis Research Society, as Chair of the Board of Directors for the American Thrombosis and Hemostasis Network, and currently as Chair of the Medical and Scientific Advisory Committee to the National Hemophilia Foundation.
J Fraser Wright, Ph.D.
Professor of Pediatrics, Center for Definitive and Curative Medicine, Stanford University School of Medicine, Principal, Wright Biologics Consulting
Dr. Wright is Professor of Pediatrics at the Stanford University School of Medicine, Center for Definitive and Curative Medicines, and is Principal at Wright Biologics Consulting. He is co-founder and former Chief Technology Officer at Spark Therapeutics, Inc. He was the founding Director of the gene therapy Clinical Vector Core Laboratory at The Children's Hospital of Philadelphia, and Professor of Pathology and Laboratory Medicine at the University of Pennsylvania Perleman School of Medicine. Dr. Wright also previously served as Director of Development and Clinical Manufacturing at Avigen, Inc., and as a Scientist at Sanofi Pasteur. His career has focused on clinical gene therapy for over 25 years, contributing to several investigational product development programs including those for Luxturna and Kymriah. Dr. Wright is trained in biochemistry, immunology, and virology and received his B.S. and Ph.D. in biochemistry from the University of Toronto.