NeuBase Therapeutics, Inc., is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides.
The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.
NeuBase is using its proprietary PATrOL™ platform to develop targeted drugs for rare genetic disease.
Dr. Stephan is an industry veteran who is considered one of the fathers of the field of precision medicine, having trained with the leadership of the Human Genome Project at the NIH and then going on to lead discovery research at the Translational Genomics Research Institute and serve as professor and chairman of the Department of Human Genetics at the University of Pittsburgh. Stephan has identified the molecular basis of dozens of genetic diseases and published extensively in journals such as Science, the New England Journal of Medicine, Nature Genetics, PNAS and Cell. In parallel, Dr. Stephan has founded or co-founded 14 biotechnology companies and has advised an additional 12 companies. These companies are backed by the top-tier investors such as Sequoia Capital, KPCB, Thiel Capital, and Khosla Ventures as well as corporate partners such as Life Technologies, Pfizer, and Mayo Clinic. Dr. Stephan received his Ph.D. from the University of Pittsburgh and his BS from Carnegie Mellon University.
Dr. Friedlander is a renowned neurologist. Prior to NeuBase, he became the fourth Chairman of the Department of Neurological Surgeons at the University of Pittsburgh School of Medicine and the University of Pittsburgh Medical Center. Prior to joining the department, Dr. Friedlander was professor of neurosurgery at Harvard Medical School and vice-chairman of neurosurgery and associate director of cerebrovascular surgery at Brigham and Women’s Hospital in Boston. Presently, Dr. Friedlander is the Walter E. Dandy Professor of Neurosurgery, Neurology and Neurobiology, and Co-Director of the UPMC Neurological Institute. His work has been published in many top tier journals including Nature, Science, Nature Medicine, Nature Neuroscience and PNAS, and he has been recognized through many academic awards. In 2006, he was elected as a member of the prestigious American Society for Clinical Investigation. Dr. Friedlander is one of only three neurosurgeons elected as a member of the American Association of Physicians, and in 2018, he was elected to the National Academy of Medicine.
Mr. Backenroth has extensive executive experience in financing and building biotechnology companies and most recently comes from Ohr Pharmaceutical. While at Ohr, he was instrumental in the company’s growth from startup to a market capitalization of several hundred million and in moving a product from preclinical into late stage clinical development. He previously worked as an investment banker with The Benchmark Company LLC, an investment banking firm specializing in micro-cap biotechnology transactions. While at Benchmark, Sam helped numerous small biotechnology companies raise equity growth capital through a variety of structures. Mr. Backenroth also acted as an advisor to multiple public and private biotechnology and pharmaceutical companies in assisting with business development activities, joint ventures, licensing, strategic partnerships, and mergers & acquisitions. He graduated with honors from Touro College with a BS in finance.
Dr. Lorenz is an experienced clinical development professional with 25 years in upper management positions at Johnson & Johnson and Cor Therapeutics, as well as chief medical officer at Orexigen, Catalyst Biosciences and Corgentech. Most recently, as senior vice president of clinical development and scientific licensing at Portola Pharmaceuticals, Dr. Lorenz held primary responsibility for the Phase 3 APEX clinical development program of betrixaban that resulted in FDA approval. Dr. Lorenz is also an advisor to the Stanford University SPARK program and holds a B.A. in Chemistry and an M.D. from Case Western Reserve University, School.
Dr. Zamboni has 25 years of experience in the pharmaceutical and biotechnology industry. His career has been devoted to chemistry drug discovery, which has resulted in many compounds in clinical development and over 114 publications and 28 patents. Previously, he served as Vice President of Research and as a Senior Research Chemist at Merck & Co., Inc. and as an adjunct professor of chemistry at McGill University. Dr. Zamboni is a Member of the American Chemical Society and is the recipient of numerous prestigious awards, including the Prix Galien and the Prix Lionel-Boulet and a Heroes' of Chemistry Award from the American Chemistry Society in recognition of his work in discovering and developing Singulair, for the treatment of asthma. Dr. Zamboni received a BSc in Honors Chemistry and his PhD in Organic Synthesis from McGill University and completed postdoctoral fellowships at the University of Pittsburgh and Yale University.
Board of Directors
Dr. Goldstein brings to the NeuBase board of directors more than two decades of experience in the biopharmaceutical industry as a venture capital investor, an executive and a member of the boards of directors of a number of successful companies including ADMA Biologics (ADMA), Cempra Pharmaceuticals (CEMP), Durata Therapeutics, Esperion Therapeutics (ESPR), and Loxo Oncology (LOXO). The companies where Dr. Goldstein has played a leadership role have resulted in the approval of 2 drugs: Eraxis (anidulafungin) and Dalvance (dalbavancin. Dr. Goldstein was the chief financial officer of Schrödinger, LLC from 2017 to 2018. Dr. Goldstein served as a Managing Partner at Aisling Capital, a healthcare dedicated private investment firm, from 2014 to October 2017, Partner from 2008 to 2014 and a Principal at Aisling Capital from 2006 to 2008. Dr. Goldstein served as the chief financial officer of Loxo Oncology, Inc. between July 2014 and January 2015, and was its acting chief financial officer from January 2015 to May 2015. From 2000 to 2005, Dr. Goldstein served as chief financial officer of Vicuron Pharmaceuticals, Inc., which was acquired by Pfizer, Inc. in September 2005, and helped lead the M&A process. Prior to joining Vicuron, Dr. Goldstein was director of venture analysis at HealthCare Ventures. Dr. Goldstein serves as a director and chairman of the compensation committee of ADMA Biologics, Inc. (Nasdaq: ADMA). He serves as a director and member of the audit committee of Esperion Therapeutics (ESPR).. Dr. Goldstein received a B.S. from Stanford University, an MBA from Columbia Business School and an M.D. from Yale School of Medicine.
Dr. Diego Miralles brings successful drug development expertise at both large and early-stage pharmaceutical companies to the board of NeuBase. Dr. Miralles is the chief executive officer of Vividion Therapeutics, Inc., a biotechnology company with a platform to discover and develop small molecule therapeutics. Prior to Vividion, Dr. Miralles had an extensive career at Johnson & Johnson, culminating in his position as the Global Head of Innovation and a member of the management committee of Janssen. He started his career at Johnson & Johnson at Tibotec BVBA, a leading virology company where he was a member of the management board and was involved in the development and approval of PREZISTA® and INTELENCE®. He was the head of the Janssen Research and Early Development unit in La Jolla, CA. While at Johnson & Johnson, he founded and launched the JLABS incubator for start-up life science entrepreneurs, and was instrumental in developing and launching Johnson & Johnson’s Innovation center model. Prior to Johnson & Johnson, Dr. Miralles held R&D positions at Trimeris, Inc. and Triangle Pharmaceuticals, Inc., and he was an assistant professor at Duke University Medical Center, where he was a bench scientist and an infectious disease physician, with a focus on HIV. Dr. Miralles is currently an adjunct professor in the Department of Pharmacology at the University of California San Diego. He received his M.D. degree from the Universidad de Buenos Aires, Argentina, completed his internal medicine residency at the Mayo Clinic and was a fellow in Infectious Diseases at Cornell University-New York Hospital.
Dr. Franklyn Prendergast brings decades of significant experience to Neubase, in addition to his scientific expertise in peptide nucleic acids, membrane biochemistry and oncology. Currently, Dr. Prendergast is the chairman of the board of directors of the Infectious Disease Research Institute and serves on the boards of several start-up companies. Previously, Dr. Prendergast was a member of the board of directors of Eli Lilly & Co. from 1995 to 2017 while simultaneously serving as a director of the Mayo Clinic Cancer Center. He has also held several additional positions at the Mayo Clinic, including as a member of the board of governors and the board of trustees, as the director of the Mayo Clinic Center for Individualized Medicine and as the Emeritus Edmond and Marion Guggenheim Professor of Biochemistry and Molecular Biology and an Emeritus Professor of Molecular Pharmacology and Experimental Therapeutics at the Mayo Medical School. He was named a Mayo Clinic Distinguished Investigator in 1989. Throughout his career, Dr. Prendergast has served with the NIH on a number of grant review groups and advisory committees, including as a member of the board of advisors for the Division of Research Grants and on the National Advisory General Medical Sciences Council. In addition to these positions, Dr. Prendergast held a Presidential Commission for service on the National Cancer Advisory Board. Dr. Prendergast obtained his M.D. degree from the University of West Indies and attended Oxford University as a Rhodes Scholar, earning an M.A. degree in Physiology. He obtained his Ph.D. in Biochemistry at the University of Minnesota.
Mr. Richman brings experience in investing in and operating early stage biotech companies. He was involved with the development and launch of several orphan drugs including CytoGam®, Synagis®, Cinryze®, Ethyol®, Respigam®, and others and has decades of experience at PharmAthene, MedImmune and Tyrogenex. Currently, Mr. Richman is the interim chief executive officer of LabConnect, Inc., a provider of global central laboratory services, a position he has held since November 2018. Mr. Richman was previously a venture partner at Brace Pharma Capital, a life science venture capital firm, from January 2016 to September 2018 and is involved with several private and public biotechnology companies. He served as chief executive officer of Tyrogenex Inc., a biopharmaceutical company, from 2016 to 2018. Mr. Richman served as the president and chief executive officer of PharmAthene, Inc., subsequently acquired by Altimmune, Inc., between October 2010 and March 2015. He also served on PharmAthene’s board of directors, when the company was listed on the NYSE, from 2010 to 2017. Prior to joining PharmAthene, Mr. Richman was a member of the founding team of MedImmune, where he held various commercial and strategic positions, including director of international commercialization. In addition, he has served as a member of numerous boards of directors. Mr. Richman currently serves as a director of Adma Biologics, Inc. (Nasdaq: ADMA), including audit, compensation and governance and nominating committees; Variant Pharmaceuticals, Inc.; NovelStem International Corp. (OTCMKTS: NSTM); and LabConnect, Inc. where he serves as the chairman of the board. Mr. Richman has also served as a director of Lev Pharmaceuticals, Inc. and as chairman of its commercialization committee, and served as a director of American Bank Incorporated (acquired by Viropharma). Mr. Richman received a B.S. in Biomedical Science from the Sophie Davis School of Biomedical Education (CUNY Medical School) and a M.B.A. from the American Graduate School of International Management.
Scientific Advisory Board
Dr. George Church is a pioneer of genome engineering as well as systems and synthetic biology. He brings significant expertise both in genetics and the biotech industry to the NeuBase SAB. He has cofounded more than 14 biotech companies, authored over 400 papers and holds 60 patents in his name. Dr. Church is the Robert Winthrop Professor of Genetics at Harvard Medical School, a professor of Health Sciences and Technology at Harvard and the Massachusetts Institute of Technology (MIT) and a core faculty member of the Wyss Institute. He is the principal investigator of the Center for Genomically Engineered Organs (CGEO), the Harvard/MIT DOE Genomes-to-Life Center, the Lipper Center for Computational Genetics and is a founding core member of the Wyss Institute for Biologically Inspired Engineering. Dr. Church is a member of the National Academy of Sciences (2011) and Engineering (2012) and has received the Franklin Institute’s Bower Award for Achievement in Science (2011). He holds a Ph.D. in biochemistry and molecular biology from Harvard University.
Mr. Randall Davis brings significant expertise in molecular biology and DNA sequencing to the NeuBase SAB. He cofounded Genia Technologies and served as vice president of research and development, helping to build the company’s single molecule DNA sequencer. Today, Mr. Davis holds 20 issued or pending patents in molecular detection and DNA sequencing. He has served as manager of the DNA Array and Molecular Analysis Core Labs at the UCSF Comprehensive Cancer Center and has over 20 years of experience in various technical, marketing and sales roles in the semiconductor industry. Mr. Davis holds a B.S. and M.S. in molecular biology from California State University East Bay.
Dr. Robert Friedlander, who serves as NeuBase’s consulting chief medical officer in addition to his role on the SAB, is a renowned neurologist. He served as the fourth Chairman of the Department of Neurological Surgeons at the University of Pittsburgh School of Medicine and the University of Pittsburgh Medical Center. Prior to joining the department, Dr. Friedlander was professor of neurosurgery at Harvard Medical School and vice-chairman of neurosurgery and associate director of cerebrovascular surgery at Brigham and Women’s Hospital in Boston. Presently, Dr. Friedlander is the Walter E. Dandy Professor of Neurosurgery, Neurology and Neurobiology, and co-director of the UPMC Neurological Institute. His work has been published in many top tier academic journals and he has been recognized through many academic awards. In 2006, he was elected as a member of the prestigious American Society for Clinical Investigation. Dr. Friedlander holds a B.A. and M.A. in biochemistry from Brandeis University and his M.D. from Harvard Medical School.
Dr. Charles Thornton is a neurologist specializing in neurogenetic disorders, including myotonic dystrophy. His research focuses on antisense approaches to treating these disorders. Dr. Thornton is a professor of neurology at the University of Rochester Medical Center as well as a practicing neurologist. Dr. Thornton received his B.S. and M.D. from the University of Iowa and completed postdoctoral training and residency in experimental therapeutics and neuromuscular disease at the University of Rochester School of Medicine.