NeuBase Therapeutics, Inc., is accelerating the genetic revolution developing a new class of designer synthetic medicines.
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 genetic diseases (remembering that every disease is genetic), including those that can only be treated through accessing of double-stranded DNA and RNA structures. Using PATrOL™ technology, NeuBase aims to first tackle genetic neurological disorders.
NeuBase is using its proprietary PATrOL™ platform to accelerate the genetic revolution
Senior Management
Dr. Stephan is an industry veteran having had a career in academia and in industry. Stephan served as Deputy Director for Discovery Research and chair of the neurogenomics division at TGen, and served as professor and chairman of the Department of Human Genetics at the University of Pittsburgh/UPMC. He has had academic affiliations with Harvard Medical School, Johns Hopkins University and Children's National Medical Center. Stephan has identified the molecular basis of dozens of rare and common diseases. Stephan has founded or co-founded 14 biotechnology companies to chaperone novel innovations to market with a focus on molecular diagnostics and first-in-class therapeutics. Stephan co-founded Navigenics, Inc. (a pioneer in direct-to consumer genomic testing), was founding Chairman of the Board of Pendulum, Inc. (microbiome modulating therapies), was an early advisor to Guardant Health, Inc. (liquid biopsy), is founding Chairman of the Board Peptilogics, Inc. (deep machine learning to develop peptide therapies), was part of the team that developed Genia Technology, Inc.'s single molecule electrical detection DNA sequencing platform acquired by Roche, and others. Stephan received his B.S. from Carnegie Mellon University, Ph.D. from the University of Pittsburgh and did his fellowship at NHGRI/NIH.
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 B.S. in finance.
Dr. Mann has decades of experience in the biopharma industry. Most recently, he served as the president and chief executive officer of Helsinn Therapeutics (U.S.), Inc. Before joining Helsinn, he held multiple positions at Sapphire Therapeutics, Inc., rising to vice president of corporate development, where he was instrumental in the sale of the company to Helsinn in 2009. Dr. Mann began his professional career at Novartis, where he led a multidisciplinary drug discovery program and served as director of business development. He received both a bachelor's degree and a doctorate in biochemistry from the University of Aberdeen and went on to obtain an MBA from Rutgers University.
Ms. McCarthy is the Chief People Officer (CPO) of Neubase Therapeutics. Her responsibility is to ensure that our people, the foundation of the company, are given the framework and support within which to be maximally effective. This includes ensuring a healthy culture of authenticity, real-time unemotional team-based creative problem solving, growing communication skills and fully aligning teams toward corporate objectives; all of which are critically necessary for ultra-peak performance to maintain the growth trajectory of the business. The Company believes now, move than ever in this difficult period in history, that caring for our team is essential and we have invested to ensure our team can outperform.
Ms. McCarthy’s background is as a former corporate CEO, executive coach, strategic advisor and since 1992. She draws from her extensive studies in psychology, business strategies, systems thinking and high-performance training to create a unique methodology which produces transformational results to enable leaders and teams to make vastly better decisions, faster.
She has designed and facilitated programs on leadership, personal mastery, team learning, organizational effectiveness and coaching for hundreds of executives and companies worldwide. This includes to many of the world's leading companies including Nasa, Google, Amazon, Facebook, Miraval, Wells Fargo, the Federal Reserve, Livermore Labs, Levi’s, The Gap, Bank of America, and Charles Schwab, among others.
As an organizational leader Ms. McCarthy brings a distinctive ability to develop and align the people strategy with the business strategy and brings an innovative and strategic mindset which allows her to develop new and creative ways of working across a diverse talent pool to optimize team performance.
Most recently, Dr. Motesharei was Senior Vice President, Business Development & Corporate Strategy at Akcea Therapeutics, a late-stage development and commercial biopharmaceutical company focused on rare diseases, where he led and executed the regional partnership of Akcea’s marketed products Tegsedi® and Waylivra® with Sobi in Europe and the Middle East. Prior to Akcea, Dr. Motesharei headed Global Licensing & Business Development, Neurology & Immunology (N&I) at EMD Serono, the biopharmaceutical business of Merck KGaA. He was a core member of the N&I Franchise Leadership Team that executed the overall strategy of the $1.8 billion franchise, including product and pipeline development, partnering, regulatory and commercial and marketing decisions. He managed the global licensing team responsible for search and evaluation and transactions across the entire R&D spectrum for the immunology, neurology, allergy, fertility, medical device and global health franchises. Previously, Dr. Motesharei was a member of the management team and investor relations team at Dyax Corporation, a pharmaceutical company focused on development and commercialization of novel biotherapeutics for prevention of hereditary angioedema. He led the business development, alliance management and competitive intelligence functions covering Dyax’s phage display platform as well as pipeline products including Kalbitor® and DX-2930 (now approved as Takhzyro®) that contributed to its approximately $6.5 billion acquisition by Shire. Earlier in his career, he held a series of leadership positions at Genfit Corporation, ActivX Biosciences and Lion Bioscience. He currently serves on the board of Ariana Pharma. Dr. Motesharei received a Ph.D. in organic chemistry from the UCLA and a B.A. in chemistry from Colorado College. He completed his postdoctoral training as an NIH fellow at The Scripps Research Institute.
Dr. Rojas-Caro has broad R&D leadership, executive management and team-building experience in private and public biotech companies and large pharma. She has been directly involved in successful global regulatory submissions, including an FDA and EMA approval and more than 10 investigational new drug (IND) applications. Most recently, she was Chief Medical Officer for Gemini Therapeutics, a company focused on redefining age-related macular degeneration (AMD) and linked disorders with precision medicine. At Gemini, she led development through several milestones, including the company’s first IND and the first cohorts of genetically selected patients dosed with the company’s leading biologic therapeutic. Prior to Gemini, Sandra served as Chief Medical Officer for Aeglea BioTherapeutics (Nasdaq: AGLE), a biotechnology company developing a new generation of enzyme-based therapeutics to treat inborn errors of metabolism (IEM). Prior to Aeglea, she served as Group Vice President of Clinical Research and Development at Synageva BioPharma where she was instrumental in leading the clinical development team that secured the U.S. and E.U. approval of Kanuma® (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency, as well as advancing the clinical development of other IEM programs. Following the acquisition of Synageva by Alexion, Dr. Rojas-Caro served as Vice President and R&D Project Team Leader for the Metabolic Rare Diseases Unit, and she supported the post-merger integration. Earlier in her career, she held roles in clinical and translational research with increasing levels of responsibility at Roche, Array BioPharma and Pfizer, where she was responsible for the design and implementation of early development clinical strategy across a broad range of indications.
Board of Directors
Dr. Stephan is an industry veteran having had a career in academia and in industry. Stephan served as Deputy Director for Discovery Research and chair of the neurogenomics division at TGen, and served as professor and chairman of the Department of Human Genetics at the University of Pittsburgh/UPMC. He has had academic affiliations with Harvard Medical School, Johns Hopkins University and Children's National Medical Center. Stephan has identified the molecular basis of dozens of rare and common diseases. Stephan has founded or co-founded 14 biotechnology companies to chaperone novel innovations to market with a focus on molecular diagnostics and first-in-class therapeutics. Stephan co-founded Navigenics, Inc. (a pioneer in direct-to consumer genomic testing), was founding Chairman of the Board of Pendulum, Inc. (microbiome modulating therapies), was an early advisor to Guardant Health, Inc. (liquid biopsy), is founding Chairman of the Board Peptilogics, Inc. (deep machine learning to develop peptide therapies), was part of the team that developed Genia Technology, Inc.'s single molecule electrical detection DNA sequencing platform acquired by Roche, and others. Stephan received his B.S. from Carnegie Mellon University, Ph.D. from the University of Pittsburgh and did his fellowship at NHGRI/NIH.
Dr. Goldstein has served as a member of our board of directors since July 2019. Dr. Goldstein is currently a private investor. Dr. Goldstein previously was the Chief Financial Officer at Schrödinger, LLC from the fourth quarter of 2017 to the second quarter of 2018. Dr. Goldstein served as a Managing Partner at Aisling Capital, a 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. Prior to joining Vicuron, Dr. Goldstein was Director of Venture Analysis at HealthCare Ventures. Dr. Goldstein also completed an internship in the Department of Medicine at Columbia-Presbyterian Hospital. Dr. Goldstein serves as a director at ADMA Biologics, Inc. (Nasdaq: ADMA). He also previously served as a director of Loxo Oncology, Inc. (Nasdaq: LOXO), Esperion Therapeutics, Inc. (Nasdaq: ESPR), and Cempra, Inc. (which was acquired by Melinta Therapeutics, Inc.). Dr. Goldstein received a B.S. from Stanford University, an M.B.A. from Columbia Business School and an M.D. from Yale School of Medicine.
Gerry McDougall’s passion is to link scientific breakthroughs to clinical application for the benefit of patients and society. For more than 25 years, Mr. McDougall has been the driving force behind large-scale strategic alliances, joint ventures, and industry partnerships across the healthcare industry to advance innovations in precision medicine and cancer. He has spent almost his entire career as a senior partner at PricewaterhouseCoopers (PwC) where he built and led the firm's Global Health Science consulting practice before retiring. He has worked across the entire ecosystem of the healthcare industry and advised an array of Fortune 500 companies, including leading global pharmaceutical companies. Mr. McDougall has been instrumental around the globe in building public-private partnerships to address human health imperatives. These include the creation and maturation of the Translational Genomics Research Institute (TGen), Arizona's renowned bio-cluster; the design and launch of the Multiple Myeloma Research Consortium (MMRC); the strategic plan for the California Institute of Regenerative Medicine (CIRM) and the Country of Luxemburg's biotechnology commercialization ecosystem.
Dr. Franklyn Prendergast has served as a member of our board of directors since July 2019. Dr. Prendergast retired from the Mayo Clinic in 2014 and is currently the Emeritus Edmond and Marion Guggenheim Professor of Biochemistry and Molecular Biology and Emeritus Professor of Molecular Pharmacology and Experimental Therapeutics at Mayo Medical School. At the Mayo Clinic, he served in several capacities, most significantly, as the Director for Research 1989 – 1992, inclusive, Member of the Mayo Clinic Board of Governors and Executive Committee 1991 – 2007, and Member of the Mayo Clinic Board of Trustees from 1991-2009, inclusive. From 1994 to 2006, he served as a director of Mayo Clinic Cancer Center. He also previously held several other teaching positions at the Mayo Medical School from 1975 through 2014. Dr. Prendergast has served for the National Institute of Health on numerous study section review groups; as a charter member of the Board of Advisors for the Division of Research Grants, now the Center for Scientific Review; the National Advisory General Medical Sciences Council; and the Board of Scientific Advisors of the National Cancer Institute. He held a Presidential Commission for service on the National Cancer Advisory Board. Dr. Prendergast also has served in numerous other advisory roles for the National Institute of Health and the National Research Council of the National Academy of Sciences. He is a member of the board of directors of Cancer Genetics, Inc. (Nasdaq: CGIX) and a member of the board’s audit, compensation and nominating committees. He is also a member of the board of directors of Medibio Limited (ASX:MEB) (OTCQB:MDBIF) and the Infectious Disease Research Institute (IDRI), and he previously served on the board of directors of Eli Lilly & Co. from 1995 to 2017 and was a member of the board’s science and technology committee and public policy and compliance committee. Dr. Prendergast obtained his medical degree with honors 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 has served as a member of our board of directors since July 2019. Mr. Richman is CEO of Gain Therapeutics, and 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 also 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. (“PharmAthene”), 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 held various commercial and strategic positions of increasing responsibility over a 12-year period at MedImmune, Inc. from its inception and was Director, International Commercialization at that company. Mr. Richman served as a director of Lev Pharmaceuticals, Inc. (acquired by Viropharma) and as Chairman of its Commercialization Committee and served as a director of American Bank Incorporated (acquired by Congressional Bancshares). Mr. Richman currently serves as a director of Adma Biologics, Inc. (Nasdaq: ADMA) (as well as a member of such board’s audit, compensation and governance and nominating committees), Zyversa Pharmaceuticals, Inc., NovelStem International Corp. (OTCMKTS: NSTM), co-founder and director of InFuse Holdings and LabConnect, Inc. where he serves as the Chairman of the Board. 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.
Advisory Board
Dr. Friedlander is a renowned neurosurgeon. 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. In addition to serving as the CMO of NeuBase, 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, PNAS, JAMA and the New England Journal of Medicine. He was first to demonstrate a functional role of caspases in a neurological disease and first to delay mortality in both ALS and Huntington’s disease mice. 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.
Dr. Church is a pioneer of genome engineering, DNA sequencing and synthetic biology. He brings significant expertise both in genetics and the biotechnology industry to the NeuBase SAB. He has co-founded 24 biotechnology companies, authored over 500 papers and 140 patent publications. Dr. Church is professor of genetics at Harvard Medical School, and professor of health sciences and technology at Massachusetts Institute of Technology and Harvard Medical School. He is the director of the Center for Genomically Engineered Organs (CGEO), the Harvard DOE Technology 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 the National Academy of 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. Randy Davis is a seasoned industry veteran with expertise in the field of biotechnology and was one of the founding members of Genia Technologies, a company developing a next-generation sequencing platform, which was acquired by Roche in 2014. In addition, he has served as a member or investor in half a dozen biotech companies and has authored or co-authored over 20 patent applications. Mr. Davis graduated from Tokyo Electrical University with a degree in electrical engineering and went on to receive an MBA from San Jose State University. In 2002, he continued his education at California State University East Bay, where he received a bachelor's and master's degree in molecular biology before moving on to various industry roles.
Dr. Dowdy is a cancer biologist, specializing in the development of RNA therapeutics and in understanding cell cycle controls. He is a professor of cellular and molecular medicine at the University of California, San Diego, School of Medicine, where his research focuses on the delivery of therapeutics into cells, including the development of targeting and endosomal escape technologies. Dr. Dowdy is also a member of the Board of Directors of the Oligonucleotide Therapeutics Society.
Dr. Ly was born and raised in Cambodia. He received his Bachelor Degree in Chemical Engineering and Minor in Philosophy from Georgia Institute of Technology in 1994. He received his Ph.D in Organic Chemistry in 1998, also from Georgia Institute of Technology under the supervision of Prof. Gary B. Schuster. He did his postdoctoral work at UC Berkeley and at Scripps Research Institute under the supervision of Prof. Peter G. Schultz, 1998–2001. He joined the Department of Chemistry at Carnegie Mellon University in 2001 as an Assistant Professor. Ly's research program lies at the interface of chemistry and biology, with emphasis on the development of molecular tools and reagents to regulate gene expression in mammalian systems. On-going work in the group includes chemical synthesis, DNA recognition, gene regulation and correction, cellular delivery, peptide therapeutics, infectious diseases, unstable repeat disorders, and molecular self-assembly.
Dr. Peter Nielsen is a leading expert in gene targeting, RNA interference and chemical replication and translation and was one of the inventors of PNAs in 1991. He is currently a professor at the University of Copenhagen where his lab focuses on PNAs in regard to drug discovery, gene targeting, antisense principles, cellular and in vivo delivery and administration of biopharmaceuticals. He is the co-author of more than 400 scientific papers and reviews as well as over 20 patents and patent applications, and he serves on the advisory board of four scientific journals. In addition to his esteemed academic career, Dr. Nielsen is the co-founder of two biotech companies in Denmark and is a member of EMBO and the Danish Academy of Technical Sciences. He received his Ph.D. in 1980 from University of Copenhagen.
Dr. Eriks Rozners is a leading expert in the chemistry and biochemistry of nucleic acids and brings his expertise to NeuBase as the Company is optimizing and developing its PATrOL platform. He is a professor and the chairman of the Department of Chemistry at Binghamton University, where his lab focuses on the use of organic chemistry to develop unique model systems and tools for the studies and practical applications of nucleic acid biochemistry. Dr. Rozners received a bachelor's degree in chemical engineering and a doctorate in organic chemistry from Riga Technical University.
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 the 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 B.Sc. in Honors Chemistry and his Ph.D. in Organic Synthesis from McGill University and completed postdoctoral fellowships at the University of Pittsburgh and Yale University.