Our Company

Abeona Therapeutics Inc. (NASDAQ: ABEO) is a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases.

Developing therapies for rare disease requires new approaches and strong collaboration between researchers, industry, regulators and patient groups.

Abeona was forged from the company’s close collaborations with key stakeholders all dedicated to transforming new biotechnology insights into breakthrough treatments for rare diseases.

 

Our Mission

Advancing treatments for rare diseases…Working together to find a cure.

cureA rare disease is one that affects fewer than 200,000 people in the United States.  There are nearly 7,000 rare diseases, which may involve chronic illness, disability, and often, premature death.  More than 25 million Americans and 30 million Europeans have one.

While rare diseases can affect any age group, about 50% of people affected are children (15 million); and rare diseases account for 35% of deaths in the first year of life.

These rare diseases are often poorly diagnosed, very complex, and have no treatment or not very effective treatment—over 95% of rare diseases do not have a single FDA or EMA approved drug treatment.  However, most rare diseases are often caused by changes in genes—80% are genetic in origin and can present at any stage of life.

We believe emerging insights in genetics and advances in biotechnology, as well as new approaches and collaboration between researchers, industry, regulators and patient groups, provide significant opportunities to develop breakthrough treatments for rare diseases.

 

Product Pipeline

Abeona Therapeutics is working on a number of exciting technologies and products aimed at curing various rare diseases. Find out what we’re working on and what we have planned.
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Gene Therapy

In many disorders, particularly genetic diseases caused by a single genetic defect, gene therapy aims to treat a disease by delivering the correct copy of DNA into a patient’s cells.
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Plasma-Based Therapeutics

Abeona Therapeutics’ proprietary Salt Diafiltration Process™ (SDF) focuses on ethanol-free extraction of therapeutic biologics from human plasma.
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Management

Tim Miller, PhD Tim Miller, PhD became our President and Chief Executive Officer and Director on May 15, 2015. Dr. Miller was President & CEO of Abeona Therapeutics LLC from 2013 to 2015. He has 16 years of scientific research, product development, regulatory and clinical operations expertise, with a focus on transitioning novel biotherapeutics through pre-clinical phases and into Phase 1 and 2 human clinical trials. Dr. Miller was President & CEO of Red5 Pharmaceuticals from 2013 until 2015 and was Vice President, Business Development of BioEnterprise Inc in 2015. He was Senior Director of Product Development at SironRX Therapeutics from 2010 to 2013. Between 1996 and 2010 Dr. Miller held various positions at several companies focusing on gene therapy and regenerative medicine. Dr. Miller earned his PhD in Pharmacology with a focus on Gene therapy/Cystic Fibrosis from Case Western University. He also holds a B.S. in Biology and M.S. in Molecular Biology from John Carroll University (Cleveland, OH).

Jeffrey B. Davis became a director in March 2006. Since January 19, 2015, Mr. Davis is our Chief Operating Officer. Mr. Davis was our Chief Executive Officer from December 26, 2007 until September 19, 2014. Mr. Davis became Acting Chief Financial Officer, Treasurer and Secretary on November 1, 2013 through September 19, 2014. Previously, Mr. Davis served in a variety of senior investment banking and management positions, and in senior management at a publicly traded healthcare technology company. Prior to that, Mr. Davis was an investment banker with various Deutsche Bank banking organizations, both in the U.S. and Europe. Mr. Davis also served in senior marketing and product management positions at AT&T Bell Laboratories, where he was also a member of the technical staff, and at Philips Medical Systems North America.  Mr. Davis is currently on the board of Uluru, Inc., a public biotechnology company.  Mr. Davis holds a B.S. in biomedical engineering from Boston University and an M.B.A. degree from the Wharton School, University of Pennsylvania. 

Stephen B. Thompson, the Company’s Vice President Finance, became the Chief Accounting Officer, Secretary and Treasurer on January 1, 2015. Mr. Thompson consulted with the Company from December 1, 2013 through December 31, 2014. Prior to December 1, 2013 Mr. Thompson was our Vice President from 2000 and our Chief Financial Officer from 1996. From 1990 to 1996, he was Controller and Administration Manager of Access Pharmaceuticals, Inc., a private Texas corporation. Previously, from 1989 to 1990, Mr. Thompson was Controller of Robert E. Woolley, Inc., a hotel real estate company where he was responsible for accounting, finances and investor relations. From 1985 to 1989, he was Controller of OKC Limited Partnership, an oil and gas company, where he was responsible for accounting, finances and SEC reporting. Between 1975 and 1985 he held various accounting and finance positions with Santa Fe International Corporation.

David P. Nowotnik, PhD is our Senior Vice President Research and Development. He has served the company as Vice President and Senior Vice President since November 1998. From November 2013 until December 2014 he was a consultant to the Company. From 1994 until 1998, Dr. Nowotnik had been with Guilford Pharmaceuticals, Inc. in the position of Senior Director, Product Development and was responsible for a team of scientists developing polymeric controlled-release drug delivery systems. From 1988 to 1994 he was with Bristol-Myers Squibb researching and developing technetium radiopharmaceuticals and MRI contrast agents. From 1977 to 1988 he was with Amersham International leading the project which resulted in the discovery and development of Ceretec, a brain perfusion diagnostic agent.
Kaye Spratt, PhD , Vice President, Regulatory & Quality Assurance, joined our company on November 1, 2015. Dr. Spratt has experience defining nonclinical development plans and strategies to US regulatory authorities. She has more than 20 years of scientific research, product development and translational expertise, with a focus on transitioning novel biologics through nonclinical and translation into Phase 1/2 human clinical trials. Dr. Spratt was Director of Preclinical and Quality Control at Sangamo BioSciences with increasing responsibilities from 1997 until 2015 and was responsible for leading a team of scientists developing novel cell and gene therapy products utilizing the Sangamo Zinc Finger Therapeutic strategy platform. She also has experience in the non-GLP and GLP toxicology and nonclinical safety evaluation of novel biologics and products for product development. Her primary biotech experience has focused on the development of non-viral and viral-mediated gene therapy approaches for monogenic and infectious diseases. Prior to joining Sangamo, Dr. Spratt was a Senior Scientist/Project Leader at Somatix Therapy Corporation. Dr. Spratt has over 40 publications in the Gene Therapy. Dr. Spratt earned her PhD in Microbiology with a focus in molecular biology in infectious diseases from Meharry Medical College. She also holds a B.S. in Biology from Langston University (Langston, OK).
Christine Berni-Silverstein , Vice President, Investor Relations, Vice President, Investor Relations, joined the company in May of 2016. Prior to her current position, Silverstein was a Director of Investor Relations at Relmada Therapeutics, Inc. a clinical stage, publicly traded specialty pharmaceutical company. Additionally, she served as a Managing Director at SCO Financial Group, a New York based corporate advisory firm and held senior positions at both The Investor Relations Group and Corporate Profile LLC, two boutique investor and public relations firms, where she consulted C-level management on capital markets strategies, media, fundraising initiatives, and regulatory issues. Silverstein brings with her over 15 years of experience in financial and business matters across a wide range of industries, including healthcare. Furthermore, she began her career in the financial services industry as a financial advisor for Royal Alliance Associates. Christine holds a B.S. from the Peter Tobin College of Business, St. John’s University.
Michelle Berg joined the company in June of 2015, serving as Vice President, Patient Advocacy. Previously, she was the first hire with Aldevron, LLC now a leading global contract manufacturing organization specializing in biological reagents for R&D through clinical trial use. During her time there, she held multiple positions with increasing responsibility, contributing to the growth and reach of the organization, focused on developing strong relationships, collaborations, and services. Additionally, she performed research on behalf of the Department of Plant Sciences, North Dakota State University. Her B.S. in Biotechnology was attained from North Dakota State University in 1997. She brings over 17 years of experience in the gene and cell therapy, vaccine, gene editing and molecular diagnostic fields.

Board of Directors

Steven H. Rouhandeh became our Executive Chairman, Principal Executive Officer, on January 1, 2015. Mr. Rouhandeh has been a director and Chairman of the Board since March 4, 2008. He has been Chief Investment Officer of SCO Capital Partners, a group of New York based life sciences funds since 1997. Mr. Rouhandeh possesses a diverse background in financial services that includes experience in asset management, corporate finance, investment banking and law. He has been active throughout recent years as an executive in venture capital and as a founder of several companies in the biotech field. His experience also includes positions as Managing Director of a private equity group at Metzler Bank, a private European investment firm and Vice President, Investment Banking at Deutsche Bank.  Mr. Rouhandeh was also a corporate attorney at New York City-based Cravath, Swaine & Moore. Mr. Rouhandeh holds a J.D., from Harvard Law School, Harvard University and B.A. Political Science, from Southern Illinois University.

Todd Wider, M.D. is a surgeon. Dr. Wider joined our board May 7, 2015. Dr. Wider has served as consultant to numerous entities in the biotechnology space. Dr. Wider holds a M.D. from Columbia College of Physicians and B.A. from Princeton University.

Stephen B. Howell, MD has served as our director since 1996. Dr. Howell is a member of the Compensation Committee of the Board. Dr. Howell is a Distinguished Professor of Medicine at the Moore Cancer Center at the University of California, San Diego where he has been since 1977. He is the Co-Leader of the Solid Tumor Therapeutics Program and the Center for Engineering in Cancer and Director of the Cancer Therapeutics Training Program. Dr. Howell is a recipient of the Milken Foundation prize and was elected as a Fellow of the American Association for the Advancement of Science based on his contributions to the field of cancer therapy. He has served on the National Research Council of the American Cancer Society and on the editorial boards of multiple medical journals. Dr. Howell founded DepoTech, Inc. and served as a member of its board of directors from 1989 to 1999. Dr. Howell served on the board of directors of Matrix Pharmaceuticals from 2000 to 2002. Dr. Howell received his A.B. at the University of Chicago, his M.D. from Harvard Medical School, and subsequent training at the Massachusetts General Hospital, the NIH and the Dana Farber Cancer Institute.

Mark J. Alvino became a director in March 2006 initially as a designee of SCO Capital Partners LLC and is chairman of the Audit Committee. He is no longer a designee of SCO Capital Partners LLC. Mr. Alvino is also a member of the Nominating and Corporate Governance Committee. Mr. Alvino is currently with Hudson Square Capital LLC since October 2014. From 2013 to October 2014 Mr. Alvino was leading the LifeSciences efforts of Bradley Woods, & Co. Ltd. Mr. Alvino was Managing Director for Griffin Securities from 2007 to 2013. Mr. Alvino was Managing Director for SCO Financial Group LLC from 2002 to 2007. Mr. Alvino was a member of the board of directors of MacroChem Corporation from 2007 until February 2009. He previously worked at Feinstein Kean Healthcare, an Ogilvy Public Relations Worldwide Company. There he was Senior Vice President, responsible for managing both investor and corporate communications programs for many private and public companies and acted as senior counsel throughout the agency’s network of offices. Prior to working at FKH, Mr. Alvino served as Vice President of Investor Relations and managed the New York Office of Allen & Caron, Inc., an investor relations agency. His base of clients included medical devices, biotechnology, and e-healthcare companies. Mr. Alvino also spent several years working with Wall Street brokerages including Ladenburg, Thallman & Co. and Martin Simpson & Co.

Scientific Advisory Board

Gene Therapy

Executive Vice President of Research, Sanford Health

Scientist, Children’s Health Research Center

Professor for the Department of Pediatrics, Sanford School of Medicine of the University of South Dakota

Research in the Pearce Lab is focused on understanding the molecular basis of several inherited pediatric neurodegenerative diseases including the infantile, late infantile and juvenile onset forms of Batten disease. Dr. Pearce and his team use mouse and miniature pig models of these rare, fatal diseases to reveal molecular and cellular pathomechanisms, to identify new therapeutic targets and to test new therapeutic approaches.

Brian Kaspar, PhD is Professor, Nationwide Children’s Hospital and Ohio State University Brian K. Kaspar, PhD is a principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and Associate Professor in the Department of Pediatrics and Department of Neuroscience at The Ohio State University College of Medicine. In 2013, Dr. Kaspar was named Fellow of the American Association for the Advancement of Science (AAAS).Dr. Kaspar is the Scientific Founder of Avexis and Milo Biotechnology, two AAV-focused companies focused on developing gene therapies for rare diseases.
Kevin M. Flanigan, MD, is an attending neurologist at Nationwide Children’s and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. Certified by the American Board of Psychiatry and Neurology, Dr. Flanigan is the director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s and a leading expert in neuromuscular disorders. He is also on the physician team for Nationwide Children’s world-class Neuromuscular Disorders program, which provides coordinated care with pediatric experts from more than a dozen specialties.

Dr. Flanigan earned his medical degree from Rush Medical College and completed his internship at the University of Michigan Health System before completing his Neurology residency and fellowship in Neuromuscular Disorders at the Johns Hopkins Hospital, and an additional post-doctoral laboratory fellowship in Human Molecular Biology and Genetics at the University of Utah.

His primary research interest is in the genetic and molecular characterization of inherited neuromuscular diseases, and the development of therapies directed toward these diseases. A major focus of his laboratory concerns genotype/phenotype correlation in, with the intention of increasing our understanding of the pathogenesis in this disease and translating this understanding into improved therapies. Dr. Flanigan is named among the “Best Doctors in America” and joined Nationwide Children’s Hospital in October 2009.

Maria Escolar, MD, MS is Director, Program for the Study of Neurodevelopment in Rare Disorders (NDRD) and Associate Professor, Pediatrics, University of Pittsburgh School of Medicine. The NDRD was established in 2002 because of the need of help children and their families understand the overall impact of rare neurological diseases in child development. Dr. Escolar is a graduate of the Escuela Colombiana de Medicina. She has a master’s of science in human nutrition from Columbia University and completed a residency in general pediatrics and fellowship in child development and behavior at Cornell University Medical Center in 1995. Dr. Escolar is board-certified in neurodevelopmental disabilities. She has 15 years of experience as a practicing clinician and researcher. Dr. Escolar has authored multiple original manuscripts, including two New England Journal of Medicine articles. She is nationally and internationally known for her work in neurodevelopment of children with leukodystrophies and mucopolysaccharidosis. Her research focuses on behavioral and neuroimaging outcome measurements.
Tammy Kielian, PhD
Steven Gray, PhD
Erika F. Augustine, MD is a Senior Instructor of Neurology and Pediatrics at the University of Rochester Medical Center. She completed undergraduate studies at Harvard College and received her M.D. degree from the University of Rochester. Dr. Augustine completed Pediatrics and Child Neurology residency training at Children’s Hospital Boston in 2008, followed by fellowship training in Pediatric Movement Disorders and Experimental Therapeutics in 2010 at the University of Rochester. Dr. Augustine’s research interests include methodology of clinical research and experimental therapeutics in rare pediatric neurological disorders. Current work focuses on therapeutics in Juvenile Neuronal Ceroid Lipofuscinosis and on the epidemiology of Tourette Syndrome in minorities. She currently serves as Assistant Program Director for the NINDS-funded Experimental Therapeutics training program at the University of Rochester. She is also a member of the NIH Taskforce on Childhood Motor Disorders and is a consultant to the FDA Neurological Devices Panel.
Jonathon Mink, MD, PhD, is a Pediatric Neurologist who specializes in movement disorders starting in childhood. I was attracted to pediatric neurology when I was a medical student and realized that I had an intellectual interest in the brain but enjoyed the challenges and rewards of caring for children. As a movement disorders specialist, I care for children with a variety of conditions that impair voluntary movements, cause involuntary movements, or both. Such conditions include dystonia, chorea, tics, myoclonus, tremor, stereotypies, parkinsonism, and combinations of these such as may occur in conditions like cerebral palsy. In addition to movement disorders, neurodegenerative diseases are also a focus of my clinical practice.

My research has been focused on understanding brain mechanisms involved in the control of movement or where disorders cause involuntary movements. The research involves neurophysiology, direct measurement of movement abnormalities, rating scales, and longitudinal assessments over the course of development and disease. More recently my work as included clinical trials and studies of factors that impact the function of children with movement disorders.

In addition to my clinical practice and research, I direct the Child Neurology residency at the University of Rochester. I serve on a number of advisory boards including the Tourette Syndrome Association, the Dystonia Medical Research Foundation, the Batten Disease Support and Research Association, the NINDS Board of Scientific Counselors, and the Pediatric Advisory Committee of the FDA. I am also an Associate Editor of Neurology

Plasma-Based Products

Appointed Chairman of the SAB, Zurlo, the inventor of PlasmaTech’s fractionation process, was an executive of Baxter Laboratories, including five years as Sr. Vice-President of its Hyland Division, where he had responsibility for fractionation operations, including development of the process for producing the first commercial AHF concentrate (Hemofil). Subsequently he was Sr. Vice-President of Millipore Corporation, (which developed diafiltration and nanofiltration) and Chief Operating Officer of the New York Blood Center, Inc., when he led the development and licensing of the Solvent-Detergent Method for inactivating lipid-enveloped viruses. He then founded Alpine Biologics Inc., which sourced Recovered Plasma for the Central Laboratory of the Swiss Red Cross and Octapharma Inc. and in return marketed their plasma biologics in the United States. Mr. Zurlo, who is listed on 5 patents as an inventor, is the lead inventor of the Plasma Technologies plasma process technology.
Dr. Heldebrant is the Chief Scientific Officer of PSC Biotech. He has accumulated experience in over 28 years of increasingly responsible and varied positions at Alpha Therapeutic Corporation, successively owned by Abbott, Green Cross, and now Grifols, where he oversaw the development, FDA licensing, and production of the company’s alpha-1 antitrypsin (“AAT”) product which was eventually sold to Baxter. Subsequently he was with National Genetics Institute/LabCorp for over four years. He has extensive experience in biological and pharmaceutical product development, regulatory affairs, quality systems, process design, equipment and process validation, analytical methods validation, and computer validation. Dr. Heldebrant holds 14 patents and is the author or co-author of 31 scientific/technical papers and over 40 extracts.
Robert A. Sandhaus, MD, PhD, FCCP serves as an Executive Vice President and Medical Director at AlphaNet, Inc. Dr. Sandhaus is the Founder of AlphaNet. Dr. Sandhaus is a Professor of Medicine, National Jewish Health at University of Colorado, Denver, CO. He serves as Medical Director of the Alpha-1 Foundation. He serves as Clinical Director of Alpha-1 Foundation. He is an extensive pharma industry expert in pathogenesis of emphysema. He has extensive plasma therapeutics industry experience. He serves as a Director of The Alpha-1 Project, Inc. He serves as a Director of Alpha-1 Program of National Jewish Medical and Research Center, Denver, CO. He serves as Co-Director of University of Colorado Center for Genetic Lung Disease, Denver, CO. He serves as a Director of AlphaNet, Inc. He has been a Member of Scientific Advisory Board at Arriva Pharmaceuticals, Inc. since January 14, 2009. In addition to his ongoing academic career, he worked for a decade in research and development in the biopharmaceutical industry, running clinical development programs at Cortech, NeXstar Pharmaceuticals, and Gilead Sciences. He is an expert in the pathogenesis of emphysema. Dr. Sandhaus completed his Internship of Internal Medicine from Harvard Medical School, Boston, MA. He is a Fellow of Pulmonary and Critical Care Medicine at University of California, San Francisco. He received his bachelor degree in molecular biology from Haverford College. He earned MD and PhD from Stony Brook University, Stony Brook, New York.
Dr. Charlie Strange is a Professor of Pulmonary and Critical Care Medicine at MUSC with faculty appointments in the Department of Medicine and the Department of Graduate Studies. His clinical interests include chronic obstructive pulmonary disease (with specialty interest in alpha-1 antitrypsin deficiency and bronchoscopic lung volume reduction) and interstitial lung disease (with specialty interest in the connective tissue diseases).

Dr. Strange has interest in rare lung diseases and participates in NIH programs to advance their treatment. He is the Principal Investigator for the Alpha-1 Foundation Research Registry, the largest registry of individuals with alpha-1 antitrypsin deficiency or the carrier state in the world. The Registry is used to support research on new therapies, genetic linkage studies, and social sciences research. The Alpha Coded Testing (ACT) study provides confidential testing for alpha-1 antitrypsin deficiency.

Dr. Strange is available for physician consultations on the above research studies and continues active inpatient and outpatient clinical service. For this activity and teaching he has been listed in “Best Doctors in America” 2003-2015 for General Pulmonary and Critical Care Medicine, Interstitial Lung Diseases, and Chronic Obstructive Pulmonary Disease