Abeona Therapeutics Inc. (NASDAQ: ABEO) is a clinical-stage biopharmaceutical company focused on developing novel 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.
Advancing treatments for rare diseases…Working together to find a cure.
A 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.
His proven track record demonstrates a start-to-finish scope, from early preclinical / proof of concept studies, to manufacturing, regulatory IND and IMPD submissions, clinical trial design and execution, as well as manufacturing and quality control of the adenoviral vectors.
Dr. Ruiz has been instrumental in achieving multiple Orphan Product Designations, has led teams for successful Scientific Advice and Protocol Assistance meetings with the FDA and EMA, and has helped design Patient Reported Outcomes (PRO) in the USA to assess skin outcome measures in patients with Systemic Sclerosis for use as primary endpoints in late-stage clinical trials.
He is the author of over forty peer-reviewed journal articles and book chapters and is an inventor on multiple issued patents.
Dr. Ruiz received his medical training at the University of Navarra, where he also completed a PhD in molecular biology. He was a post-doctoral fellow at the University of Connecticut and earned an MBA the IESE Business School, also at the University of Navarra.
Board of Directors
Scientific Advisory Board
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.
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.