“Harnessing RNA Editing Technologies to Create Genetic Medicines for Rare Disease”
Presented by: Joseph C Ruiz, PhD
Dr. Joseph Ruiz, President, has over 30 years of experience as an investigator in academia and industry in the areas of embryonic and adult stem cell biology, genome engineering, hepatic cell biology, cell transplantation, and histotechnology. He obtained his PhD at UC San Diego and completed post-doctoral work at Columbia and Harvard Universities. Dr. Ruiz held faculty positions at the Indiana University School of Medicine and at the Wistar Institute and has directed scientific development at several biotech startup companies. In particular, Dr. Ruiz established and directed stem cell and genome engineering research programs at several biotech startup ventures in addition to his efforts at Enzerna (Vesta Therapeutics, Transposagen Biopharmaceuticals and Epigenos Biosciences). Dr. Ruiz also holds an adjunct faculty appointment in the Department of Biology at UNC-Chapel Hill where he is active in mentoring undergraduate and graduate students interested in pursuing careers in the biotech sector.
Enzerna Biosciences is a pre-clinical stage company focused on the development and commercialization of its patented gene therapy approach, called ASRE, for the treatment of rare genetic diseases. ASREs, or Artificial Sequence-specific RNA Endonucleases, are chimeric human-derived proteins consisting of RNA endonuclease (cutting) and RNA binding domains. ASREs can be rationally designed to bind and cut any specific 8-nucleotide RNA target. Our technology is uniquely suited to target genetic diseases, namely nucleotide expansion disorders, that cannot be addressed using state of the art CRISPR/Cas DNA editing technologies. Specifically, our scientific founder has published that ASREs can preferentially destroy pathogenic RNAs, which carry expanded CUG repeats in patients with Myotonic dystrophy and mitigate molecular consequences of the disease in laboratory studies. Our approach will deliver viral vectors coding for therapeutic ASRE proteins to affected tissues to directly destroy pathogenic RNA molecules that underlie the genetic basis of disease. We are developing a portfolio of ASRE based therapeutics to treat nucleotide expansion disorders, which include Huntington’s Disease (HD), Amyotrophic lateral sclerosis (ALS), Friedreich’s Ataxia (FA), Myotonic dystrophy (DM1), and Spinocerebellar ataxia (SCA). Enzerna’s team has the deep experience needed for rare disease drug development, including experience with two previous drug approvals for Duchenne muscular dystrophy.