Join Connecting Families Urea Cycle Disorders Foundation
as we host a LIVE webinar event
(in collaboration with Arcturus Therapeutics)
for an interactive opportuntiy to learn more about an up-and-coming mRNA therapy for OTC Deficiency
(Ornithine Trans-Carbamylase)
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Arcturus Therapeutics is a leading clinical-stage messenger RNA medicines company focused on the discovery, development and commercialization of therapeutics for rare diseases and vaccines.
Arcturus has a number of mRNA medicine development programs that leverage their proprietary LUNAR® delivery system and mRNA design and production capabilities.
One such wholly owned program is LUNAR-OTC.
To read more, please visit the ArcturusTherapeutics website to learn more about this exciting news: https://arcturusrx.com/rna-medicines/
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You can watch a short video demonstration of the LUNAR® Delivery System here:
Connecting Families is so excited to host the following guest speakers joining us for this LIVE event:
Dr. David E. Geller, MD is the Vice President of Pulmonary and Rare Diseases Clinical Development at Arcturus Therapeutics, a leading clinical-stage messenger RNA medicines and vaccine company focused on the development of novel therapies targeting the root cause of respiratory and rare diseases. Arcturus has extensive experience in the discovery and development of mRNA medicines and vaccines, and design and utilization of lipid-mediated drug delivery systems. Current programs include an mRNA therapeutic for the urea cycle defect, ornithine transcarbamylase (OTC) deficiency, an inhaled mRNA replacement strategy for cystic fibrosis, and a COVID-19 vaccine program.
Dr. Geller received his medical degree from the University of Iowa, and trained in Pediatrics and Pulmonology at the University of California Los Angeles and the University of Arizona, respectively. He spent over 25 years devoted to the care of patients with complex medical problems, educating physicians in training, and running a clinical research unit to investigate novel medicines. His clinical research unit in Orlando consistently enrolled the most individuals for the studies they took on, as his patients shared his passion to find life-changing medicines, reduce their burden, and live more normal lives.
Professor Emeritus of Psychiatry, Pediatrics and Human Genetics at UCLA.
Dr. Steven Cederbaum, MD was born in Brooklyn, NY. He received his BS degree from Amherst College in 1959 majoring in chemistry. It was there that he developed his lifelong interest in biochemistry and genetics. He received his MD degree from New York University School of Medicine in 1964. He did residency training in Internal Medicine at Barnes Hospital/Washington University School of Medicine in St. Louis. He was trained in Medical Genetics at the University of Washington in Seattle following a two year research fellowship in The Laboratory of Biochemistry in the National Heart Institute at NIH. He has been at UCLA for the entirety of his working career.
Dr. Cederbaum has been studying and caring for patients with inborn errors of metabolism for nearly 50 years. His special clinical and laboratory focus is disorders of the urea cycle, particular arginase 1 deficiency, and is a past-president of the Society for Inherited Metabolic Disorders.
Dr. Cederbaum lives in Santa Monica, CA with his wife, with two children, their spouses and four grandchildren nearby. His outside interests include opera, symphonic music, and reading literature and history.
Debra Geary Hook, RDN, PhDc is a doctoral candidate at the University of California, Davis within the Graduate Group of Nutritional Biology. Debra’s focus is in the area of clinical research with respects to measuring diet, using technology to measure diet, and understanding the nutritional needs of patients with inborn errors of metabolism (IEM). She is a pediatric dietitian with 30 years of experience. Twenty-seven of those years were specializing in the care of individuals with IEMs. Debra’s two master degrees which were earned at Loma Linda University: one in nutrition and the other in health education and promotion. Debra utilized both of her passions, nutrition and education, to help build a clinic which provided diagnostic, management, and comprehensive patient education which included in person cooking classes. To meet the needs of a diverse patient population, all materials and classes were provided in 2 to 3 languages. Before returning to school at Davis, Debra worked at several university-based children’s hospitals with her last clinical work at Children’s Hospital Los Angeles.
Debra speaks 3 languages (English, German and French), has lived in Europe, travels internationally, and feels passionate about collaborative work in international and national settings. Debra’s passion for international collaboration is reflected in her work on nutritional databases and increasing the communication between databases and the sharing of information.
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If you would like to attend this event, please click the registration link at the bottom of this page to complete and submit the form. Please be advised that this event will be recored for future post to our website after final edit of this event.
The Q&A portion will not be part of the final edit as to maintain privacy of our members and attendees.