January 13, 2025
The presentation will review our development of a personalized antisense oligonucleotide (ASO) medicine for two children with CLN3 Batten disease. We will detail the collaborative process of designing the ASO therapy, achieving FDA IND approval, and establishing a clinical trial protocol.
Dr. Hastings is the Pfizer Upjohn Professor of Pharmacology at the University of Michigan Medical School, Professor of Medicinal Chemistry in the College of Pharmacy and Director of the RNA Therapeutics Initiative in the Center for RNA Biomedicine. Her research is aimed at understanding the genetic and molecular basis of disease and developing therapeutics that correct faulty gene expression by targeting the RNA transcript using small molecule effectors and antisense oligonucleotides (ASOs). Work in her lab has resulted in the discovery of new treatment approaches for diseases such as cystic fibrosis, Usher syndrome, Alzheimer’s, Parkinson’s, and Batten disease. More recently, she designed, tested and was involved in the development of an ASO medicine that is being used to treat an ultra-rare form of CLN3 Batten disease, and is developing a similar type of ASO to treat the more common cause of the disease.
Yael Shiloh-Malawsky, MD is a Clinical Associate Professor at the University of North Carolina at Chapel Hill, board-certified in child neurology and epilepsy. Clinical and research focus in neurogenetics, with experience as a principal investigator in clinical trials and natural history and biomarker studies. Current roles include serving as the clinical core leader of the UNC Epilepsy NeuroGenetics Initiative (ENGI) and directing the UNC Rett Syndrome Center of Excellence, DRPLA Center of Excellence, and the Batten Disease Center of Excellence Affiliate.
Tiffany Sepp is a passionate executive with a unique background in clinical research,
holding degrees in both Biology and English Literature from the University of California,
San Diego. Before founding Vanguard Clinical, Inc. in 2012, Tiffany gained valuable experience in
clinical research through roles such as Research Coordinator, Clinical Monitor, Clinical
Trial Manager, and Director of Clinical Operations at various CROs, pharmaceutical,
and biotechnology companies. Tiffany’s passion for bringing life-changing treatments to patients and families in need drives her ambition to cultivate the “white glove service” at the core of Vanguard’s offerings. Vanguard is a boutique CRO headquartered in San Diego, CA with a focus on
rare disease and oncology drug development. In her personal life, Tiffany finds joy in travel, sports cars, and collecting wines and first edition literature. She resides in Rancho Santa Fe, CA with her husband and four children ages 7-12.
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