Developing Treatment for SCN2A

Webinar 1: ASO treatment for gain-of-function SCN2A

At the 2018 American Epilepsy Society meeting, Prof Petrou presented results from a mouse model of an antisense oligonucleotide (ASO) treatment for SCN2A epilepsies developed by RogCon and Ionis Pharmaceuticals. Praxis Precision Medicines have joined the collaboration to advance that treatment program (RC-222).

In this webinar, Prof Petrou discusses the ASO research in SCN2A as well as talks about what ASOs are and their safety. Dr Kiran Reddy discusses the process of taking an ASO from the laboratory to clinical trials, the steps involved in that process, and how the process can be sped up and facilitated by the SCN2A community.

Professor Steven Petrou, PhD FAHMA, is the Director of the Florey Institute of Neuroscience and Mental Health and Head of the Florey Department at The University of Melbourne. Prof Petrou is a PhD trained neuroscientist who focuses on the disease biology of neurogenetic disorders. His interdisciplinary research includes ion channel biophysics, neurophysiology, animal models of neurogenetic disorders, molecular and cellular biology, computational biology and instrumentation. Prof Petrou is the co-founder and Chief Scientific Officer of US based biotechs, RogCon Inc and Praxis Precision Medicines, companies pioneering breakthroughs in epileptic and neurological as well as psychiatric disorders. Prof Petrou has attracted over $50 million in research support, including $14 million as Chief Investigator on an NHMRC Program Grant.

Dr Kiran Reddy, MD, MBA is President & CEO of Praxis Precision Medicines, Inc a biotech compay focused on developing new therapies for neurologic and psychiatric disorders. Dr. Reddy serves on the business advisory board of the National Epilepsy Foundation, the board of the New England Epilepsy Foundation, and the Council for the National Center for Translational Sciences within NIH. Dr. Reddy holds MD and MBA degrees from Georgetown University. He completed his internship in medicine and his neurology residency at Harvard/Massachusetts General Hospital. He was previously a Howard Hughes science fellow, and has authored several peer-reviewed scientific papers in the field of epilepsy, neuroimmunology, and neurodegenerative diseases.

Transcript:

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