Patient Representative Webinar
Presenter: Daniel O’Reilly and Joseph Ochaba (Oligonucleotide Therapeutics Society)
Date: November 19, 2019
With a constantly growing list of oligonucleotide therapies that are entering the clinics, these exciting research molecules-turned-therapeutic strategy are starting to lead the way in treatments for many devastating diseases. At their core, an Oligonucleotide therapeutic is a short synthetic DNA or RNA that is constructed to target a specific sequence of a gene. Whilst the concept is simple, converting this idea to an effective therapeutic required the development of changes to the properties of oligonucleotides.
We are excited to bring you this webinar series (19th November, 11 AM EST) which will give a brief introduction into the world of these types of medicines, how scientists create them in the lab, and how we can harness these medicines and apply them to treat various genetic diseases. From CRISPR to ASOs and siRNAs, we hope to break down the complex scientific jargon and walk you step-by-step through these exciting tools being used to treat patients around the world. In addition to these educational sessions, we will also discuss the Oligonucleotide Therapeutic Society (OTS) and how the society is fostering collaboration between patient groups and researchers for the development of novel therapeutics.
Recording of the webinar: Click play to view
Daniel O’Reilly is currently a PhD student in Masad Damha’s lab at McGill University in Montreal, Canada. Dan earned his MSc in Chemistry from the University of Southampton, UK working with Jonathan Watts on the synthesis of peptide nucleic acids. His PhD research focuses on utilizing chemical modifications to enhance therapeutic effects of oligonucleotides. A highlight has been probing the structural and chemical requirements for modification of crRNA in the CRISPR-Cas9 system. This work was in collaboration with Keith Gagnon at Southern Illinois University. During his PhD Dan spent two months in David Corey’s lab at University of Texas Southwestern, where he investigated the upregulation of Frataxin protein using antisense oligonucleotides.
Dan’s fascination with the field started at an early age when a family member was diagnosed with Huntington’s Disease, and even more so when he himself discovered that he carried the disease gene. He now volunteers for the Huntington’s Disease Association (UK) research panel, which advises on research grant proposals. Dan is excited to serve as a trainee representative on the OTS Board and to contribute to the OTS community as a whole.
Joseph Ochaba received his BSc in Biology from San Diego State University in 2011 and his PhD in 2016 from the University of California, Irvine working on Huntington’s disease (HD) with Leslie Thompson. His research focused on studying the role that SUMOylation (a post-translational modification pathway) plays in neurodegenerative disorders. He identified a factor within the SUMO-conjugation pathway which may serve as a therapeutic target for the disease. He also identified a novel role for the huntingtin protein in autophagy and how expansion of the poly-Q repeat may impair this function and protein clearance networks in disease settings.
In 2017, he started his current position as postdoctoral fellow at Ionis Pharmaceuticals, where his work has focused on interrogating the role of autophagy in antisense oligonucleotide activity and trafficking in both healthy and disease states. Joseph has a keen interest in developing junior scientists through serving as a mentor for both high school and undergraduates working in the lab. He also has a history of participating in and establishing various community outreach programs to increase scientific awareness and involvement.