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SUMMARY:PULSE Challenge: Properties and challenges with therapeutic oligonucleotides illustrated by hybridization-dependent off-target assessment
DESCRIPTION:This presentation will describe this workflow and discuss considerations for various ONT classes\, emphasizing the importance of ONT-specific factors such as chemistry\, delivery systems\, and tissue distribution in OffT evaluation. It will also cover strategies for experimental verification and risk assessment and discuss the potential of machine learning models to enhance OffT prediction. \n\n\n\nThe seminar series PULSE Challenge is connected to the MSCA co-funded* postdoctoral program SciLifeLab PULSE\, that will train 48 future leaders in life sciences. The program focuses on innovative\, fundamental and translational research carried out in supportive and diverse academic and industrial environments\, preparing postdocs with necessary skills for long-term career sustainability. Click HERE to find out more about SciLifeLab PULSE \n\n\n\n\n\n\n\nOn-line event via Zoom \n\n\n\nRegister Here\n\n\n\n\n\n\n\n\n\n\n\n\n\nPresenter: Dr Patrik Andersson\, PhDSenior Director\, RNA Therapeutics SafetyAstraZeneca\, Gothenburg\, Sweden \n\n\n\nAbstract:Hybridization-dependent off-target (OffT) effects\, occurring when oligonucleotides bind via Watson-Crick-Franklin hybridization to unintended RNA transcripts\, remain a critical safety concern for therapeutic oligonucleotides (ONTs). Despite the importance of OffT assessment of clinical trial ONT candidates\, formal guidelines were for many years lacking\, with only brief mentions in Japanese regulatory documents (2020) and FDA recommendations for HBV (hepatitis B virus) treatments (2022). A subcommittee of the Oligonucleotide Safety Working Group (OSWG)\, was formed to gather experiences and develop updated industry recommendations for assessing OffTs of ONTs. The proposed workflow encompasses five key steps: 1) OffT identification through in silico complementarity prediction and transcriptomics analysis\, 2) focus on cell types with relevant ONT activity\, 3) in vitro verification and margin assessment\, 4) risk assessment based on the OffT biological role\, and 5) management of unavoidable OffTs. \n\n\n\nThis presentation will describe this workflow and discuss considerations for various ONT classes\, emphasizing the importance of ONT-specific factors such as chemistry\, delivery systems\, and tissue distribution in OffT evaluation. It will also cover strategies for experimental verification and risk assessment and discuss the potential of machine learning models to enhance OffT prediction. \n\n\n\nBiography:Patrik joined AstraZeneca in Gothenburg\, Sweden 2004 as “Discovery Toxicologist”\, leading the safety work from target identification to candidate selection for numerous projects. From 2012\, he developed into an expert in the safety of nucleotide-based drugs and their delivery. This includes leading the non-clinical safety work internally at AstraZeneca and in collaborations with world leading companies in the fields of antisense oligonucleotides and therapeutic mRNA. Author of several peer reviewed primary and review papers and book chapters in the area of preclinical safety assessment of oligonucleotide therapeutics and active member in several cross-industry collaboration groups. Most recently active member of the EFPIA/PDEG and IQ/DruSafe oligo working groups and leading the OSWG subcommittee that resulted in a recently accepted paper on revised industry recommendations for off-target safety assessment (DOI: 10.1089/nat.2024.0072)
URL:https://www.scilifelab.se/event/pulse-challenge-properties-and-challenges-with-therapeutic-oligonucleotides-illustrated-by-hybridization-dependent-off-target-assessment/
LOCATION:Online event via Zoom
CATEGORIES:Event
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