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Session 7 Track 2: RNA Activation
Session Chair(s)
Patrik Andersson, PhD
Senior Director, RNA Therapeutics Safety
AstraZeneca R&D, Sweden
Imran Khan, PhD
Pharmacologist, OMPT, OND, ODEI, DPP, CDER
FDA, United States
A rapidly evolving area for nucleotide based medicines is upregulation of protein for treatment of a wide range of disease types. This session will cover mechanisms, delivery and some unconventional nonclinical studies for three different platforms. You will learn about two different approaches that induce expression of target mRNA for treatment of cancer and Dravet syndrome as well as therapeutic application of mRNA expressing VEGF for treatment of heart failure.
Learning Objective : At the conclusion of this session, participants should be able to:
- Compare different strategies to treat disease via protein upregulation
- Define key points to consider for nonclinical development of these platforms
- Recognize the need for unconventional nonclinical programs for novel approaches
Speaker(s)
MTL-CEBPA Transcriptional Level Changes in Machinery
Nagy Habib, MD, PhD
MiNA Therapeutics Limited , United Kingdom
Head of Research and Development
AZD8601, VEGF mRNA for Cardiac Regeneration
Anna Collén, PhD
AstraZeneca, Sweden
Global Project Leader, Early Cardiovascular, Renal and Metabolism
Targeted Augmentation of Nuclear Gene Output (TANGO) Technology for Upregulating Target Protein Expression
Meena Meena, PhD
Stoke Therapeutics, United States
SVP of Translational DMPK and Clinical Pharmacology
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