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DIA/FDA Oligonucleotide-Based Therapeutics Conference
Session 7 Track 2: RNA Activation
Session Chair(s)
Patrik Andersson, PhD
- Principal Scientist, Discovery Safety Specialist
- 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
- Head of Research and Development
- MiNA Therapeutics Limited , United Kingdom
AZD8601, VEGF mRNA for Cardiac Regeneration
Anna Collén, PhD
- Global Project Leader, Early Cardiovascular, Renal and Metabolism
- AstraZeneca, Sweden
Targeted Augmentation of Nuclear Gene Output (TANGO) Technology for Upregulating Target Protein Expression
Meena Meena, PhD
- VP of Bioanalytical, DMPK and Biomarker Development
- Stoke Therapeutics, United States
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