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Challenges and Opportunities in Early Development of RNA Therapeutics
Session Chair(s)
Philip (P.J.) Brooks, PhD
Deputy Director, Division of Rare Diseases Research Innovation
National Center for Advancing Translational Sciences (NCATS), NIH, United States
RNA molecules play a variety of important roles in cells, including structural (ribosomal RNA), enzymatic (small nuclear RNA), and information transfer (messenger RNA). The relatively large size and well-known instability of RNA would make it an unlikely vehicle for drug development. However, in recent years, it has become clear that RNA molecules are an emerging as a class of therapeutics of relevance to multiple diseases, most recently including vaccines. In this session, we will be discussing different examples of RNA based therapeutics, as well as some regulatory considerations.
Learning Objective : Describe different types of RNA therapeutics; Discuss some of the unique challenges involved in the production of RNA therapeutics.
Speaker(s)
Messenger RNAs as Drugs
Philip Santangelo
Georgia Institute of Technology & Emory University School of Medicine, United States
Professor, Coulter Department of Biomedical Engineering
Nonclinical Development of Oligonucleotide Therapeutics
Girish R Chopda, PhD
Dicerna Pharmaceuticals, United States
Associate Director, Nonclinical Development
Regulatory Preclinical Considerations for mRNA Therapeutic Products
Christopher Saeui, PhD
FDA, United States
Biologist, Pharm/Tox/OTAT, CBER
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