Expert Led Breakout Session: Translating an RNA-Targeting Small Molecule from a Hit to a Human Using Novel Preclinical Modeling Strategies to Fast-Track Safe Therapeutics Towards Patients
Time: 2:00 pm
day: Focus Day
Details:
A major challenge for the translation of safe RNA targeting therapeutics is understanding RNA conservation across different animal models. Disease causing RNA sequences can change from animal to animal, therefore, new and innovative approaches need to be developed to enable reliable pre-clinical testing for accelerating safe and efficacious therapeutics towards the patients in need.
During this interactive session, join forces with your peers to discuss different strategies for overcoming this challenge as well as uncovering novel and innovative approaches to preclinical modeling. Here are some questions to think about during this session:
- Are there ways to manipulate animal models to improve their representation of human RNA?
- Which models and screening methods are used to successfully translate a small molecule drug from hit discovery and into the clinic?
- How to ensure the animal models are representative of the mechanism which is occurring in humans?
- What new technologies have been developed to minimize the need to use animal models and more accurately represent humans?
- What are the opportunities and challenges of organoid and organ-on-chip models for advancing RNA-targeting drugs?
