Streamlining Translation & De-risking RNAi Therapeutic Development with Optimal Selection of Preclinical Models & Appropriate Assays to Ensure Tolerable, Potent Therapies
Time: 12:01 pm
day: Pre-Conference Workshop Day
Details:
There are many promising RNAi therapeutic candidates at both the preclinical and clinical phase of development, but few drugs have yet been approved. To maximize clinical success, the right preclinical models should be used, mimicking the genotype of human patients. Assays to determine toxicity and efficacy must be precise, and dosing should be considered. Therefore, this Workshop will address:
- How to derisk novel chemical modifications in in vitro and in vivo preclinical models to safeguard against off-target effects and select animal models which mimic human genotype and pathology
- How to identify assays to determine toxicity, immuno-stimulation and therapeutic potency
- How to plan effective clinical trials considering human dose projections, PK and toxicity
- How to consider IND filing and regulatory approval throughout the translational pipeline
