Reducing the Burden of In Vivo Studies for IND & MAA Submission by Optimizing In Vitro Studies to Streamline Oligonucleotide Development to the Clinic for Ultra Rare Disease
Time: 4:00 pm
day: Conference Day One
Details:
- Exploring the crucial minimum requirements for in vitro and in vivo studies needed for IND and MAA submission, where ultra rare disease patients might have limited treatment window
- Using AI, chemistry and scaffold of the oligonucleotide to lower risk for candidate toxicity
- Assessing dose-response, immune-response, immunogenicity and cell viability to get the most out of in vitro studies to reduce need for in vivo studies and satisfy regulatory requirements
- Reviewing the value of longer-term studies to support chronic clinical dosing
