Reducing the Burden of In Vitro and In Vivo Studies for IND Submission by Streamlining Oligonucleotide Development to the Clinic
Time: 1:30 pm
day: Conference Day Two
Details:
- Identifying mis-splicing targets with FlexASO technology to recover loss of TDP- 43 in ALS
- Using in silico analysis, chemistry and scaffold of the oligonucleotide to forecast candidate toxicity of test material to avoid unexpected toxicity
- Getting the most out of in vitro studies prior to in vivo studies to give as much credibility as possible for IND submissions to prove safety and efficacy to regulator standards in a more streamlined timeline
