Category | Parameters |
---|---|
Structure/Identity |
Oligonucleotide sequence Backbone structure Other modifications Purity of test article achieved |
In vitro activity |
Treatment effect size on mRNA expression Treatment effect size on protein expression Dose response relationship for the above Selectivity/off-target effect to related target Selectivity /Broad panel |
In vivo activity (such as target engagement/proximal downstream effect or efficacy) Indicate dose and route of administration |
Concentrations of testing agent in the serum and target tissue Brain penetration Serum half-life, AUC or other pharmacokinetic properties Optimize Dose Selection and Schedule ED50, Minimal effective dose, and Optimal effective dose Treatment duration optimization Treatment window optimization Interactions with standard of care Selectivity/off-target effect in vivo |
Safety |
Tolerability Antibodies to product Tumorigenesis Assess host immune response Tissue Cross-Reactivity Local reactogenicity Cardiovascular, respiratory, renal and CNS safety pharmacology studies if any Genotoxicity |
CMC |
Process development status Analytical Assays for release testing with specifications Scale-up feasibility Formulation needs/development Stability Analysis Potency assays (in vitro/in vivo) Delivery devices |
GLP analytical method development/validation |
Potency assays, assays for purity, pharmacokinetics and pharmacodynamics etc |
Interactions with regulatory agencies |
Pre-IND |
Patent protection? |
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