The following table represents example categories to consider in describing your current results/status and desired results at the end of the funding period.
| Category | Parameters |
|---|---|
|
Structure/Identity |
Class and sub-class Light Chain Composition Amino acid (AA) sequence Carbohydrate content AA-sequence confirmed by peptide mapping 3D structure Epitope mapping |
|
In vitro activity |
Target binding: affinity/saturation Bioactivity/potency Stimulation of biological cascade Tissue Selectivity Selectivity/off-target effect to related target |
|
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 |
|
CMC |
Master/Working Virus Bank Status 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? |
|