CREATE Bio Example: Anti-sense-oligonucleotides Agent Profile Table

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?