CREATE Bio Example: Gene Therapy Agent Profile Table/Reference

The following table represents example categories to consider in describing your current results/status and desired results at the end of the funding period.

Vector backbone, Transgene, Serotype, promoter, reagent purity achieve etc
In vitro activity
Treatment effect size on mRNA, protein expression, and dose response
Selectivity/off-target effect to related target
Selectivity to broader targets
Target tissue/cell biodistribution
and expression/tropism
Short-term distribution
Short-term expression (mRNA/Protein)
Long-term distribution
Long-term expression (mRNA/protein)
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
Antibodies to virus or gene product
T-cell response
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
Patent protection?

Reference to consider when preparing you application and managing your project:

Cheever, Thomas et al.  Perspectives on Best Practices for Gene Therapy Programs.  Hum Gene Ther. 2015 Mar 1; 26(3): 127–133.