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.

*/

Category

Parameters

Structure/Identity

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

Safety

Tolerability
Antibodies to virus or gene product
T-cell response
Tumorigenesis

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-Pre-IND
Pre-IND

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.
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4367233/