Biotherapeutic Control Systems.
Intrexonâs technology involves the use of DNA as the natural control modality for the delivery, targeting, activation, regulation and location of biological therapeutics. Our R&D efforts are focused on implementing control systems that significantly improve the safety and efficacy of proven biologics, while enabling new classes of biotherapeutics that can combat the complexity of mutation-based diseases.
Our technology enables key control features:
- Induction — Activation of bio-molecular expression can be timed to occur once a transduced cell is in a target location and/or desired state.
- Level — Amount of expression can be regulated by the dosing of an externally administered activator ligand plus internal auto-regulation.
- Duration — Duration of expression can be limited or stopped by withdrawal of the activator ligand.
- State — Cell type, developmental stage, receptor status, and other state-specific conditions can be used to regulate expression levels.
- Location — Constraint of biologics to predetermined locations to limit or eliminate off-location toxicity effects and enhance efficacy.
- Integration — Stable integration of controllable expression systems into preferred genomic loci for safety and transcriptional viability.
Our control systems are designed to deliver prospective biologics through either ex vivo or in vivo transduction of target cell types. Once positioned in vivo, the biologics can be activated and controlled through dosing of an activator drug (ligand) plus internal auto-regulation.