Preclinical Strategy & Pilot Studies

Start with an in vitro cell model for uncharacterized novel mutations rather than proceeding directly to animal model generation, unless the functional relevance and disease phenotype of the specific alteration are already definitively established. Your strategy depends on three core variables: baseline functional characterization (verifying downstream pathway activation, target expression, or cell viability changes in […]

Choose conventional pharmacological agents if your primary therapeutic endpoint is transient, titration-dependent reduction of intraocular pressure (IOP); choose gene therapy when your mechanism focuses on sustained long-term IOP regulation or direct retinal ganglion cell neuroprotection that standard topicals cannot achieve. Your strategy depends on four critical variables: therapeutic objective (whether targeting aqueous outflow resistance or