Once vector-payload candidates have been screened in vitro to assess their main biological properties, including receptor-mediated targeting and endocytosis in relevant cellular models, the most promising candidates are subsequently evaluated in vivo to assess their potential to trigger efficient and specific targeting and delivery of the payload at the right place and with an adequate exposure.
In vivo evaluation of vector-payload candidates can be performed at Vect-Horus through different modalities and most relevant read-outs, depending on their nature as well as their intended application:
- Pharmacokinetics (PK) and biodistribution in vivo studies: re-engineering of a therapeutic or imaging payload should translate into a modified PK and distribution profile, with a higher exposure in the target tissue/organ. In order to assess the efficiency of our targeted delivery approach, in vivo PK and biodistribution studies are performed using different modalities, including ELISA-based bioanalysis, fluorescence live-imaging, PET imaging…
Vect-Horus has also developed a deep and specific expertise in brain/neuro-PK. Beyond the quantification of the vector-payload in the whole brain, deciphering the extent of exposure in different brain compartments is crucial to guide selection of candidates. Thus, our brain PK studies not only include the quantification of candidates in brain microvessels but, most importantly, also in brain extracellular fluid (ECF) as well as brain parenchyma (i.e. neural cells including neurons, microglial cells, astrocytes…).
- Efficacy in vivo studies: Efficacy studies in relevant animal models (CNS diseases, in particular neurodegenerative diseases, cancer models…) can also be performed to evaluate the benefit obtained from vectorization of a pharmacological agent, using histopathological criteria or behavioral, learning and memory tests.