Pipeline and Therapeutic proof of concept
- The platform supports a wide range of therapeutics, including small molecules, gene-silencing agents such as siRNAs and ASOs, and larger biologics such as antibodies, peptides, and other oligonucleotides. In vivo studies show superior efficacy, including the first demonstrated halt of disease progression in a Huntington’s disease mouse model, achieved at low doses with delivery outperforming competing approaches.
- Proof of concept has been established in wild-type and humanized mouse models for ASOs, siRNAs, and proteins, with proof of biology demonstrated in non-human primates for ASOs. OCCT can shuttle both noncovalently and covalently bound cargo to the brain and is applicable across a broad range of neurological indications.
- OCCT has shown a strong safety profile, with no adverse events observed in non-human primates after up to eight weeks of repeat dosing or in mice after up to six months of dosing at ten times the efficacious dose.
