eRNP platform designed to be safer and broaden applications

Spotlight’s engineered RiboNucleoProtein (eRNP) platform is designed to unlock the full potential of in vivo gene editing therapies. The high modularity of eRNPs allows the creation of fit-for-purpose molecules by adding or substituting protein domains on Cas nucleases to enable specific functionalities such as adenine or cytosine base editing.

We also are exploring an exciting new class of cell-targeted eRNPs. Taking a page from the antibody-drug conjugate playbook, we have developed bioconjugation tools to fuse eRNPs to cell-targeting moieties such as antibodies and antibody fragments, enabling cell-selective editing.

eRNPs address gene editing delivery challenges in ocular diseases

Spotlight is applying eRNPs to address ophthalmic diseases of high unmet need, including inherited retinal disorders, as well as larger ocular indications. We have demonstrated highly efficient localized in vivo editing of retinal pigmented epithelium (RPE) and photoreceptors via subretinal delivery of eRNPs and eRNP-base editors.

Additional applications for eRNPS in CNS and delivery to any targeted tissue

Studies are underway to explore localized delivery of eRNPs to the CNS to address neurodegenerative diseases. In addition, ongoing engineering efforts are aimed at tuning the stability and PK properties of targeted eRNPs to enable selective delivery to different cell or tissue types and ultimately, to enable systemic administration. Taken together, the eRNP platform holds the promise to vastly broaden the applications and accessibility of gene editing therapeutics.