Spotlight Therapeutics

In vivo Gene Editing

Undisclosed RD HSC-mediated diseases, hemoglobinopathies; undisclosed RD ophthalmic diseases

Spotlight Therapeutics is developing engineered RiboNucleoProteins (eRNPs) to enhance the delivery of CRISPR-based therapeutics. To unlock the full potential of in vivo gene editing therapies, the biomolecules must be delivered to the correct tissue successfully. Using their eRNP platform, Spotlight is exploiting the high modularity of eRNPs to create fit-for-purpose molecules by adding or substituting protein domains on Cas nucleases to enable specific-functionalities such as adenine or cytosine base editing. To attain specificity, Spotlight is developing bioconjugation tools to fuse eRNPs to cell-targeting moieties such as antibodies enabling cell-selective editing. Initially, Spotlight is applying eRNPs to address ophthalmic diseases.