Ribosomal RNAs (rRNAs) form the translation machinery that
generates functional proteins from genetic sequences; modulating
the ribosome subunits provides a therapeutic approach to addressing
a number of diseases.
Although the ribosome provides a rich target, the development of disease-specific ribosome modulators has been a challenge. At Zikani, we’re dedicated to rising to this challenge.
Zikani’s unique proprietary platform (TURBO-ZM™) allows rapid synthesis of novel ribosome modulating agents (RMAs) that can be optimized to target the human ribosome in a disease-specific manner.
Establish TURBO-ZM™ as the preeminent platform for discovering and developing novel molecules that target the ribosome in a disease-specific manner
Develop a clinical stage pipeline of treatments for rare diseases with a high unmet need, beginning with class 1 cystic fibrosis (CF), familial adenomatous polyposis (FAP), APC mutant colon cancer, and recessive dystrophic epidermolysis bullosa (RDEB)
Expand the preclinical pipeline to programs in other rare genetic diseases and rare cancers