Zikit

Zikani Therapeutics was a clinical-stage biopharmaceutical company focused on developing novel small molecule therapies that modulate the ribosome to treat rare diseases caused by nonsense mutations. The company's proprietary TURBO-ZM platform enabled the rapid synthesis and optimization of Macrolide-derived Ribosome Modulators (MRMs) designed to induce translational readthrough of premature termination codons. By targeting the ribosome with high specificity, Zikani aimed to restore the production of full-length, functional proteins in patients with conditions such as Cystic Fibrosis and Duchenne Muscular Dystrophy. The company was acquired by Lexeo Therapeutics in 2021 to integrate its protein restoration expertise with Lexeo's gene therapy pipeline.