Z005

Z005 is a self-amplifying RNA (saRNA) therapeutic candidate being developed by Ziphius for the treatment of cystic fibrosis. Utilizing Ziphius's proprietary saRNA platform, Z005 is designed to enable the intracellular expression of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, addressing the underlying genetic cause of the disease. Unlike conventional mRNA, saRNA molecules contain the genetic information for their own replication, which can lead to higher and more prolonged protein expression at lower doses. The program is currently in the preclinical stage of development and aims to restore chloride transport in the lungs of patients with cystic fibrosis.