NEW YORK – The US Food and Drug Administration has granted ViGeneron permission to begin a clinical trial testing VG801, a gene therapy candidate for Stargardt disease and other retinal dystrophies linked with ABCA4 mutations, the company said Wednesday.
VG801 is the first drug cleared for in-human testing that utilizes mRNA trans-splicing, in which two separate mRNA molecules are joined together, Munich, Germany-based ViGeneron said. That process is the basis of the firm's dual adeno-associated virus REVeRT, or REconstitution Via mRNA Trans-splicing, vector platform, which is useful for delivering genes such as ABCA4 that are too large for standard gene therapy.
In VG801, two vectors each carry half of the ABCA4 coding sequence, producing two pre-mRNAs that are trans-spliced to form a full-length mRNA, which is translated into the functional protein.
Now that the FDA has approved the investigational new drug (IND) application for VG801, the company plans to launch an open-label, dose-escalation Phase I/II trial to evaluate its safety, tolerability, and preliminary efficacy. ViGeneron is also planning to submit a clinical trial application to the European Medicines Agency in the coming months.
Last week, Barcelona-based SpliceBio said it is preparing for a Phase I/II trial of its own Stargardt disease gene therapy, which uses protein splicing to deliver a full-length ABCA4 gene.