Avrobio announced that its investigational gene therapy candidate for cystinosis, AVR-RD-04, is cleared to begin a Phase 1/2 clinical trial, following acceptance of the investigational new drug, or IND, application by the FDA. The Phase 1/2 clinical trial is a planned investigator-sponsored trial to be conducted by the University of California, San Diego. AVR-RD-04 is designed as one-time gene therapy and is being investigated for the treatment of cystinosis by inserting the functional gene for human cystinosin, or CTNS, and is designed to maximize the likelihood of sustained CTNS production in patients to correct for the single-gene defect in the CTNS gene that causes cystinosis.
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