Published on 18 May 2022 on Zacks via Yahoo Finance

AVROBIO (AVRO) Stock Up on Upbeat Data From Cystinosis Study

AVROBIO AVRO announced positive interim data from a phase I/II study, which is evaluating the safety and efficacy of AVR-RD-04, an investigational gene-therapyfor cystinosis, a rare but progressive genetic disease.

Initial results from the phase I/II study indicate that following administration of AVR-RD-04, participants produce and distribute functional cystinosin protein throughout the body, thereby preventing the accumulation of cystine crystals. A build-up of cystine crystal can cause progressive organ damage, including shortened lifespan. In fact, data from this study also showed key visual-motor integration, visual perception and motor coordination measures that were impacted by cystinosis to have stabilized and even improved in participants following administration of the gene therapy.

The phase I/II study, sponsored by the University of California San Diego (UCSD), is currently evaluating AVR-RD-04 in adult patients who were diagnosed with the infantile form of cystinosis, previously treated with cysteamine, the current standard of care (SOC) for cystinosis.