AVRO - AVROBIO gains after interim data from cystinosis study for gene therapy

AVROBIO (NASDAQ:AVRO) is trading ~11% higher in the pre-market on Wednesday after the company reported data from an ongoing Phase 1/2 clinical trial for AVR-RD-04 in adults with Cystinosis. Cystinosis is a condition characterized by the accumulation of cystine in the cellular components known as lysosomes. It can lead to health issues such as corneal damage and swallowing dysfunction. The current standard of care is cysteamine. In the collaborator-sponsored, the first three patients treated with the investigational gene therapy remained off oral cysteamine with 12- and 26-months of post-treatment follow-ups, the company said. While a fourth patient was treated with AVR-RD-04 in November 2021, none of the patients who received the therapy so far have indicated adverse events (AEs) related to the drug product. Subject to FDA feedback, AVROBIO (AVRO) plans to begin a company-sponsored study for AVR-RD-04 in 2023, starting from the U.S. The results were part of a presentation conducted at

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AVROBIO gains after interim data from cystinosis study for gene therapy