AVRO - Avrobio stock rises as gene therapy for rare disorder shows promise in interim data from trial

Avrobio (NASDAQ:AVRO) said new interim data from a phase 1/2 trial of its gene therapy AVR-RD-04 for cystinosis showed stabilized or improved symptoms related to the disease following treatment. Cystinosis is a rare, genetic disorder in which an amino acid called cystine builds up in different tissues and organs, including the kidneys, liver, pancreas, eyes, muscles, and brain. The phase 1/2 study is evaluating AVR-RD-04 in adult patients diagnosed with the infantile form of cystinosis who previously had been treated with the current standard of care (SOC) cysteamine. The company said key visual motor integration, visual perception and motor coordination measures impacted by cystinosis stabilized or improved after the single dose gene therapy. Data suggested that after the gene therapy, functional cystinosin was produced throughout the body as seen by clinical measures in multiple tissues, including the eyes, skin, gastrointestinal mucosa and neurocognitive system, Avrobio (AVRO) said in a May 17 press release. No

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Avrobio stock rises as gene therapy for rare disorder shows promise in interim data from trial