Research Update: Investigational Gene Therapy for Cystinosis

Print Friendly, PDF & Email

Data from the AVROBIO Phase 1/2 investigational gene therapy for cystinosis was released today at WORLDSymposium in San Diego.

“These interim data increase our confidence in the safety and efficacy of our gene therapy approach using the patient’s own hematopoietic stem cells and lay the groundwork for the AVROBIO-sponsored clinical trial for cystinosis planned to begin in 2023.”

Access the full briefing here: