Pipeline
Our clinical stage program, a gene modified hematopoietic stem and progenitor cell (HSPC) therapy for treatment of Cystinosis, reported positive data in a Phase 1/2 clinical trial and is being advanced by Novartis.
Funding awarded by the California Institute of Regenerative Medicine (CIRM) and NIH to our scientific founders totals over $30 million towards our Cystinosis, Friedreich's ataxia, and Danon disease programs.
Cystinosis Program: Positive Ph1/2 Data
Most patients remain off oral cysteamine up to 36 months post gene therapy
Sustained engraftment and durable reduction in leukocyte cystine levels across all patients
Data show investigational HSC gene therapy durably and systemically impacts neurocognitive measures and reduces cystine levels in the blood, and crystal accumulation in the skin and gastrointestinal mucosa
Safety and tolerability profile remains strong
(May 18, 2023: AVROBIO Announces Positive Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis at the ASGCT 26th Annual Meeting)
