- AVROBIO Inc AVRO reported new interim data from the Phase 1/2 gene therapy trial of AVR-RD-04 for cystinosis.
- Cystinosis is a condition characterized by the accumulation of the amino acid cystine (a building block of proteins) within cells.
- Excess cystine damages cells and often forms crystals that can build up and cause many organs and tissue problems.
- AVR-RD-04 genetically modifies patients' hematopoietic stem cells (HSC) to express a functional version of cystinosin, the protein deficient in cystinosis patients.
- Related: After Fabry Trial Setback, Avrobio Hopes Rebound With Lysosomal Storage Disorder Data.
- Preliminary data suggest that post gene therapy, functional cystinosin has been produced throughout the body, as evidenced by clinical measures in multiple tissues.
- No adverse events related to the drug product have been reported.
- Following discontinuation of cysteamine, average handgrip strength remained stable up to 27 months after dosing.
- The first three patients observed a decline in cystine crystals in the skin and gastrointestinal mucosa biopsies.
- Safety data from the five patients dosed indicate no AEs related to the drug product. All AEs were related to myeloablative conditioning, stem cell mobilization, underlying disease, or pre-existing conditions.
- The company expects to initiate an AVROBIO-sponsored trial in 2023 in the U.S.
- AVROBIO's current plan involves a two-part strategy, beginning in a pre-renal transplant population followed by a post-renal transplant population.
- Price Action: AVRO shares are up 3.26% at $0.81 during the market session on the last check Tuesday.
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