AVROBIO Shares Jumps On Encouraging Data From Early-Stage Gene Therapy Study

AVROBIO Inc (NASDAQ: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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