After Fabry Trial Setback, Avrobio Hopes Rebound With Lysosomal Storage Disorder Data


Avrobio Inc's AVRO gene therapy for a rare lysosomal storage disorder showed potential durability in the first three patients more than one year after infusion.

  • The data comes from a Phase 1/2 trial testing the safety and efficacy of AVR-RD-04 for cystinosis who had previously been given cysteamine, which is a standard treatment that reduces the buildup of cystine crystals in the kidneys for people with the inherited disease.
  • The first three patients on the gene therapy, dubbed AVR-RD-04, remain off oral cysteamine between 12 and 26 months after the treatment infusion. 
  • A fourth patient received the therapy in November 2021. None of the patients have experienced adverse events to date.  
  • In January, the Company axed its Fabry disease gene therapy program.
  • Biopsies of the skin and intestinal mucosa, or the inner lining of the intestinal tract, showed the gene therapy led to one-year reductions in average crystals per cell of 35%, 64%, and 81% in the three patients. 
  • Rectal biopsies showed a 53% reduction in the first patient after 18 months, and the other two patients experienced drops of 28% and 86% after 12 months.
  • The Company expects to initiate the AVROBIO-sponsored trial in 2023 in the U.S.
  • Price Action: AVRO shares are down 1.69% at $1.73 during the market session on the last check Wednesday.
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Posted In: BiotechNewsPenny StocksHealth CareGeneralBriefsFabry diseasegene therapyPhase 1 TrialPhase 2 Trial
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