Avrobio Touts New Positive Data From Gaucher Disease Gene Therapy

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  • AVROBIO Inc AVRO announced new interim data from AVR-RD-02, an investigational gene therapy for Gaucher disease.
  • The data showed stabilization or reversal of multiple clinically relevant measures in five patients with Gaucher disease after receiving a single dose of AVR-RD-02.
  • An 11-year-old patient was dosed. Fifteen months post gene therapy, the patient has normalized peripheral glucocerebrosidase (GCase) enzyme activity and plasma chitotriosidase, a marker of activated macrophages, and remains off enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).
  • Related: Avrobio's Pompe Disease Gene Therapy Shows Efficacy, Safety In Preclinical Study.
  • The patient's albumin levels increased by 33% eight months post-gene therapy, reflecting improvements in lymphadenopathy and enteropathy​. 
  • ​Additionally, the patient did not develop any new lesions post-gene therapy.
  • All four adult GD1 patients in the Guard1 trial saw sustained engraftment with vector copy numbers (VCN) between 0.54 - 0.86 per diploid genome 14 weeks to two years post-gene therapy. Reconstitution of GCase enzyme activity in plasma and peripheral blood leukocytes within the normal range.
  • Notably, three of the four dosed patients demonstrate a reduced liver and spleen volume below their ERT baseline. 
  • AVROBIO plans to initiate a global, registrational Phase 2/3 trial in Gaucher disease type 3 (GD3) in 2H of 2023.
  • Price Action: AVRO shares are down 0.90% at $0.83 on the last check Wednesday.
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