AVROBIO reports positive clinical data for Gaucher gene therapy; plans Phase 2/3 trial for GD3 in H2 2023

Tectonic Therapeutic, Inc.

GD3 pediatric patient (age 11) shows normalized GCase enzyme and biomarker; remains off ERT/SRT 15 months post AVR-RD-02; no new neurological signs.
Four GD1 patients: liver volume reduced up to 24%, spleen up to 23%; lyso-Gb1 decreased 21%-70% below ERT baseline.
AVROBIO plans global, randomized Phase 2/3 trial for GD3 in H2 2023, ~40 patients, 1:1 gene therapy vs. ERT, following FDA/MHRA feedback.
No drug-related AEs; all AEs related to conditioning or underlying disease, mostly mild/moderate.
plato platform late-stage ready; no major CMC changes anticipated for late-stage trials.

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