AVROBIO reports preclinical Pompe gene therapy data; >99% glycogen reduction in heart; trial start 2023

Tectonic Therapeutic, Inc.

Median GAA enzyme activity in bone marrow reached 300-534 nmol/h/mg (p<0.005) vs 4-6 in normal mice eight months post-infusion.
>99% glycogen reduction in heart with reversal of cardiac hypertrophy; >97% reduction in diaphragm, >85% in quadriceps.
>95% glycogen reduction in brain and >99% in spinal cord; improved locomotor function and blood-brain barrier crossing.
Stable engraftment at eight months with median vector copy numbers 1.9-3.6 per diploid genome; no clonal dominance observed.
AVROBIO plans to engage regulators in 2022 and initiate a clinical trial for AVR-RD-03 in 2023.

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