AVROBIO reports positive Phase 1/2 gene therapy data for cystinosis; all patients off oral cysteamine up to 36 months

Tectonic Therapeutic, Inc.

All six patients remain off oral cysteamine up to 36 months post gene therapy.
Durable reduction in leukocyte cystine levels; tissue crystals declined below baseline in first 4 patients.
Neurocognitive tests (Beery VMI) show improvement/stabilization up to 36 months, suggesting CNS impact.
No drug-related adverse events or serious adverse events reported to date.
Positive regulatory feedback from FDA and MHRA to align on registration-enabling trial path.

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