AVROBIO reports positive Phase 1/2 gene therapy data for cystinosis; all 5 patients off oral cysteamine

Tectonic Therapeutic, Inc.

All five dosed patients remain off oral cysteamine post gene therapy; no drug-related adverse events reported.
Early data show stabilization/improvement in visual motor integration, perception, and motor coordination scores (Beery VMI).
Cystine crystals reduced in skin (8-81%) and intestinal mucosa (28-83%) across patients 1-3 post gene therapy.
Photophobia scores improved or stable; patients #1,3,4,5 off cysteamine eye drops.
AVROBIO-sponsored trial planned to begin in 2023 after regulatory interactions.

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