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FDA's written INTERACT feedback (following the July 16 meeting) noted our nonclinical data support a proposed early, exploratory clinical study, recommended plasma lyso-Gb3 as the primary pharmacodynamic readout, endorsed at least two years of eGFR follow-up, and confirmed CMC knowledge (vector backbone, packaging system, process steps) can be leveraged across Glafabra's Fabry, Pompe, and Gaucher programs with appropriate justification.
That CMC point matters beyond Fabry: manufacturing investment in GT-GLA-S03 has direct read-through to GT-GAA-S04 (Pompe) and GT-GBA1-S05 (Gaucher), rather than three separate manufacturing builds.
INTERACT feedback is preliminary and non-binding. FDA recommended a Pre-IND meeting before submission, which Glafabra intends to request; that meeting's outcome isn't yet known.
We're actively in conversation with CDMOs and vector partners who work in autologous lentiviral stem-cell therapy -- reach out if that's you.
Follow the whole series: https://bit.ly/Glafabra-TTW
@Brian Christie @Neva West @Brian Christie @Tetsu Yung
Testing the Waters disclosure: We are considering a capital raise and are "Testing the Waters" under Regulation Crowdfunding. No money or other consideration is being solicited, and any that is sent in response will not be accepted. We cannot accept any offer to buy securities, and no part of the purchase price can be received until an offering statement is filed and only through the BioTech Funding Portal platform. Any indication of interest is non-binding and involves no obligation or commitment of any kind.
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