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Two months. One platform. Three diseases. Building in public the whole way.
Fabry, Pompe, and Gaucher disease — three diseases the platform is designed to address by correcting one cell type and swapping in the disease-specific gene. An FDA meeting behind us, an IND protocol taking shape, University of Utah Health planned as the clinical site, and a Japan strategy running in parallel from day one.
If you've been here since Day 1 in June — thank you. If September is when you found us — welcome, and thank you for reading two months of a rare disease company's actual work in public.
Reply with the one milestone you're most looking forward to over the next few months, and we'll follow up with you directly when it happens.
Follow the whole series: https://bit.ly/Glafabra-TTW
@Brian Christie @Neva West @Chris Hopkins @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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