[Author Disclosure: This content reflects the views of the author and is provided for informational purposes only. It does not constitute investment advice and is not a recommendation or endorsement by BioTech Funding Portal. Any investment decision should be based solely on the issuer’s official offering materials.]
GT-GAA-S04 (Pompe) is preclinical, with efficacy demonstrated in animal models. GT-GBA1-S05 (Gaucher) is in preclinical development in animal models. Both apply the same Live-cel approach as the Fabry program: a patient's own stem cells, corrected with a working copy of the relevant gene (GAA or GBA1).
Interpretation: The value of the two later programs today is platform leverage: shared vector, process, and regulatory learning from Fabry. See the Sep 25 post.
Uncertainty: Animal-model results often don't translate to humans. Neither program has human data, and each will need its own regulatory path.
Follow the series for updates as each program advances.
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.
Register for FREE to comment or continue reading this article. Already registered? Login here.
0