[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.]
In the FACTS trial (5 patients, Canada, NCT02800070): a 48% reduction in plasma lyso-Gb3 vs. no-ERT baseline (p<0.0001) at 5-year follow-up; 4 of 5 patients below the lyso-Gb3 threshold at year 5; all 5 maintained durable enzyme expression; zero product-attributable serious adverse events; polyclonal integration with no clonal dominance.
This shows the approach can produce a durable biomarker response over five real years of follow-up, in the specific population and endpoints measured.
It's five patients, not five hundred, and lyso-Gb3 reduction is a biomarker, not a direct measure of decades-long organ outcomes. The U.S. Phase 1/2 trial is designed to test the approach in a larger, FDA-aligned framework.
Read the underlying publication (PMID 36816757) if you want the full data.
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.
Register for FREE to comment or continue reading this article. Already registered? Login here.
0