[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.]
eGFR (estimated glomerular filtration rate) measures kidney function. Kidney involvement (Fabry nephropathy) is one of the main ways the disease progresses. In its INTERACT feedback, FDA recommended at least two years of eGFR follow-up for GT-GLA-S03.
Interpretation: eGFR changes slowly, so a short study can't tell a meaningful trend from normal variation. Two years is the minimum window to see whether the slope of kidney function is changing. Pairing it with lyso-Gb3 gives the trial both a fast biochemical signal and a slower organ-level one.
Uncertainty: Two years is a minimum. It won't answer every long-term question, and the final protocol will be shaped by the Pre-IND meeting, which hasn't happened yet.
Next Monday: what the known risks of lentiviral HSC gene therapy are, stated plainly.
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