I have a question for Matt, if you are still here. How did you get in touch with Genzyme? What do they have to gain by helping you? Developing a recombinant therapeutic drug is an exceedingly expensive and technically-difficult undertaking. Why are they developing a treatment for a condition for which the potential market size is one single patient?
Your question is valid. I wouldn't expect Genzyme to do much unless there's profit (or at least little cost) involved.
Fortunately, Genzyme might not need to do much, since the enzyme is already in production, but for laboratory rather than human use.
I'll admit I don't know much more will be required to take what they injected into mice to the point where we can inject it into Bertrand.
We're learning on the fly.
Since Genzyme holds the patent, I think we'll at least need their permission to use it in a clinical setting.
We're also actively searching for other NGLY1 patients. There may yet be a market.
We're also investigating other possible clinical applications of N-Glycanase 1. My own early research indicates that it may help in cases of severe jaundice--a much larger market.
I'm optimistic that we can get Genzyme's attention.
