biOasis Technologies Ord Shs V.BTI.H

RE:RE:RE:Bigger/Gleitz MPSII Research

[BearDownAZ]

The Bigger/Gleitz research description states "We developed and cloned 6 novel enzyme-peptide fusion IDS genes into our most effective and clinically relevant lentiviral vector." I agree with JD's comment that it is not clear that they have six distinct peptides; instead, they may be referring to different versions/combinations of the IDS enzyme, the linker, and the peptide. And also as JD pointed out, and taken from the BiOasis homepage, "The Transcend Platform consists of a diverse suite of peptide carriers and linkers." So although Mtfp is one peptide, the Transcend platform consists of wider selection of peptides and linkers.

The pdf of the research description is a preliminary study of the 6 differnt enzyme-peptide fusions along with some proof of principle studies. Data in Figure 4 is just from 2 days post-injection of plasmid DNA. The end of the research description describes the longer duration studies that were later completed and were the basis of the recent Gordon Conference and Brains for Brain abstracts.

Yes, Figure 4 of the research description shows that the injection of DNA encoding IDS alone generated roughly the same degree of enzyme activity in the brain as injecting DNA encoding IDS-fusion proteins. However, these were being injected into normal (wild-type) mice that express normal levels of IDS in the brain. So in these studies in Figure 4, what they see is that injection of plasmid DNA encoding IDS alone, or any of the IDS-peptide fusions, more or less fails to elevate brain IDS enzyme activity above baseline control levels (mice injected with just PBS, phosphate-buffered saline). These plasmid DNA injections allow for only transient expression over a few days and are not amendable to long term studies. Basically, that experiment only showed that they couldn't elevate IDS enzyme activity above normal levels. They hypothesized that with longer term treatment with their engineered stem cells in mice deficient in the IDS enzyme, that they would elevate brain IDS activity and correct cognitive defects.

BDAZ