RE:RE:New paper on MTf-pep This
new paper, presented by GPIAnchor, is important, I think. This morning my interests are divided between the paper and the Spanish Grand Prix. I can't help it. I have chronic nerd disease for which there is only temporary treatment with the ambitious satisfaction of curiosity.
I'm going to have to do some research and thinking about the consequences of the work described in the paper but they could be significant.
First of all, gene therapy can be employed to cause the liver to produce the missing enzymes in at least some Lysosomal Storage Diseases. Such production should eliminate enzyme replacement therapy, the direct injection of the missing enzymes. Of course, the problem is that the normal enzymes produced in this manner can't cross the BBB, leaving the CNS untreated.
And this is where it gets interesting. By the gene therapeutic methods described in this paper, the scientists caused the livers of mice to produce a version of the missing enzyme with the MTf peptide (MTfp) as an integral part of the liver-manufactured enzyme. And it worked! The mouse livers were genetically programmed to produce, basically, xB3-IDUA, which is xB3 on alpha-L-iduronidase, the missing enzyme on MPS I, or Hurler syndrome. The liver-produced MTfp enzyme crossed the mouse BBB and was efficacious.
Good for MTfp, which might mean, "Good for xB3," one might think.
But, it could actually be a big problem. Does this approach work around Bioasis patents? Is that why there is no mention of Bioasis? Is it applicable beyond the delivery LSD enzymes? Is it a threat to Bioasis? Do Bioasis patents cover all uses of the the 12-amino acid peptide, xB3, or is Bioasis going to be left behind with this? Is the actual 12-amino acid peptide structure, and its use thereof, covered sufficiently by Bioasis patents?
I'm not going to pursue this today but I think some questions ought to be fired at Bioasis, Colwell Capital, etc. Answers are needed. Is the genetically programmed production of xB3 drugs in the liver protected by Bioasis patents?
jd