Bigger/Gleitz MPSII ResearchJust to maybe break us out of the boring and start a discussion.......
The other day I
posted on the upcoming Brains for Brain conference. In that post I mentioned a potential competing peptide-mediated blood brain barrier crossing technology. Fouremm
posted on the Agoracom board some more info about this.
The
description of this research from Dr. Brian Bigger and Ms. Hlne Gleitz sounds like they are chasing a very similar lysosomal storage disease strategy as is BiOasis. The key difference may be that "
[Their] goal is to increase the amount of enzyme produced by stem cells after transplant using gene therapy mediated by lentiviral vectors in a mouse model of MPSII." So they are engineering stem cells to transplant and produce the peptide-enzyme fusion protein. Once transplanted, these cells will act as factories to continually produce more and more of the fusion protein. In contrast, the Scarpa/BiOasis study used peptide-enzyme fusion proteins that were produced in the lab (outside of the body), and injected these into the mice. This latter approach requires repeated fusion protein production and repeated injection into the animal/patient.
The abstracts/research description does not disclose the sequence of the peptides they are using, the receptors (if known) to which they bind, or provide any information that allows for a comparison to Transcendpep. On the surface, I would conclude that this is along the same lines as the Angiopep and Transcendpep strategy of identifying a peptide-mediated approach to cross the blood brain barrier. By using a peptide-mediated approach, all of these technologies are amendable to conjugating the respective peptide to a drug, antibody, enzyme or siRNA.
Has anyone heard any more details about the Bigger/Gleitz research?
BearDownAZ