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Getting proteins through the brainís barriers
February 2011
by Jeffrey Bouley  |  Email the author


LEIDEN, NetherlandsóDutch brain therapeutic delivery company to-BBB technologies BV and Lexington, Mass.-based Synageva BioPharma Corp. announced in early January that they inked a research collaboration deal under which they will jointly evaluate the potential of transporting therapeutic proteins across the blood-brain barrier into the central nervous system (CNS).

The companies note that a number of rare diseases which could benefit from protein therapeutics have a CNS component and therefore require that the therapies be transported across the blood-brain barrier. The blood-brain barrier, however, which is a protective filter for the CNS, can be an obstacle for protein therapeutics and prevent such treatments from reaching their site of action within the nervous system.

The companies plan to use their collaboration as the basis for a development program that will explore therapies for multiple rare diseases, among them lysosomal storage diseases (LSDs).

The CEO of to-BBB, Willem van Weperen, tells ddn that no dollar amounts about the deal are being publicly disclosed, as does a Synagenva spokesperson. However, van Weperen notes that the deal will "reimburse to-BBB for its research efforts." The two companies will, he says, be "performing a collaborative preclinical research project with to-BBB's brain delivery technology (G-Technology) with an undisclosed protein of Synageva in the rare disease field" for as-yet-undisclosed indications and diseases.
It's the first time the two companies have worked together, van Weperen notes, but he says that Synageva and to-BBB both have a focus on unmet needs in the rare disease area and have complementary technologies, so there is "definitely a lot of synergy."
"The fact that both CEOs originate from Genzyme can explain the passion for both companies to contribute to therapy development for patients with rare diseases," he continues.

"Synageva is pleased to have entered into this collaboration with to-BBB to evaluate their drug delivery technology," said Anthony Quinn, Synageva's chief medical officer and head of research and development, in a prepared statement. "This collaboration, which combines Synageva's proprietary compounds and technology with to-BBB's expertise in CNS delivery, provides Synageva with an opportunity to extend the therapeutic benefits of our pipeline products targeting rare diseases with CNS manifestations."

"We are very pleased to collaborate with Synageva," added Pieter Gaillard, chief scientific officer of to-BBB, in a prepared statement. "to-BBB's brain delivery technology, combined with Synageva's significant rare disease expertise, should result in substantial progress towards the development of innovative medicines for untreated CNS diseases."

Looking at the larger strategic picture for his company, van Weperen tells ddn that for to-BBB, "this 12th collaboration within a short time shows that brain delivery is very high on the agenda of pharma and biotech companies. Furthermore, it illustrates the merits of our G-Technology."

He adds that his company's delivery technology is potentially safe and flexible toward the formulation of different classes of compounds, adding, "The platform has shown to enhance brain delivery for some small molecules and peptides, but more work needs to be done to explore the possibilities for larger biologics. The partnerships with companies like Synageva will help to-BBB to achieve this. If these collaborations lead to positive data, to-BBB and Synageva could envision discussing a license deal on the G-Technology combined with a Synageva protein as a potential next step."

At the same time, van Weperen says, to-BBB is developing value internally by bringing its lead product 2B3-101 for brain cancer into the clinic in the second quarter of 2011.

G- Technology, to-BBB's proprietary brain delivery platform, combines the established drug delivery approach of pegylated liposomes with the endogenous tripeptide glutathione as a targeting ligand.
Code: E021106



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