Getting proteins through the brain’s barriers

Synageva and to-BBB to collaborate on drug delivery for rare CNS-related diseases

Jeffrey Bouley
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LEIDEN, Netherlands—Dutch brain therapeutic delivery companyto-BBB technologies BV and Lexington, Mass.-based Synageva BioPharma Corp.announced in early January that they inked a research collaboration deal underwhich they will jointly evaluate the potential of transporting therapeutic proteinsacross the blood-brain barrier into the central nervous system (CNS).

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

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

The CEO of to-BBB, Willem van Weperen, tells ddn that no dollar amounts about the deal are beingpublicly disclosed, as does a Synagenva spokesperson. However, van Weperennotes that the deal will "reimburse to-BBB for its research efforts." The twocompanies will, he says, be "performing a collaborative preclinical researchproject with to-BBB's brain delivery technology (G-Technology) with anundisclosed protein of Synageva in the rare disease field" for as-yet-undisclosedindications 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 onunmet needs in the rare disease area and have complementary technologies, sothere is "definitely a lot of synergy."

"The fact that both CEOs originate from Genzyme can explainthe passion for both companies to contribute to therapy development forpatients with rare diseases," he continues.

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

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

Looking at the larger strategic picture for his company, vanWeperen tells ddn that for to-BBB, "this12th collaboration within a short time shows that brain delivery is very highon the agenda of pharma and biotech companies. Furthermore, it illustrates themerits of our G-Technology."

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

At the same time, van Weperen says, to-BBB is developingvalue internally by bringing its lead product 2B3-101 for brain cancer into theclinic 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 theendogenous tripeptide glutathione as a targeting ligand.

Jeffrey Bouley

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