Synageva BioPharma & to-BBB to Collaborate on Brain Drug Delivery for Rare Diseases


Synageva BioPharma Corp. and to-BBB technologies BV, the Dutch drug brain delivery company, have recently entered into a research collaboration to evaluate the potential of transporting therapeutic proteins across the blood-brain barrier (BBB) into CNS. The BBB, a protective filter for the CNS, can be an obstacle for protein therapeutics and prevent these treatments from reaching their site of action within the CNS. A number of rare diseases that could benefit from protein therapeutics have a CNS component and require the therapies to be transported across the BBB. This research collaboration will be the basis for a development program for therapies for multiple such rare diseases, including Lysosomal Storage Diseases (LSDs).

“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 R&D. “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, CSO of to-BBB. “to-BBB’s brain delivery technology combined with Synageva’s significant rare disease expertise should result in substantial progress toward the development of innovative medicines for untreated CNS diseases.”

Synageva BioPharma Corp. is dedicated to discovering, developing, and commercializing therapies for patients with rare conditions and high-unmet medical need. The company has developed a pipeline of novel therapeutics for under-served populations and has assembled a team with a proven record of bringing orphan therapies to patients.

to-BBB is a private biotechnology company focusing on enhanced drug delivery across the BBB. The company is developing novel treatments for devastating brain disorders, such as brain cancer, neurodegenerative diseases, and lysosomal storage diseases, by combining existing drugs with the G-Technology, to-BBB’s proprietary brain delivery platform. This technology combines the well-established drug delivery approach of pegylated liposomes with the endogenous tripeptide glutathione as targeting ligand in a novel and safe way. Together with several top tier pharma and biotech companies, to-BBB is investigating the versatility of the G-Technology for drugs that are unable to reach the brain within a tolerable therapeutic window. to-BBB is applying the G-Technology for the delivery of doxorubicin for the treatment of brain cancer as its internal lead product 2B3-101.