ALS Therapy Development Institute & to-BBB Collaborate on Motor Neuron Disease

The ALS Therapy Development Institute (ALS TDI) recently announced it has entered into a new collaboration with to-BBB to investigate potential treatments for ALS Motor Neuron or Lou Gehrig’s disease).

“This partnership brings a new potential tool to safely enhance the delivery of drugs that may slow or stop the progression of ALS,” said Steve Perrin, PhD, CEO, and CSO of ALS TDI. “Being able to dose a drug systemically, but yet have it cross the blood-brain barrier, allows us to try things we weren’t able to do before. It enhances the arsenal of weapons we have available in our search for effective treatments for ALS.”

“We strongly believe that we can facilitate in creating treatment options for devastating brain diseases faster by providing sustained and enhanced delivery of known compounds to the Central Nervous System (CNS),” said Pieter Gaillard, PhD, Founder and Chief Scientific Officer of to-BBB. “It is a major acknowledgement from ALS TDI to partner with to-BBB in applying such a novel approach to ALS. This collaboration will provide to-BBB with the opportunity to jointly pursue several treatment options for ALS, and we look forward to collaborating with the experts of the Institute.”

Under the terms of the agreement, ALS TDI will use to-BBB’s CNS-targeted liposomal drug delivery system, the G-Technology, as a tool to safely enhance the transport of several different compounds to the brain across the blood-brain barrier. The Institute will screen compounds in a preclinical model of ALS to determine if the enhanced formulation of the proposed treatments have an improved effect on disease course. The collaborators have been working together since early in 2012, designing and completing proof-of-concept experiments confirming that to-BBB’s G-Technology indeed accomplishes sustained and effective delivery of drugs to the CNS in the SOD1 preclinical model of ALS.

The mission of ALS TDI is to develop effective therapeutics that slow or stop ALS as soon as possible for patients today. Focused on meeting this urgent unmet medical need, ALS TDI executes a robust target discovery program, while simultaneously operating the world’s largest efforts to preclinically validate potential therapeutics; including a pipeline of dozens of small molecules, protein biologics, gene therapies, and cell-based constructs. For more information, visit www.als.net.

to-BBBis a clinical-stage biotechnology company focusing on enhanced drug delivery across the blood-brain barrier. 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 widely used 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 at systemically tolerable therapeutic doses. For more information, visit www.toBBB.com.