The discovery and availability of therapies for central nervous system (CNS) disorders are severely limited by the blood-brain barrier. This formidable biological obstacle regulates entry of both natural and foreign molecules into the CNS, creating a significant challenge for drug discovery and therapeutic delivery. Fewer than 5 percent of small-molecules drugs pass through this barrier, and biologic therapies such as antibodies and gene therapies are essentially excluded from reaching the CNS. As a result, patients with many neurological diseases such as Alzheimer’s and Parkinson’s cannot benefit from these cutting-edge therapies.
SRI International’s proprietary DiaCyt platform technology identifies peptide-based delivery agents that can selectively transport molecular cargos into the CNS without physically disrupting the blood-brain barrier. These delivery agents, known as molecular transport systems (MTS), can be conjugated to a wide variety of therapeutic molecules including monoclonal antibodies, enzymes, nucleic acids for gene therapy, small-molecule therapeutics, and diagnostic and imaging agents.
SRI research shows that when MTSs are injected intravenously in pre-clinical models, the MTS peptides travel through the blood system. When they reach the target cell, they bind and are then transported across the blood-barrier into the CNS without disrupting the barrier.
“Due largely to challenges presented by the blood-brain barrier, individuals with many CNS diseases and disorders still have very few or no treatment options available to them,” said Kathlynn Brown, senior director of Molecular Sciences at SRI. “DiaCyt could help solve this long-standing delivery problem and thereby open a floodgate of new CNS-targeted therapies.”
The DiaCyt platform is designed to be combined with a therapeutic “cargo.” SRI is currently looking for commercial partners who are interested in the company’s pipeline of MTS-drug candidates and/or have cargo compounds and/or platforms that align well with SRI’s current MTS library. Contact biosciences@sri.com to learn more.