NEW HAVEN, Conn.— Bioasis Technologies Inc., a biopharmaceutical company focusing on delivering therapeutics across the blood-brain barrier (BBB) and the treatment of central nervous system (CNS) disorders in areas of high unmet need, has reported the publication of research validating the ability of the company’s xB3 platform to efficiently deliver siRNA across the BBB to the CNS in therapeutically relevant doses.
Scientists from Bioasis and Dr. Wilfred A. Jefferies, professor at the University of British Columbia, evaluated Bioasis’ xB3 platform technology by investigating its application to siRNA brain delivery. This was followed by an efficacy study in an ischemic stroke model, induced by transient middle cerebral artery occlusion. The research shows that the xB3 platform can act as a delivery vector to facilitate BBB translocation of siRNA, where the siRNA targeting NOX4 — a gene thought to be responsible for oxidative stress in ischemic stroke — can elicit effective therapeutic knockdown of a gene in the CNS.
“This study demonstrates for the first time the translocation of a small peptide, nanomule, across the intact blood brain barrier, carrying a payload sufficient to modify a disease in the central nervous system — in this case an siRNA that ameliorates ischemic stroke,” noted Jeffries. “The implications of this discovery are wide ranging, and this platform may provide a general method to intervene in diseases of the brain.”
The successful delivery was demonstrated by reduced stroke damage in the brain, and improved neurological function. The article, “A Nanomule Peptide Carrier Delivers siRNA Across the Intact Blood-Brain Barrier to Attenuate Ischemic Stroke,” has been published in Frontiers in Molecular Biosciences.
The data presented in the publication provide evidence for the utility of xB3 peptide (previously known as MTfpep) as a platform technology for delivery of recombinant and chemically conjugated drug across the BBB. This study demonstrates both siRNA-carrier delivery and therapeutic efficacy in a CNS disease model where the BBB remains intact. These results could offer new avenues for potential siRNA-focused treatments in a variety of neuropathologies.
“The results from this research collaboration further validate the utility of our xB3 platform technology to achieve delivery of therapeutic compounds, including siRNA, across the BBB at levels that may help treat a variety of CNS diseases. This data is consistent with the previous studies where our technology successfully delivered antibodies and enzymes across the BBB with demonstrated efficacy in both the brain and the periphery in settings as diverse as brain cancers, neuropathic pain, and lysosomal storage disorders,” added Dr. Deborah Rathjen, executive chair of the board at Bioasis.