Many neurodegenerative diseases such as Huntington’s Disease are extremely difficult to treat due to the blood-brain barrier, which blocks promising drugs from reaching the brain. Current administration routes are either too invasive, like intracranial injections that require over 12 hours of intensive neurosurgery, or not specific enough, like intrathecal injections that flood the entire brain and cause unwanted side effects in non-diseased brain regions.

Traction on the Science:
Our approach has been well-validated and published in high-impact journals, including Nature Nanotechnology. Compared to competitor microbubbles, MsQ improves targeting selectivity by over 20x in vivo, achieved by increasing cell-specific targeting by 5x while decreasing off-target non-specific delivery by 4x. In a first proof-of-concept study, we used MsQ to deliver STING agonists into antigen-presenting cells for anticancer immunotherapy. MsQ-STING achieved a 100% complete response rate in melanoma mouse models, a 60% complete response rate in ER+ breast cancer mouse models, a 75% increase in median survival time for mice with metastatic triple-negative breast cancer, and improved survival in glioblastoma models. We have also demonstrated delivery of siRNA into cancer cells using MsQ, achieving complete knockdown of a gene of interest in mouse breast tumors. More recently, we have modified MsQ to target the BBB and are now conducting proof-of-concept studies to noninvasively and safely deliver gene therapies like ASOs to the brain using protocols developed by our team, creating new avenues for treating neurodegenerative diseases like Huntington’s.

Traction on the Business:
We have secured over $50k in dilutive and non-dilutive capital so far to advance the platform. We have filed 2 PCT patents on the technology and has exclusively licensed the platform from the home institution. We have received letters of support from leading foundations and a prospective pharma partner (Bracco) for a clinical stage study, and we are currently in active discussions with another partner for a potential R&D collaboration for our neurodegenerative disease pipeline. We have already secured CDMOs, CROs and clinical/regulatory/CMC consultants and advisors to advance to our next stages of therapeutic development.