Biological barriers like cell membranes and the blood-brain barrier protect the body from harmful infections and disease. However, they also prevent drugs from reaching the right cells in the body, leading to almost 80% of these therapies failing in clinical trials.

This is especially devastating in indications with a high unmet need and low survival rate like treatment-resistant cancers and rare neurodegenerative diseases, both of which have no effective therapies today.

Traction on the Science:
We have developed and tested our microbubble platform, MsQ, in several animal cancer models and with different payloads, using $2.2M+ in academic lab funding at UT Southwestern Medical Center. Our core technology and its use in drug delivery are patented and exclusively licensed to the company.

To summarize, our MsQ microbubbles show >20x improved targeting selectivity over competitor/unmodified microbubbles, thus enabling delivery to only the right cells in the body.

For our oncology pipeline, we have shown that MsQ is able to deliver STING agonists directly into immune cells for cancer-agnostic immunotherapy (https://doi.org/10.1038/s41565-022-01134-z, https://doi.org/10.1002/advs.202416596). MsQ-STING resulted in a 100% complete response rate in a melanoma mouse model vs just 14% with the current clinical standard of care (SOC). It also improved the median survival time of mice with PDL1-negative metastatic triple-negative breast cancer (mTNBC) by 76% when combined with immune checkpoint inhibitors vs SOC. Additionally, we've shown early data delivering siRNA to improve gene knockdown in cancer cells by >10x compared to siRNA alone.

For our neuro pipeline, we’ve previously conducted studies with competitor formulations to validate gene delivery to the brain (https://dx.doi.org/10.3791/61352, https://doi.org/10.1038/s41467-024-48974-y, https://doi.org/10.1002/btm2.70062). Now, we are using MsQ to safely and noninvasively deliver antisense oligonucleotides specifically to the striatum, the brain region involved in the progression of fatal Huntington’s Disease. We have early data demonstrating that our BBB-targeting MsQ microbubbles can bind to brain vasculature unlike competitor/unmodified microbubbles.

Traction on the Business:
We have received letters of support from leading foundations and a prospective pharma partner (Bracco) for our oncology pipeline, 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.

We were selected to apply for a full $3M CPRIT-SEED grant (one of 24 teams out of 164 total applicants) to further develop our STING program.