Technology Platform

Genomic Driven Drug Discovery

miRecule’s™ genomics-based drug discovery platform integrates genomic sequencing, expression, and prognostic data from hundreds of cancer patients with high throughput screening data of microRNA “mimics”. This process allows us to identify the best microRNA candidates for miR replacement therapy. We implement chemical modifications on our lead miR mimics that improve their pharmacokinetic and therapeutic profile. Our mimics are then formulated into tumor-targeted nanoparticles optimized for clinical grade efficacy.

miRecule’s™ core competencies revolve around the know-how of utilizing our bioinformatic process to identify microRNA targets, and our expertise in nucleic acid chemistry. This robust platform approach allows us to repeatedly create lead miR candidates with strong therapeutic rationale.

Our Platform Applied
Head and Neck Squamous Cell Carcinoma (HNSCC)

Head and Neck Squamous Cell Carcinoma (HNSCC) afflicts ~55,000 new patients in the U.S. each year. HNSCCs are highly mutated, resulting in small populations of tumors cells that are often latently resistant to standard drug therapy. In 30-40% of HNSCC cases, this results in the cancer returning in an aggressive, drug resistant state after initial therapy has already been given. The response rate for standard drug therapy in recurrent HNSCC is only 8-18%. 


Applying our platform to HNSCC, miRecule has discovered that miR-30 is widely repressed in HNSCC patients. miRecule has discovered miR-30’s unique ability to regulate  EGFR, the primary mutational driver of HNSCC, as well as MET, IGF-1R, and several other genes that promote drug resistance. miR-30 demonstrates a high correlation with patient survival. In a subset of HNSCC, 100% of patients that retain normal expression of miR-30 survive. 

miRecule has developed MC-30, a chemically-modified mimic of miR-30 formulated into a tumor targeted nanoparticle. MC-30 is currently undergoing efficacy and pre-clinical toxicology studies in animal models.