Many diseases remain undruggable with current therapeutic modalities. Nearly all diseases are driven by genetic mutations. NeuBase is advancing first-in-class precision genetic medicines to “drug the genome” and address disease at its base level to help millions of patients, who currently have no therapeutic options.
NeuBase uses its proprietary PATrOL™ platform to design, develop, and scale precision genetic medicines to target genetic mutations at the DNA or RNA level, with ultra-high affinity and single base selectivity. Our PATrOL-enabled therapies have broad cell and tissue distribution, with the ability to penetrate cell nuclei and the deep brain.
PATrOL Technology Platform
With the ability to engage different genetic mutations, NeuBase has the potential to treat a wide range of diseases. The Company's precision genetic medicine pipeline includes a myotonic dystrophy type 1 program expected to have an IND filed by the end of 2022 and preclinical programs for Huntington's disease and cancer (KRAS G12V and G12D mutations).
Precision Genetic Medicine Pipeline