At NeuBase, we are advancing an entirely new class of medicines to address many currently undruggable diseases. NeuBase's ultra-precision genetic medicines have demonstrated in proof-of-concept preclinical studies treatment tolerability, selective engagement of different disease-driving mutations at the DNA or RNA level, and broad tissue distribution after systemic administration. With PATrOL, we have the potential to address a wide range of diseases, from rare to common.
Most diseases are undruggable with biologic or small molecule approaches, leaving millions of patients with limited options. Precision genetic medicines represent a new class of therapies that target the DNA and RNA mutations that drive disease. Nearly all diseases are caused by genetic changes, underscoring the critical importance of new medicines that can "drug the genome" for the future of health.