PM360 2021 Innovative Startup NeuBase Therapeutics

NeuBase Therapeutics

Dietrich Stephan, PhD, CEO and Chairman

Nearly all diseases are driven by genetic mutations. Thousands of diseases, including certain cancers, are considered “undruggable” with current small molecule and biologic approaches. That’s because these drugs target the proteins that are causing the disease and not the underlying genetic mutations.

Founded in 2018, NeuBase Therapeutics is taking a different approach by “drugging the genome” to treat rare and common diseases. NeuBase is designing genetic medicines to selectively interact with genetic mutations at the DNA or RNA level that do not alter the genome (like CRISPR).

Many genetic medicines have a delivery problem. When dosed systemically they often get filtered by the liver or kidneys, but NeuBase has designed molecules that can be delivered broadly throughout the body after intravenous (IV) or subcutaneous (SC) injection. Furthermore, NeuBase is developing therapeutics that can selectively engage with the genetic mutation driving disease. For example, in Huntington’s disease, a toxic form of the huntingtin protein is made that causes neuronal cell death. However, huntingtin protein is essential for cell function, so inhibiting both the mutant and normal gene could cause issues. But by solving these two major hurdles in genetic medicines through whole-body delivery after IV or SC injection, NeuBase hopes to rapidly advance their research to make meaningful, durable, disease-modifying treatments.

This year, the company made significant advancements in preclinical research and currently has three drug development programs for myotonic dystrophy, type 1 (DM1); Huntington’s disease (HD); and cancers with KRAS mutations. By the end of next year, NeuBase is looking to file an investigational new drug (IND) application with the FDA to begin clinical trials for a first-in-class genetic medicine for the treatment of DM1.


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