The sequence of DNA holds the instructions to assemble proteins that accomplish each cell's functions. When a genetic mutation in DNA disrupts the normal production of proteins, disease occurs. Since the sequencing of the human genome more than two decades ago, the underlying genetic mutations, both inherited and acquired, have been identified for thousands of disorders, laying the foundation to treat disease at the base cause: the altered sequence of DNA bases.
NeuBase has developed its PNA-enabled platform to overcome the hurdles of delivery, tolerability, selectivity, manufacturability, durability, and scalability that limit other genetic medicine technologies.
to enter cell nuclei and the deep brain after systemic administration
to be available for millions of patients who currently have no treatment options
to bind single- or double-stranded DNA or RNA with ultra-high affinity and single-base selectivity for differentiation between disease-causing and healthy alleles
to be produced using standard peptide synthesis
to be stable and non-immunogenic for chronic use and a durable effect
to address the different mechanisms underlying a wide range of diseases
In preclinical studies, NeuBase has successfully validated the platform's ability to efficiently deliver genetic medicines with broad tissue distribution, including into the deep brain, and precisely engage mutant genes in a manner that is well-tolerated with the potential for sustained efficacy.
Our preclinical studies have shown we can therapeutically modulate in vivo gene function with precise target engagement in three different disease indications. To date, we have used our platform to design drug candidates to inhibit DNA transcription, RNA translation, and mutant protein production, as well as displace bound proteins to rescue mis-splicing. Today NeuBase is developing nuclease-free “stealth editing” technology that has been shown in preclinical studies to operate with ultra-high fidelity, which improves the potential for safe in vivo gene editing in the clinic. Stealth Editors™ do not appear to trigger an immune response, thus enabling re-dosing to potentially address tissue turnover for durable therapeutic effects.