One technology for precision genetic medicines

HOW NEUBASE'S PATrOL™ PLATFORM ADDRESSES DIFFERENT DISEASE MECHANISMS

The sequence of DNA and RNA hold the instructions to assemble proteins that accomplish each cell's functions. When a genetic mutation in DNA or RNA 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 and RNA bases.

NeuBase's three lead programs highlight the broad potential of the PATrOL™ platform to address different genetic mutations that drive disease.

PATrOL: Peptide-Nucleic Acid Antisense Oligonucleobase Platform

1

New Nucleobases

NeuBase's A, G, T, C, or U are highly selective to binding the target DNA or RNA mutation while minimizing off-target effects.

2

Neutral-Charged Backbone

Compared to negatively-charged sugar phosphate-based backbones, NeuBase's neutral-charged backbone confers high-binding affinity and key drug-like properties.

3

Novel Delivery Shuttles

Proprietary chemistry enables NeuBase therapies to be delivered systemically and achieve broad tissue distribution or be targeted.

The PATrOL Platform

NeuBase has developed its PATrOL platform to overcome the hurdles of delivery, tolerability, selectivity, manufacturability, durability and scalability that limit other genetic medicine technologies.

With PATrOL, NeuBase's ultra-precision genetic medicines are designed to be:

Broad Delivery

Broad Delivery

to enter cell nuclei and the deep brain after systemic administration

Well Tolerated

Well Tolerated

to be available for millions of patients who currently have no treatment options

Highly Selective

Highly Selective

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

Manufacturable

Manufacturable

to be produced using standard peptide synthesis

Enduring

Enduring

to be stable and non-immunogenic for chronic use and a durable effect

Scalable

Scalable

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 DNA and RNA mutations 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 at the DNA or RNA level with precise target engagement in three different disease indications with different genetic mutations driving disease. To date, we have used the PATrOL 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.