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NeuBase Therapeutics, Inc., is a company that is accelerating the genetic revolution by developing a new class of designer synthetic medicines.
The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform enables rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people who are affected by genetic diseases, including people that can only be treated accessing of double-stranded DNA and RNA structures. NeuBase aims to address genetic neurological disorders using PATrOL™ technology,.
The Peptide-nucleic acid Antisense Oligo platform of NeuBase or PATrOL™ platform, uses sequences of engineered peptide-nucleic acids (PNAs), while PNAs of NeuBase are designed to bind mutant genes and inhibit them from manifesting into disease.
The PATrOL platform yields therapeutics with the broad tissue distribution that is the hallmark of small molecules, while maintaining the specific targeting that is characteristic of traditional genetic medicines.
NeuBase develops its PATrOL-enabled therapies using multiple types of nucleic acid bases, that include those that are engineered or natural, enabling increased selectivity and optimization of the therapy for the target disease. NeuBase is the first company to create bifacial Janus bases, engineered bases that target double-stranded DNA by engaging both strands at the same time.
NeuBase has developed a proprietary chemistry to decorate its candidates, which allows the therapies to be delivered systemically and achieve broad tissue distribution. It also possesses programs in place that enable more focused delivery to target tissues when they are advantage for maximizing efficacy.
NeuBase's chemists invented a method that gives its PATrOL-enabled drugs the advantages of small molecule therapies. These PATrOL-enabled drugs have proprietary “linkers” on each end, which allow cooperative binding across the gene target. This self-assembly capability confers the ability to address various mutational types such as large expansions and small single-base mutations.
