PARAMUS, N.J., Oct. 17, 2023 /PRNewswire/ -- NS Pharma, Inc. announced today the publication of preclinical data on NS-089/NCNP-02 (brogidirsen), an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy, in the journal Molecular Therapy Nucleic Acids. The article, "Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual targeting antisense oligonucleotide," is available under open access here.
NS-089/NCNP-02 is an antisense nucleotide discovered through joint research between NS Pharma's parent company, Nippon Shinyaku, and the National Center of Neurology and Psychiatry (Kodaira City; President, Kazuyuki Nakagome). The article, co-authored with the National Center of Neurology and Psychiatry, describes the process that led to the discovery of NS-089/NCNP-02 and its molecular properties, including nucleic acid sequences that target two separate sites within exon 44 of the dystrophin pre-mRNA sequence. The article also presents in vitro and in vivo preclinical efficacy data regarding dystrophin protein expression.
Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.
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