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Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"MiNA Therapeutics Enters Research Collaboration with Nippon Shinyaku to Develop RNAa Therapeutics Targeting Rare Neurodegenerative Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform"}]
Find Clinical Drug Pipeline Developments & Deals by Nippon Shinyaku
Under the collaboration, MiNA Therapeutics will utilize its proprietary RNAa algorithm and technology platform to identify and characterise RNAa molecules targeting rare genetic diseases of the central nervous system (CNS).
NS-229 is a potent and selective Janus kinase (JAK) 1 inhibitor. It is being evaluated in phase 2 clinical trials for the treatment of eosinophilic granulomatosis with polyangiitis.
NS-089/NCNP-02 (brogidirsen) targets a gene mutation that can be treated by exon 44 skipping. It is being evaluated in phase 2 clinical trials for the treatment of Duchenne Muscular Dystrophy.
ELZONRIS® (tagraxofusp), a recombinant protein is the only approved treatment for patients with blastic plasmacytoid dendritic cell neoplasm, and the first and only approved CD123-targeted therapy, in both the United States and Europe.
NS-018 (ilginatinib), is a highly selective and potent inhibitor of JAK2. It is being investigated for the treatment for myelofibrosis (MF), a rare and incurable blood cancer.
NS-089 (brogidirsen) which is a novel phosphorodiamidate morpholino oligomer that consists of a novel sequence design involving connected antisense oligonucleotides, targeting exon 44, to serve as an effective treatment for DMD patients amenable to exon 44 skipping.
NS-089/NCNP-02 (brogidirsen) which is a novel phosphorodiamidate morpholino oligomer that consists of a novel sequence design involving connected antisense oligonucleotides, targeting exon 44, to serve as an effective treatment for DMD patients amenable to exon 44 skipping.
NS-050/NCNP-03, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 50 skipping therapy. Study assessments will include dystrophin production, muscle strength, mobility and functional exercise capacity.
DFP-10917 (radgocitabine) is a nucleoside analog delivered as a low-dose continuous infusion for the treatment of relapsed/refractory acute myeloid leukemia.
NS-089/NCNP-02 which is a novel phosphorodiamidate morpholino oligomer that consists of a novel sequence design involving connected antisense oligonucleotides, targeting exon 44, to serve as an effective treatment for DMD patients amenable to exon 44 skipping.
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