Gene TherapyBreakthrough TherapyOligonucleotideDrug ApprovalClinical Study
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Source: Pharmaceutical Technology
Robert Barrie
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Source: Pharmaceutical Technology
Duchenne Muscular Dystrophy is one of the more prevalent genetic conditions – it affects 1 in every 3,500 male births worldwide. Image credit: Shutterstock/ChiccoDodiFC
The news follows another recent FDA win for NS Pharma, which is owned by Japanese pharmaceutical company Nippon Shinyaku, after its therapy was awarded a rare paediatric disease (RPD) designation earlier in July.
View allCompanies IntelligenceNippon Shinyaku Co LtdNS Pharma, Inc.View all
A first-in-human clinical trial (NCT04129294) in six Japanese patients with DMD amenable to exon 44 skipping showed that the therapy increased dystrophin protein expression, and improved the motor function of patients as measured by The North Star Ambulatory Assessment. NS-089/NCNP-02 is an antisense nucleotide that helps cells skip over a particular exon. In the case of DMD patients, this is the mutated – and therefore dysfunctional – exon 44 in the DMD gene.
Sarepta was recently in the news after the FDA approved the much-anticipated gene therapy Elevidys (delandistrogene moxeparvovec-rokl). The treatment, which joined an exclusive club of the 32 FDA-approved cell and gene therapies, is for children with DMD who have a confirmed mutation in the DMD gene. The Muscular Dystrophy Association hailed the therapy as “ground-breaking”.
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