AU2019414608A1 — Oligomeric nucleic acid molecule and application thereof
Assigned to Ractigen Therapeutics · Expires 2021-07-22 · 5y expired
What this patent protects
Provided are a small-activating nucleic acid molecule for treating spinal muscular atrophy, and an application thereof. The small-activating nucleic acid molecule comprises a sense nucleic acid strand and an antisense nucleic acid strand, wherein the sense nucleic acid chain and …
USPTO Abstract
Provided are a small-activating nucleic acid molecule for treating spinal muscular atrophy, and an application thereof. The small-activating nucleic acid molecule comprises a sense nucleic acid strand and an antisense nucleic acid strand, wherein the sense nucleic acid chain and the antisense nucleic acid chain are respectively oligonucleotide chains having the length of 16-35 nucleotides, and one of nucleotide chains has at least 75% base homology or complementarity with a target selected from a target-gene SMN2 promoter region. Also provided are a pharmaceutical composition comprising the small-activating nucleic acid molecule and an optional medicinal carrier, and a method for up-regulation of an expression of the target gene in a cell and treatment of a disease caused by underexpression of the target gene by using the small-activating nucleic acid molecule or the pharmaceutical composition comprising the small-activating nucleic acid molecule.
Drugs covered by this patent
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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