Patent · US Active

Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof

US11932852B2 · kind B2 · utility

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1References
22Claims
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Assignee

Inventors

Key dates

Filing dateSep 1, 2021
Grant dateMar 19, 2024
Priority date—
Expiry dateApr 2, 2042

Classification

  • Technology area (CPC C)Chemistry; Metallurgy
  • CPC primaryC12N2750/14143
  • WIPO fieldPharmaceuticals
  • WIPO sectorChemistry

Abstract

The present disclosure relates to RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR) and short hairpin RNA (shRNA), for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto. The present disclosure also relates to the use of the RNAi reagents in combination with PABPN1 replacment reagents, such as constructs which encode functional PABPN1 protein, for treatment of OPMD, compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.

Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.