Patent · US Active

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

US11142765B2 · kind B2 · utility

1Cited by

0References

27Claims

0Family size

Assignee

Benitec IP Holdings Inc. · US

Inventors

Vanessa Strings-Ufombah · Hayward, US
David Suhy · San Ramon, US

Key dates

Filing date	Dec 14, 2017
Grant date	Oct 12, 2021
Priority date	—
Expiry date	Dec 14, 2037

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 replacement 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.