Patent · US Active

Compositions and methods for modulating Ataxin 3 expression

US10533175B2 · kind B2 · utility

3Cited by

185References

54Claims

0Family size

Assignees

Ionis Pharmaceuticals, Inc. · US
ACADEMISCH ZIEKENHUIS LEIDEN (H.O.D.N. LUMC) GMBH · NL

Inventors

Frank Rigo · Carlsbad, US
Thazha P. Prakash · Carlsbad, US
Wilhelmina Maria Clasina VAN ROON-MOM · Leiden, NL
Lodewijk Julius Anton TOONEN · Leiden, NL

Key dates

Filing date	Sep 23, 2016
Grant date	Jan 14, 2020
Priority date	—
Expiry date	Sep 23, 2036

Classification

Technology area (CPC C)Chemistry; Metallurgy
CPC primaryC12N2310/3525
WIPO fieldPharmaceuticals
WIPO sectorChemistry

Abstract

Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.

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