Therapeutic method for huntington's disease

US11268093B2 · kind B2 · utility

Assignees

• Rula Zain-Luqman · SE
• C. I. Edvard Smith · SE

Inventors

• Rula Zain-Luqman · Täby, SE
• C. I. Edvard Smith · Stockholm, SE
• Eman Zaghloul · Huddinge, SE
• Pedro Moreno · Lexington, US
• Olof Gissberg · Stuvsta-Snättringe, SE
• Karin Lundin · Malmköping, SE
• Jesper Wengel · Odense, DK
• Liam Good · London, GB
• Helen Bergquist · Stockholm, SE

Key dates

• Filing date: Dec 29, 2017
• Grant date: Mar 8, 2022
• Expiry date: May 11, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Q2600/156
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention relates to anti-gene oligonucleotides adapted to hybridize to DNA in a HTT gene, which are based on locked nucleic acids, phosphorodiamidate morpholino oligomers, (PMO) or equivalent oligonucleotide analogues comprising a (CAG)n sequence, and whose target is a sequence where the majority of the repeats are CAG/CTG, for use in down regulating the expression of the HTT gene, resulting in reduced HTT mRNA and protein levels in afflicted subjects, or in diagnosis, treatment and/or prevention of Huntington's disease, and where the anti-gene oligonucleotides target non-canonical DNA structures, including hairpin and cruciform. The invention also relates to a delivery system comprising said oligonucleotides and said use thereof.