Gene-therapy vectors for treating cardiomyopathy

US11773408B2 · kind B2 · utility

Assignees

• Lucie Carrier · DE
• Thomas Eschenhagen · DE
• Thomas Voit · GB
• Giulia Mearini · DE
• Oliver Mueller · DE
• Doreen Stimpel · DE

Inventors

• Lucie Carrier · Hamburg, DE
• Thomas Eschenhagen · Hamburg, DE
• Thomas Voit · Gif-sur-Yvette, FR
• Giulia Mearini · Hamburg, DE
• Oliver Mueller · Heikendorf, DE
• Doreen Stimpel · Hamburg, DE
• Julia Mourot-Filiatre · Brasília, BR

Key dates

• Filing date: Dec 9, 2019
• Grant date: Oct 3, 2023
• Expiry date: Dec 18, 2041

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2830/008
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.