Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues

US10537617B2 · kind B2 · utility

Assignee

• THE CHILDREN'S HOSPITAL OF PHILADELPHIA · US

Inventors

• Katherine A. High · Yeadon, US
• Xavier Anguela · Philadelphia, US

Key dates

• Filing date: Jun 23, 2016
• Grant date: Jan 21, 2020
• Expiry date: Jun 23, 2036

Classification

• Technology area (CPC —): General

Abstract

The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.