uniQure Strengthens Intellectual Property Portfolio with Granted Patent Claims Covering AMT-130 for Huntington’suniQore, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced the issuance of two new patents covering AMT-130, the Company’s gene therapy candidate for the treatment of Huntington’s disease. AMT-130 comprises a recombinant AAV5 vector carrying a DNA cassette, encoding a microRNA that non-selectively lowers or knocks-down human huntingtin protein in Huntington’s disease patients.
The U.S. Patent and Trademark Office issued U.S. Patent 10,174,321 on January 8, 2019 and the European Patent Office issued EP 3237618 on May 22, 2019. The claims as granted cover the RNA constructs specifically designed to target exon1 and the embedding of these Huntington’s disease RNA sequences into the miR451 scaffold, which is exclusively licensed to uniQure from Cold Spring Harbor Laboratory (CSHL). The claims also cover certain expression cassettes comprising the RNA constructs and the use of gene therapy vectors including AAV vectors encompassing the described expression cassettes.