公开(公告)号：US12084693B2

公开(公告)日：2024-09-10

申请号：US17055523

申请日：2019-05-15

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Xavier Anguela ,  Sean Armour ,  Jayme Nordin

IPC: C12N9/26 ,  A61K48/00 ,  C12N15/86

CPC classification number: C12N9/2408 ,  A61K48/005 ,  C12N15/86 ,  C12Y302/0102 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2800/22 ,  C12N2830/001 ,  C12N2830/50

Abstract: The invention provides nucleic acids encoding acid α-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5. In certain embodiments, nucleic acids encoding acid α-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid α-glucosidase (GAA) are also provided.

公开(公告)号：US20250101396A1

公开(公告)日：2025-03-27

申请号：US18828493

申请日：2024-09-09

Applicant: Spark Therapeutics, Inc.

Inventor： Xavier Anguela ,  Sean Armour ,  Jayme Nordin

IPC: C12N9/26 ,  A61K48/00 ,  C12N15/86

Abstract: The invention provides nucleic acids encoding acid α-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs: 1-5. In certain embodiments, nucleic acids encoding acid α-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid α-glucosidase (GAA) are also provided.