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：US20240110201A1

公开(公告)日：2024-04-04

申请号：US18262959

申请日：2022-01-27

Applicant: Spark Therapeutics, Inc.

Inventor： Christopher Riling ,  Francis Pankowicz ,  Sean Armour ,  William John Quinn, III ,  Michael Preston ,  Stephen Ioele ,  Daniel Cohen ,  Zhenwei Kelvin See

IPC: C12N15/864 ,  C12N15/62

CPC classification number: C12N15/8645 ,  C12N15/625 ,  C12N2830/001 ,  C12N2830/50

Abstract: Nucleic acids encoding C1 inhibitor are described. Also described are expression cassettes, vectors, cells, and cell lines containing the nucleic acids, as well as methods of using the nucleic acids to treat complement-mediated disorders, such as hereditary angioedema.

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.

公开(公告)号：US20240301380A1

公开(公告)日：2024-09-12

申请号：US18261285

申请日：2022-01-13

Applicant: Spark Therapeutics, Inc.

Inventor： Sean Armour ,  Daniel Cohen ,  Christopher Riling

IPC: C12N9/40 ,  A61K48/00 ,  A61P3/00 ,  C12N15/86

CPC classification number: C12N9/2465 ,  A61K48/0058 ,  A61P3/00 ,  C12N15/86 ,  C07K2319/02 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/50 ,  C12Y302/01022

Abstract: Polynucleotides including nucleic acids encoding α-galactosidase A (GLA) are described. Also described are expression cassettes, vectors, cells, and cell lines containing the polynucleotides, as well as methods of using the polynucleotides to treat lysosomal storage disorders such as Fabry disease.