公开(公告)号：US20240123040A1

公开(公告)日：2024-04-18

申请号：US18276010

申请日：2022-02-04

Applicant: SIGILON THERAPEUTICS, INC.

Inventor： Elina Makino ,  Erika Pearson

IPC: A61K38/46 ,  A61K9/50 ,  A61K35/30 ,  A61P3/00

CPC classification number: A61K38/465 ,  A61K9/5036 ,  A61K35/30 ,  A61P3/00 ,  C12Y204/99 ,  C12Y301/06012

Abstract: Described herein are mammalian cells engineered to express and secrete an ARSB protein and optionally a sialytransferase protein, as well as compositions, implantable devices and device preparations comprising the engineered cells, and methods of making and using the same for treating MPS VI disease.

公开(公告)号：US20230287341A1

公开(公告)日：2023-09-14

申请号：US18018371

申请日：2021-07-28

Applicant: SIGILON THERAPEUTICS, INC.

Inventor： Marissa R. Donovan ,  Elina Makino ,  Erika Pearson

IPC: C12N5/079 ,  A61P3/00

CPC classification number: C12N5/0621 ,  A61P3/00 ,  C12N2510/02

Abstract: Described herein are genetically modified cells derived from a human cell and which contain at least one exogenous transcription unit inserted into at least one of five genomic insertion sites located in Chromosomes 1, 2, 7 and X, as well as compositions, pharmaceutical preparations, and implantable devices comprising the genetically modified cells, and methods of using the same for preventing or treating a disease, disorder, or condition.

公开(公告)号：US20250144174A1

公开(公告)日：2025-05-08

申请号：US18833641

申请日：2023-01-27

Applicant: SIGILON THERAPEUTICS, INC ,  National Research Council of Canada

Inventor： Elina Makino ,  Erika Pearson ,  Gregory Hussack ,  Danica Stanimirovic

IPC: A61K38/17 ,  A61K9/48 ,  A61K35/12 ,  A61K47/36 ,  C07K14/71 ,  C12N5/0735 ,  C12N5/0775

Abstract: Described herein are BBB-transporting fusion proteins, polynucleotides encoding the fusion proteins and mammalian cells genetically modified to express and secrete the fusion proteins, as well as compositions, implantable devices and device preparations comprising the fusion proteins or genetically modified cells secreting the fusion proteins, and methods of making and using the same for treating various CNS diseases and conditions, e.g., any mucopolysaccharidosis type with CNS manifestations.

公开(公告)号：US20250136671A1

公开(公告)日：2025-05-01

申请号：US18833632

申请日：2023-01-27

Applicant: SIGILON THERAPEUTICS, INC. ,  National Research Council of Canada

Inventor： Elina Makino ,  Erika Pearson ,  Gregory Hussack

IPC: C07K16/18 ,  A61K9/48 ,  A61K35/12 ,  A61K38/46 ,  A61K47/36 ,  A61P3/00 ,  C12N9/16 ,  C12N15/62

Abstract: Described herein are ARSB fusion proteins, polynucleotides encoding the fusion proteins and mammalian cells genetically modified to express and secrete the fusion proteins, as well as compositions, implantable devices and device preparations comprising the fusion proteins or genetically modified cells secreting the fusion proteins, and methods of making and using the same for treating MPS-6.