公开(公告)号：US20240074986A1

公开(公告)日：2024-03-07

申请号：US18258981

申请日：2021-12-22

Applicant: Spark Therapeutics, Inc.

Inventor： Ali Nahvi ,  Xavier Anguela ,  Rui Zhang

IPC: A61K9/51 ,  A61K31/573 ,  A61K31/663 ,  A61K48/00 ,  C07K16/28 ,  C12N15/88

CPC classification number: A61K9/5123 ,  A61K31/573 ,  A61K31/663 ,  A61K48/0058 ,  C07K16/2866 ,  C12N15/88

Abstract: Methods of delivering a transgene to a subject in need thereof are described. In particular, the methods include administering to the subject (i) a phagocyte-depleting agent, and (ii) a pharmaceutical composition comprising a non-viral vector comprising the transgene and a pharmaceutically acceptable carrier. The methods can be used to treat a subject in need of treatment for a disease caused by a loss of function or activity of a protein, or to treat a subject in need of treatment for a disease caused by a gain of function activity or expression of a protein.

公开(公告)号：US20240350667A1

公开(公告)日：2024-10-24

申请号：US18685681

申请日：2022-07-22

Applicant: Spark Therapeutics, Inc.

Inventor： Xavier Anguel ,  Pedro Cejas ,  Ali Nahvi ,  Mustafa Yazicioglu, III ,  Rui Zhang

IPC: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/404 ,  A61K31/437 ,  A61K45/06 ,  C12N15/88

CPC classification number: A61K48/005 ,  A61K9/127 ,  A61K9/5123 ,  A61K31/404 ,  A61K31/437 ,  A61K45/06 ,  A61K48/0083 ,  C12N15/88

Abstract: The present invention features method and composition that can be used to facilitate intracellular delivery of DNA to a subject. The provided methods and compositions employ a nanoparticle for intracellular DNA delivery and a cytosolic DNA-sensing inhibitor. The cytosolic DNA-sensing inhibitor is provided to decrease the subject's immune response that can be stimulated by the DNA.