公开(公告)号：US20230040275A1

公开(公告)日：2023-02-09

申请号：US17777954

申请日：2020-11-19

Applicant: SPARK THERAPEUTICS, INC.

Inventor： David William ANDERSON ,  Brittney L. GURDA ,  Federico MINGOZZI ,  Mustafa N. YAZICIOGLU ,  William QUINN

IPC: C12N15/62 ,  A61K39/35 ,  A61K39/00 ,  C07K14/705 ,  C12N15/86 ,  A61K48/00 ,  A61P37/06 ,  A61K31/436 ,  C07K14/47 ,  C07K14/005

Abstract: Nucleic acids encoding fusion proteins that contain an unwanted antigen and a leader sequence for cell secretion 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 autoimmune, allergic and other diseases and disorders, such as multiple sclerosis.

公开(公告)号：US20220184188A1

公开(公告)日：2022-06-16

申请号：US17310378

申请日：2020-01-31

Applicant: SPARK THERAPEUTICS, INC.

Inventor： David William ANDERSON ,  Maryann TOTO ,  Sue E.I. DASEN

IPC: A61K38/48 ,  A61K48/00 ,  A61P37/06 ,  C12N15/86

Abstract: Disclosed herein are methods for treating a primate in need of tripeptidyl peptidase 1 (TPP1), comprising (a) providing a recombinant adeno-associated virus (AAV) vector comprising a nucleic acid encoding TPP1; and (b) administering an amount of the recombinant AAV vector to the central nervous system (CNS) of the primate, wherein the TPP1 is expressed in the primate.