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：US20210363192A1

公开(公告)日：2021-11-25

申请号：US17050362

申请日：2019-04-26

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Xavier ANGUELA ,  Sean ARMOUR ,  Nicholas KEISER ,  Suryanarayan SOMANATHAN ,  Mustafa N. YAZICIOGLU

IPC: C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N15/113

Abstract: The invention provides modified adeno-associated virus (AAV) capsid proteins. Modified AAV capsid proteins include, for example, capsid proteins modified to have a peptide insertion comprising a nuclear localization signal (NLS) sequence, capsid proteins modified to have an amino acid substitution at an RXXL site or a (L/P)PXY site, where X can be any amino acid, and capsid proteins modified to have one or more particular amino acid positions substituted with a different amino acid.