公开(公告)号：US20230165977A1

公开(公告)日：2023-06-01

申请号：US17997312

申请日：2021-04-22

Applicant: Pfizer Inc.

Inventor： Paola Leone ,  Jeremy Francis ,  Basel T. Assaf ,  Shoh Asano ,  Kathrine Hales ,  Allison P. Berg

IPC: A61K48/00 ,  C12N9/80 ,  C12N15/86 ,  A61P25/28

CPC classification number: A61K48/0066 ,  C12N9/80 ,  C12Y305/01015 ,  C12N15/86 ,  A61P25/28 ,  C12N2750/14121 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2800/22

Abstract: The present disclosure relates to recombinant nucleic acids and gene therapy vectors comprising a modified nucleic acid encoding aspartoacylase (ASPA), and variants thereof, for use in the treatment of diseases and disorders associated with a deficiency or dysfunction of ASPA, and in particular, Canavan disease.