公开(公告)号：WO2021144649A2

公开(公告)日：2021-07-22

申请号：PCT/IB2021/000008

申请日：2021-01-15

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： KLUGMANN, Matthias ,  ROTTENSTEINER, Hanspeter ,  HORLING, Franziska ,  LENGLER, Johannes

IPC: A61K48/00 ,  C12N15/86 ,  A61K48/005 ,  A61K48/0058 ,  A61K48/0066 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2800/22 ,  C12N2830/008 ,  C12N2830/42 ,  C12N2830/48 ,  C12N9/0071 ,  C12Y114/16001

Abstract: The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid and a codon-optimized sequence encoding a human phenylalanine hydroxylase (PAH) enzyme. The disclosure also provides a method of treating a subject having phenylketonuria (PKU), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid, and a promoter operably linked to a nucleic acid sequence that encodes PAH, and wherein administering results in a decrease in phenylalanine level in the subject.

公开(公告)号：EP4090382A2

公开(公告)日：2022-11-23

申请号：EP21709460.6

申请日：2021-01-15

Applicant: Takeda Pharmaceutical Company Limited

Inventor： KLUGMANN, Matthias ,  ROTTENSTEINER, Hanspeter ,  HORLING, Franziska ,  LENGLER, Johannes

IPC: A61K48/00 ,  C12N15/86