公开(公告)号：EP4458842A1

公开(公告)日：2024-11-06

申请号：EP22901639.9

申请日：2022-11-18

申请人： Glugenetherapeutics Inc.

发明人： JANG, Jae-Hyung ,  KIM, Yoo Jin

IPC分类号： C07K14/005 ,  A61K48/00 ,  A61P9/12 ,  C12N15/86 ,  A61P11/00

摘要： The present invention relates to a mutant of the adeno-associated virus serotype 1 (AAV1) capsid protein, a nucleic acid coding for the mutant of the AAV1 capsid protein, a recombinant AAV1 vector carrying the nucleic acid coding for the mutant of the AAV1 capsid protein, and a pharmaceutical composition comprising the vector, wherein the mutant has cysteine substituted for serine at position 430 and valine substituted for isoleucine at position 647 in comparison with the amino acid sequence of the wild-type AAV capsid protein. Specifically, when delivered in an aerosol state through the trachea, the recombinant virus vector carrying a nucleic acid coding for the mutant of the AAV1 capsid protein enhances transgene expression in pulmonary vessel targeting cells and thus is useful for preventing or treating pulmonary arterial hypertension.