公开(公告)号：US20230391861A1

公开(公告)日：2023-12-07

申请号：US18026546

申请日：2021-09-27

申请人： Philip A. BEACHY ,  Yunxiao ZHANG ,  Aashish MANGLIK ,  Wan-Jin LU ,  Shuo HAN ,  The Board of Trustees of the Leland Stanford Junior University

发明人： Philip A. Beachy ,  Yunxiao Zhang ,  Aashish Manglik ,  Wan-Jin Lu ,  Shuo Han

IPC分类号： C07K16/22 ,  C07K14/39 ,  C12N15/70

CPC分类号： C07K16/22 ,  C07K14/39 ,  C12N15/70 ,  C07K2317/569 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2317/24 ,  A61K38/00

摘要： Provided is a conformation-specific antigen binding domain (ABD) specific for the Hedgehog receptor Patched1, which may be provided in the form of a nanobody. This nanobody potently activates the Hedgehog pathway in vitro and in vivo by stabilizing an alternative conformation of a Patched1 “switch helix”. This ABD or nanobody is water soluble, i.e. does not require lipid modifications for its activity, facilitating mechanistic studies of Hedgehog pathway activation and therapeutic use.