公开(公告)号：US12290567B2

公开(公告)日：2025-05-06

申请号：US17011880

申请日：2020-09-03

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar Rajappan ,  Padmanabh Chivukula ,  Kiyoshi Tachikawa ,  Steven Tanis ,  Priya Karmali

IPC: A61K47/54 ,  C07H15/04 ,  C12N15/113

Abstract: ASGP-R binding molecular conjugates are provided. The conjugates are useful to deliver therapeutically effective amounts of biologically active molecules to target cells and tissues of a subject. Compositions are also provided comprising the molecular conjugates.

公开(公告)号：US20210292768A1

公开(公告)日：2021-09-23

申请号：US17266556

申请日：2019-08-08

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Lily Xu ,  Angel Leu ,  Marciano Sablad ,  Rajesh Mukthavaram ,  Priya Karmali

IPC: C12N15/113 ,  A61P1/16

Abstract: This disclosure encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting expression of PDGFRB in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers.

公开(公告)号：US10526284B2

公开(公告)日：2020-01-07

申请号：US15849573

申请日：2017-12-20

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  Priya Karmali ,  Steven P. Tanis

IPC: C07C333/04 ,  C07C271/22

Abstract: What is described is a compound of formula I consisting of a compound in which R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons, or a branched chain alkyl consisting of 10 to 31 carbons; L1 and L2 are the same or different, each a linear alkane of 1 to 20 carbons or a linear alkene of 2 to 20 carbons; X1 is S or O; R3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.