公开(公告)号：US20230183685A1

公开(公告)日：2023-06-15

申请号：US17876803

申请日：2022-07-29

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Zoya IGNATOVA ,  Andrew TORDA ,  Marco MATTHIES

IPC: C12N15/11

CPC classification number: C12N15/11 ,  A61K48/00

Abstract: The invention relates to a synthetic transfer RNA with an extended anticodon loop. The invention provides a synthetic suppressor transfer RNA useful for the treatment of a genetic disease like cystic fibrosis associated with a nonsense mutation. The synthetic transfer RNA contains an extended anticodon loop with two consecutive anticodon base triplets configured to base-pair to two consecutive codon base triplets on an mRNA. The first anticodon base triplet or the second anticodon base triplet is configured to base-pair to a stop codon base triplet on the mRNA.

公开(公告)号：US20220162612A1

公开(公告)日：2022-05-26

申请号：US17602251

申请日：2020-04-09

Applicant: Arcturus Therapeutics, Inc.

Inventor： Zoya IGNATOVA ,  Andrew TORDA

IPC: C12N15/113

Abstract: The invention relates to a synthetic transfer RNA with an extended anticodon loop. The invention provides a synthetic suppressor transfer RNA useful for the treatment of a genetic disease like neurofibromatosis associated with a frameshift mutation. The synthetic transfer RNA comprises an extended anticodon loop having a four-nucleotide anticodon or a five-nucleotide anticodon.