公开(公告)号：US12077754B2

公开(公告)日：2024-09-03

申请号：US17876803

申请日：2022-07-29

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Zoya Ignatova ,  Andrew Torda ,  Marco Matthies

IPC: C12N15/11 ,  A61K48/00

CPC classification number: C12N15/11 ,  A61K48/00 ,  C12N2310/3513 ,  C12N2310/531

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：US11434485B2

公开(公告)日：2022-09-06

申请号：US16980927

申请日：2019-03-14

Applicant: Arcturus Therapeutics, Inc.

Inventor： Zoya Ignatova ,  Andrew Torda ,  Marco Matthies

IPC: C12N15/11 ,  A61K48/00

Abstract: A synthetic transfer RNA with an extended anticodon loop. 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.