Title:
オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
Document Type and Number:
Japanese Patent JP7445657
Kind Code:
B2
Abstract:
The present disclosure provides a modified human OTC protein having improved properties for the treatment of OTC deficiency in a patient. Preferably, the protein of the disclosure is produced from a codon optimized mRNA suitable for administration to a patient suffering from OTC deficiency wherein upon administration of the mRNA to the patient, the protein of the disclosure is expressed in the patient in therapeutically effective amounts to treat OTC deficiency. The present disclosure also provides codon optimized mRNA sequences encoding wild type human OTC comprising a 5′ UTR derived from a gene expressed by Arabidopsis thaliana for use in treating OTC deficiency in a patient.
Inventors:
Perez-Garcia, Carlos Gee
Kiyoshi Tachikawa
Daiki Matsuda
CHIVUKULA, Padmanabh
Kiyoshi Tachikawa
Daiki Matsuda
CHIVUKULA, Padmanabh
Application Number:
JP2021531945A
Publication Date:
March 07, 2024
Filing Date:
December 05, 2019
Export Citation:
Assignee:
Arcturus Therapeutics,Inc.
International Classes:
C12N15/54; A61K38/43; A61K48/00; A61P3/00; A61P7/00; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N9/10; C12N15/63
Foreign References:
| WO2017218524A1 | ||||
| WO2015138348A1 | ||||
| WO2015017519A1 | ||||
| WO2016118697A1 | ||||
| WO2014170896A2 | ||||
| WO2018127382A1 |
Attorney, Agent or Firm:
Norito Yamao
Norifumi Tomita
Fumio Inai
Manami Sasakura
Yusuke Shirae
Norifumi Tomita
Fumio Inai
Manami Sasakura
Yusuke Shirae