Patent number: 11447486
Abstract: The present invention relates to benzimidazole derivatives having the general formula I, wherein n is 0 or 1; X1 and X2 are independently, at each occurrence, CR5 or N; Y is C1-C6 alkylene, wherein alkylene is optionally substituted with one to two C1-C3 alkyl groups; R1 is selected from the group consisting of hydrogen, halogen, C1-C6 alkoxy, —NH2, —NHR6, —NR7R8 and —NH—(R9)n—R10, n being 0 or 1; R2 is selected from the group consisting of hydrogen, halogen, C1-C6 alkyl, —NH2, —NHR6, —NR7R8 and —NH—(R9)n—R10; R3 is selected from the group consisting of hydrogen, hydroxyl, OR11, —NR7R8, C1-C6 alkoxy, C1-C6 alkyl, C3-C10 cycloalkyl, C1-C3 haloalkyl, —C(O)NHR11, aryl, heteroaryl and heterocyclyl, wherein each of said cycloalkyl, aryl, heteroaryl and heterocyclyl is optionally and independently substituted with one to four Ra groups; and R4 is selected from the group consisting of —NH2, —N(R12)(V)pR13, —NH(V)p—OR14, —NHC(O)R15, and groups of formula 1a shown below, and their use in the treatment of diseases, in
Type:
Grant
Filed:
July 31, 2015
Date of Patent:
September 20, 2022
Assignees:
INSTITUT PASTEUR KOREA, QURIENT CO., LTD.
Inventors:
Jaeseung Kim, Seohyun Ahn, Yeejin Jeon, Dongsik Park, Young-In Yang, Doohyung Lee, Saeyeon Lee, Jiye Ahn, Jeongjun Kim, Kiyean Nam, Sunhee Kang, Minjung Seo, Mooyoung Seo, Jeongjea Seo, Sung-Jun Han, Jung Hwan Kim, Sangchul Lee, Gahee Choi, Yunmi Lee
Patent number: 11384346
Abstract: The application relates to means, which derive from TAL effectors and TALENs. The structure of the means of the application is especially adapted for partial or full deletion of at least one DNA tandem repeat, more particularly for partial or full deletion of at least one DNA tandem repeat in a double-stranded DNA, more particularly for partial or full deletion of at least one DNA tandem repeat, which is contained in a double-stranded DNA and, which forms a complex secondary structure, such as a hairpin, a triple helix or a tetraplex secondary structure. The means of the application are notably useful in the treatment and/or prevention and/or palliation of a disease or disorder involving at least one DNA tandem repeat, such as DM1, SCA8, SCA12, HDL2, SBMA, HD, DRPLA, SCA1, SCA2, SCA3, SCA6, SCA7, SCA17, PSACH, DM2, SCA10, SPD1, OPMD, CCD, HPE5, HFG syndrome, BPES, EIEE1, FRAXA, FXTAS and FRAXE.
Type:
Grant
Filed:
June 8, 2018
Date of Patent:
July 12, 2022
Assignee:
INSTITUT PASTEUR
Inventors:
Guy-Franck Richard, Valentine Mosbach, David Viterbo