OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

Title:

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

Document Type and Number:

WIPO Patent Application WO/2017/062862

Kind Code:

A3

Abstract:

Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.

Inventors:

BUTLER DAVID CHARLES DONNELL (US)
DIVAKARAMENON SETHUMADHAVAN (US)
FRANCIS CHRISTOPHER J (US)
FRANK-KAMENETSKY MARIA DAVID (US)
IWAMOTO NAOKI (US)
LU GENLIANG (US)
MARAPPAN SUBRAMANIAN (US)
MEENA (US)
VARGEESE CHANDRA (US)
VERDINE GREGORY L (US)
YANG HAILIN (US)
ZHANG JASON JINGXIN (US)

Application Number:

PCT/US2016/056123

Publication Date:

May 26, 2017

Filing Date:

October 07, 2016

Export Citation:

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Assignee:

WAVE LIFE SCIENCES LTD (SG)
BUTLER DAVID CHARLES DONNELL (US)
DIVAKARAMENON SETHUMADHAVAN (US)
FRANCIS CHRISTOPHER J (US)
FRANK-KAMENETSKY MARIA DAVID (US)
IWAMOTO NAOKI (US)
LU GENLIANG (US)
MARAPPAN SUBRAMANIAN (US)
MEENA (US)
VARGEESE CHANDRA (US)
VERDINE GREGORY L (US)
YANG HAILIN (US)
ZHANG JASON JINGXIN (US)

International Classes:

C07C317/28; C07D295/088; C12N15/11; C12N15/113; C12Q1/68

Foreign References:

US20150211006A1	2015-07-30
US20150080563A2	2015-03-19
US20110269814A1	2011-11-03
US20020137921A1	2002-09-26

Other References: