WO2023047338 - PRODRUG INCORPORATED sgRNA SYNTHESIS

National phase entry is expected:
Publication Number WO/2023/047338
Publication Date 30.03.2023
International Application No. PCT/IB2022/058996
International Filing Date 23.09.2022
Title **
[English] PRODRUG INCORPORATED sgRNA SYNTHESIS
[French] SYNTHÈSE DE SGRNA SYNTHÉTIQUE PRODRUG
Applicants **
CRISPR THERAPEUTICS AG Baarerstrasse 14 6300 Zug, CH
Inventors
DAS, Rajat 610 Main Street Cambridge, Massachusetts 02139, US
SANKARAN, Ganapathy 610 Main Street Cambridge, Massachusetts 02139, US
Priority Data
63/248,272   24.09.2021   US
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Quotation for National Phase entry

Country StagesTotal
China Filing2560
EPO Filing, Examination21122
Japan Filing595
South Korea Filing575
USA Filing, Examination14510
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Total: 39362

The term for entry into the National Phase has expired. This quotation is for informational purposes only

Abstract[English] Disclosed herein include guide RNA (gRNA), such as single gRNA (sgRNA), and compositions thereof, comprising 2'-O-methyldithiomethyl modified sugar moieties which can be reduced to 2'-O-methanethiol groups in the reducing environment of cells and then converted (e.g., spontaneously converted) to 2'-OH. The resultant gRNA can bind to and direct the activity of an RNA-guided endonuclease (e.g., Cas9).[French] La présente invention comprend un ARN guide (ARNg), tel qu'un ARNg unique (ARNsg), et des compositions de celui-ci, comprenant des fractions de sucre modifiées en 2'-O-méthyldithiométhyle qui peuvent être réduites en groupes 2'-O-méthanethiol dans l'environnement réducteur des cellules et ensuite converties (par exemple, spontanément) en 2'-OH. L'ARNg résultant peut se lier à une endonucléase guidée par ARN (par exemple, Cas9) et diriger son activité.
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