WO2025181704 - RT EDITING COMPOSITIONS AND METHODS

National phase entry is expected:
Publication Number WO/2025/181704
Publication Date 04.09.2025
International Application No. PCT/IB2025/052079
International Filing Date 26.02.2025
Title **
[English] RT EDITING COMPOSITIONS AND METHODS
[French] COMPOSITIONS ET PROCÉDÉS D'ÉDITION DE RT
Applicants **
CRISPR THERAPEUTICS AG
Inventors
FELLMANN, Christof
MIR, Aamir
GREWAL, Parbir Singh
JAYACHANDRAN, Megha
BARNHOUSE, Victoria
KADAM, Bhakti Narendra
CHEN, Victor
YUAN, Qichen
AJAMI, Nassim
LOAIZA, Cristian
FREEMAN, Benjamin Thomas
Priority Data
63/558,338   27.02.2024   US
63/649,882   20.05.2024   US
63/718,994   11.11.2024   US
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译文

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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting6608
EPO Filing, Examination, Granting112277
Japan Filing, Examination, Granting4850
South Korea Filing, Examination, Granting8204
USA Filing, Examination, Granting35940
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Total: 167,879
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Abstract[English] Disclosed herein include methods, compositions, and kits suitable for use in gene editing. RT editing systems are provided herein comprising a fusion protein comprising a Cas9 nickase and a reverse transcriptase, and a template armed guide RNA (tagRNA) comprising from 5' to 3' a spacer sequence, a scaffold sequence, an editing template and a flap binding sequence. In some embodiments, the RT editing system further comprises an enhancer guide RNA (egRNA).[French] L'invention concerne des procédés, des compositions et des kits appropriés pour une utilisation dans l'édition génomique. L'invention concerne des systèmes d'édition RT comprenant une protéine de fusion comprenant une nickase Cas9 et une transcriptase inverse, et un ARN guide armé d'un modèle (ARNtag) comprenant de 5' à 3' une séquence d'espaceur, une séquence d'échafaudage, un modèle d'édition et une séquence de liaison de lambeau. Dans certains modes de réalisation, le système d'édition de RT comprend en outre un ARN guide amplificateur (ARNeg).