WO2025083617 - METHODS AND COMPOSITIONS FOR NUCLEIC CONSTRUCT DELIVERY

National phase entry:
Publication Number WO/2025/083617
Publication Date 24.04.2025
International Application No. PCT/IB2024/060231
International Filing Date 17.10.2024
Title **
[English] METHODS AND COMPOSITIONS FOR NUCLEIC CONSTRUCT DELIVERY
[French] PROCÉDÉS ET COMPOSITIONS POUR ADMINISTRER UNE CONSTRUCTION NUCLÉIQUE
Applicants **
NANOCELL THERAPEUTICS HOLDINGS B.V.
Inventors
ASHOTI, Ator Rafael Odisho
EVERS, Martijn Johannes Wilhelmus
VAN DIEST, Eline
LUBELSKI, Jacek
Priority Data
63/591,282   18.10.2023   US
63/594,639   31.10.2023   US
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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting4558
EPO Filing, Examination, Granting68939
Japan Filing, Examination, Granting4260
South Korea Filing, Examination, Granting6220
USA Filing, Examination, Granting25990
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Total: 109,967

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Abstract[English] The present disclosure includes a composition including a) one or more nucleic acid constructs, wherein the one or more nucleic acid constructs encode: at least one of Replicase 68 (Rep68) and Replicase 78 (Rep78); and at least one protein of viral origin having nucleic acid replication and/or transgene amplification promotion activity, and b) nucleic acid construct comprising a coding region encoding at least one gene of interest (GOI) and at least one inverted terminal repeat (ITR) sequence on each side of the GOI, and methods of making and using the composition.[French] La présente invention comprend une composition comprenant a) une ou plusieurs constructions d'acide nucléique, la ou les constructions d'acide nucléique codant : au moins l'un parmi la réplicase 68 (Rep68) et la réplicase 78 (Rep78) ; et au moins une protéine d'origine virale ayant une réplication d'acide nucléique et/ou une activité de promotion d'amplification de transgène, et b) une construction d'acide nucléique comprenant une région de codage codant pour au moins un gène d'intérêt (GOI) et au moins une séquence de répétition terminale inversée (ITR) de chaque côté de la GOI, et des procédés de fabrication et d'utilisation de la composition.

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