WO2026139885 - SYSTEM FOR EDITING THE F508DEL MUTATION IN THE HUMAN CFTR GENE FOR THE CYSTIC FIBROSIS TREATMENT
National phase entry is expected:
Publication Number
WO/2026/139885
Publication Date
02.07.2026
International Application No.
PCT/IB2025/063381
International Filing Date
23.12.2025
Title **
[English]
SYSTEM FOR EDITING THE F508DEL MUTATION IN THE HUMAN CFTR GENE FOR THE CYSTIC FIBROSIS TREATMENT
[French]
SYSTÈME D'ÉDITION DE LA MUTATION F508DEL DANS LE GÈNE CFTR HUMAIN POUR LE TRAITEMENT DE LA MUCOVISCIDOSE
Applicants **
MEDICO DISTRIBUTION DMCC
Inventors
SMIRNIKHINA, Svetlana Anatolievna
LAVROV, Alexander Vyacheslavovich
VOLODINA, Olga Vladimirovna
DEMCHENKO, Anna Grigorievna
ANUCHINA, Arina Arturovna
KUTSEV, Sergey Ivanovich
Priority Data
2024139556
25.12.2024
RU
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IP Australia
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The Standard Agent's Assignment
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Legal Entity
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| Entry into National Phase under |
Chapter I
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Quotation for National Phase entry
| Country | Stages | Total | |
|---|---|---|---|
| China | Filing, Examination, Granting | 2793 | |
| EPO | Filing, Examination, Granting | 14477 | |
| Japan | Filing, Examination, Granting | 2152 | |
| South Korea | Filing, Examination, Granting | 2061 | |
| USA | Filing, Examination, Granting | 11190 |
Total:
32,673
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Abstract[English]
The present invention relates to the field of biotechnology, genetic engineering and medicine, in particular, to a highly efficient system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene to the wild type, comprising a polynucleotide which comprises a nucleotide sequence, encoding the SpCas9 nickase recognizing PAM NGG or NG, and a prime editing guide RNA (pegRNA) comprising in its structure a sequence complementary to the target locus for editing the F508del mutation in human CFTR gene, a reverse transcription template (RTT) and a primer binding site (PBS), wherein the prime editing guide RNA (pegRNA) has sequence SEQ ID NO: 1-6 or a sequence comprising one or more replacements in the RTT compared to SEQ ID NO: 1-6, selected from SEQ ID NO: 7-168. The system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene to the wild type provides more efficient editing of the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene to the wild type and is a highly efficient cystic fibrosis treatment. The present invention also relates to the method of editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene using the system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene according to the present invention and the use of the system for editing the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene for treating cystic fibrosis.[French]
La présente invention concerne le domaine de la biotechnologie, de l'ingénierie génétique et de la médecine, et plus particulièrement un système hautement efficace permettant de corriger la mutation F508del dans le gène codant le régulateur transmembranaire de la mucoviscidose (CFTR) pour le ramener à son type sauvage, composé d'un polynucléotide contenant une séquence nucléotidique codant la nickase SpCas9 reconnaissant le PAM NGG ou NG, et un ARN guide d'édition primaire (ARNpeg) contenant dans sa structure une séquence complémentaire du locus cible pour l'édition de la mutation F508del dans le gène CFTR humain, une matrice de transcription inverse (RTT) et un site de liaison d'amorce (PBS), l'ARNpeg comportant la séquence SEQ ID NO: 1 à 6 ou une séquence contenant un ou plusieurs remplacements dans la séquence RTT par comparaison à SEQ ID NO : 1 à 6, choisis parmi SEQ ID NO : 7 à 168. Le système permettant d'éditer la mutation F508del dans le gène CFTR pour le ramener à son type sauvage permet une édition plus efficace de cette mutation et constitue un traitement hautement efficace contre la mucoviscidose (MV). La présente invention concerne également le procédé d'édition de la mutation F508del dans le gène du gène CFTR par l'utilisation du système d'édition de la mutation F508del dans le gène CFTR selon la présente invention, ainsi que sur l'utilisation de ce système d'édition de la mutation F508del dans le gène CFTR pour le traitement de la MV.