WO2026036016 - METHODS AND COMPOSITIONS FOR TREATING NEUROLOGIC DISORDERS
National phase entry is expected:
Publication Number
WO/2026/036016
Publication Date
12.02.2026
International Application No.
PCT/US2025/041242
International Filing Date
08.08.2025
Title **
[English]
METHODS AND COMPOSITIONS FOR TREATING NEUROLOGIC DISORDERS
[French]
MÉTHODES ET COMPOSITIONS POUR TRAITER DES TROUBLES NEUROLOGIQUES
Applicants **
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
Inventors
SARKAR, Partha, S.
PRADHAN, Subrata
Priority Data
63/680,946
08.08.2024
US
Application details
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International Searching Authority |
USPTO
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| Recordal of a Change of the Applicant's Name/Address |
Change of Applicant's Name and Address
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| Type of Assignment |
The Standard Agent's Assignment
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| Applicant's Legal Status |
Legal Entity
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| Entry into National Phase under |
Chapter I
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| Patent Delivery |
Send the Letters Patent by Courier
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| Translation |
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Quotation for National Phase entry
| Country | Stages | Total | |
|---|---|---|---|
| China | Filing, Examination, Granting | 2417 | |
| EPO | Filing, Examination, Granting | 13308 | |
| Japan | Filing, Examination, Granting | 2045 | |
| South Korea | Filing, Examination, Granting | 1854 | |
| USA | Filing, Examination, Granting | 6110 |
Total:
25,734
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Abstract[English]
Methods and compositions described herein provide a solution for treating Huntington's disease and other neurodegenerative diseases by administering a recombinant polypeptide, such as a wild-type Huntingtin (HTT) fragment (e.g., SEQ ID NO:4), Brahma-related gene 1 (BRG1), Brahma (BRM), PNKP, Matrin 3 (MATRN3), or functional variants thereof, to a subject having, at risk of developing, or suspected of having a neurodegenerative disease characterized by persistent DNA double-strand breaks and impaired RNA processing. The polypeptides, delivered via mRNA, circular RNA, or AAV vectors, restore transcription-coupled non-homologous end-joining (TC-NHEJ) activity and RNA processing, ameliorating disease progression in neuronal and non-neuronal brain cells.[French]
La présente invention concerne des procédés et des compositions qui fournissent une solution pour traiter la maladie de Huntington et d'autres maladies neurodégénératives par administration d'un polypeptide recombinant, tel qu'un fragment de Huntingtine (HTT) de type sauvage (par exemple, SEQ ID NO : 4), gène 1 lié au Brahma (BRG1), Brahma (BRM), PNKP, Matrin 3 (MATRN3), ou des variants fonctionnels de ceux-ci, à un sujet ayant, à risque de développer, ou suspecté d'avoir une maladie neurodégénérative caractérisée par des cassures de double brin d'ADN persistant et un traitement d'ARN altéré. Les polypeptides, délivrés par l'intermédiaire d'ARNm, d'ARN circulaire ou de vecteurs AAV, restaurent une activité de jonction d'extrémité non homologue couplée à la transcription (TC-NHEJ) et un traitement d'ARN, améliorant la progression de la maladie dans des cellules cérébrales neuronales et non neuronales.