WO2026022777 - 2-HYDROXYPROPYL BETA-CYCLODEXTRINS FOR USE IN A METHOD OF TREATING NIEMANN-PICK DISEASE TYPE C IN SUBJECTS WITH INFANTILE ONSET
National phase entry is expected:
Publication Number
WO/2026/022777
Publication Date
29.01.2026
International Application No.
PCT/IB2025/057566
International Filing Date
25.07.2025
Title **
[English]
2-HYDROXYPROPYL BETA-CYCLODEXTRINS FOR USE IN A METHOD OF TREATING NIEMANN-PICK DISEASE TYPE C IN SUBJECTS WITH INFANTILE ONSET
[French]
2-HYDROXYPROPYL BÊTA-CYCLODEXTRINES À UTILISER DANS UNE MÉTHODE DE TRAITEMENT DE LA MALADIE DE NIEMANN-PICK DE TYPE C CHEZ DES SUJETS PRÉSENTANT UN DÉBUT INFANTILE
Applicants **
BEREN THERAPEUTICS P.B.C.
Inventors
WINNIKE, Jack
JIAO, Lisa
SPINELLA, Jennifer
CAMM, Jason
Priority Data
63/676,065
26.07.2024
US
63/746,180
16.01.2025
US
63/746,623
17.01.2025
US
Application details
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International Searching Authority |
EPO
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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 | 5187 | |
| EPO | Filing, Examination, Granting | 92779 | |
| Japan | Filing, Examination, Granting | 4839 | |
| South Korea | Filing, Examination, Granting | 7195 | |
| USA | Filing, Examination, Granting | 28590 |
Total:
138,590
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Abstract[English]
Provided herein are methods of treatment and medicaments for treating Niemann-Pick Disease Type C (NPC) with hydroxypropyl beta-cyclodextrin (HPBCD), including in subjects with infantile-onset NPC. Provided herein are also methods of preventing NPC in in a human subject in need thereof with HPBCD wherein the human subject is 0-2 or 0-6 years of age or has an age on onset of NPC of 0-2 or 0-6 years. Provided herein are also methods of preventing NPC in in a human subject in need thereof with HPBCD for at least 2 years, at least 4 years, or at least 6 years, wherein the human subject is 0-2 or 0-6 years of age or has an age of onset of 0-2 or 0-6 years.[French]
L'invention concerne des méthodes de traitement et des médicaments pour le traitement de la maladie de Niemann-Pick de type C (NPC) avec de l'hydroxypropyl beta-cyclodextrine (HPBCD), y compris chez des sujets atteints d'un NPC à début infantile. L'invention concerne également des procédés de prévention du NPC chez un sujet humain en ayant besoin avec du HPBCD, le sujet humain étant de 0 à 2 ou de 0 à 6 ans ou ayant un âge lors de l'apparition de NPC de 0 à 2 ou de 0 à 6 ans. L'invention concerne également des procédés de prévention du NPC chez un sujet humain en ayant besoin avec du HPBCD pendant au moins 2 ans, au moins 4 ans, ou au moins 6 ans, le sujet humain étant de 0 à 2 ou de 0 à 6 ans ou ayant un âge d'apparition de 0 à 2 ou de 0 à 6 ans.