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AR129312A1 - Composiciones y métodos para inhibir la expresión de snca - Google Patents

Composiciones y métodos para inhibir la expresión de snca

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Publication number
AR129312A1
AR129312A1 ARP230101181A ARP230101181A AR129312A1 AR 129312 A1 AR129312 A1 AR 129312A1 AR P230101181 A ARP230101181 A AR P230101181A AR P230101181 A ARP230101181 A AR P230101181A AR 129312 A1 AR129312 A1 AR 129312A1
Authority
AR
Argentina
Prior art keywords
region
antisense strand
gene expression
complementarity
snca
Prior art date
Application number
ARP230101181A
Other languages
English (en)
Inventor
Bob Dale Brown
Henryk T Dudek
Seongmoon Cheong
Shiyu Wang
Travis Grim
Matthew Guese Costales
Maire Jung
Original Assignee
Dicerna Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of AR129312A1 publication Critical patent/AR129312A1/es

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
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Abstract

En el presente documento se proporcionan oligonucleótidos (por ejemplo, oligonucleótidos de ARNi) que inhiben la expresión génica de SNCA, incluyendo oligonucleótidos conjugados con un ligando de direccionamiento (por ejemplo, resto de GalNAC o resto lipídico). También se proporcionan composiciones que incluyen estas y sus usos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociadas con la expresión génica de SNCA. Reivindicación 1: Un oligonucleótido de ARNi para reducir la expresión génica de SNCA, donde el oligonucleótido comprende una hebra de sentido y una hebra de antisentido, en donde la hebra de sentido y la hebra de antisentido forman una región dúplex, en donde la hebra de antisentido comprende una región de complementariedad con una secuencia diana de ARNm de SNCA de una cualquiera de las SEQ ID Nº 1683 - 2066, y en donde la región de complementariedad tiene al menos 15 nucleótidos contiguos de longitud. Reivindicación 8: Un oligonucleótido de ARNi bicatenario para reducir la expresión génica de SNCA, donde el oligonucleótido comprende: (i) una hebra de antisentido de 19 - 30 nucleótidos de longitud, en donde la hebra de antisentido comprende una secuencia de nucleótidos que comprende una región de complementariedad con una secuencia diana de ARNm de SNCA, en donde la región de complementariedad se selecciona de las SEQ ID Nº 2067 - 2450 y (ii) una hebra de sentido de 19 - 50 nucleótidos de longitud que comprende una región de complementariedad con la hebra de antisentido, en donde las hebras de antisentido y sentido son hebras separadas que forman una región dúplex asimétrica que tiene un saliente de 1 - 4 nucleótidos en el extremo 3’ de la hebra de antisentido. Reivindicación 111: Uso del oligonucleótido de ARNi de cualquiera de las reivindicaciones 1 a 97, o la composición farmacéutica de la reivindicación 98, en la fabricación de un medicamento para el tratamiento de una enfermedad, trastorno o afección asociada con la expresión génica de SNCA. Reivindicación 114: El uso de la reivindicación 111, el oligonucleótido de ARNi o composición farmacéutica para su uso, o adaptable para su uso, de la reivindicación 112, o el kit de la reivindicación 113, en donde la enfermedad, trastorno o afección asociada con la expresión génica de SNCA es atrofia sistémica múltiple, demencia con cuerpos de Lewy y enfermedad de Parkinson.
ARP230101181A 2022-05-13 2023-05-12 Composiciones y métodos para inhibir la expresión de snca AR129312A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202263364639P 2022-05-13 2022-05-13

Publications (1)

Publication Number Publication Date
AR129312A1 true AR129312A1 (es) 2024-08-14

Family

ID=86760635

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP230101181A AR129312A1 (es) 2022-05-13 2023-05-12 Composiciones y métodos para inhibir la expresión de snca

Country Status (17)

Country Link
US (1) US20230416743A1 (es)
EP (1) EP4522748A1 (es)
JP (1) JP2025518507A (es)
KR (1) KR20250024534A (es)
CN (1) CN119630791A (es)
AR (1) AR129312A1 (es)
AU (1) AU2023267664A1 (es)
CA (1) CA3250061A1 (es)
CL (2) CL2024003390A1 (es)
CO (1) CO2024015326A2 (es)
CR (1) CR20240494A (es)
DO (1) DOP2024000226A (es)
IL (1) IL316843A (es)
MX (1) MX2024013992A (es)
PE (1) PE20250929A1 (es)
TW (1) TW202409275A (es)
WO (1) WO2023220351A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025151407A1 (en) * 2024-01-08 2025-07-17 Denali Therapeutics Inc. Compositions and methods for modulating alpha-synuclein expression

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Publication number Publication date
CA3250061A1 (en) 2023-11-16
TW202409275A (zh) 2024-03-01
PE20250929A1 (es) 2025-04-02
US20230416743A1 (en) 2023-12-28
AU2023267664A1 (en) 2024-10-31
MX2024013992A (es) 2024-12-06
CN119630791A (zh) 2025-03-14
CL2024003390A1 (es) 2025-03-14
DOP2024000226A (es) 2024-12-15
CL2025002529A1 (es) 2025-11-28
WO2023220351A1 (en) 2023-11-16
KR20250024534A (ko) 2025-02-18
CO2024015326A2 (es) 2024-12-30
JP2025518507A (ja) 2025-06-17
IL316843A (en) 2025-01-01
EP4522748A1 (en) 2025-03-19
CR20240494A (es) 2025-03-03

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