WO2026085164A2 - Formulations d'arni de snca et leurs procédés d'utilisation - Google Patents

Formulations d'arni de snca et leurs procédés d'utilisation

Info

Publication number
WO2026085164A2
WO2026085164A2 PCT/US2025/050995 US2025050995W WO2026085164A2 WO 2026085164 A2 WO2026085164 A2 WO 2026085164A2 US 2025050995 W US2025050995 W US 2025050995W WO 2026085164 A2 WO2026085164 A2 WO 2026085164A2
Authority
WO
WIPO (PCT)
Prior art keywords
formulation
snca
disease
subject
uzq
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
PCT/US2025/050995
Other languages
English (en)
Inventor
Andy PHUNG
Han YUEH
Gang Shen
Matthias Kretschmer
Kamran SIMANI
Liuwei Jiang
Bo PANG
Lubomir NECHEV
Ligang Zhang
Mei Tsung
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of WO2026085164A2 publication Critical patent/WO2026085164A2/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La divulgation concerne des formulations d'agents à base d'acide ribonucléique double brin (ARNdb) ciblant un gène SNCA, ainsi que des procédés d'inhibition de l'expression d'un gène SNCA et des méthodes de traitement de sujets atteints d'une maladie neurogénérative ou d'un trouble neurodégénératif associé(e) à la SNCA, par ex., la maladie de Parkinson (MP), l'atrophie multisystématisée, la démence à corps de Lewy (LBD), entre autres synucléinopathies, à l'aide de telles formulations d'agents à base d'ARNdb.
PCT/US2025/050995 2024-10-16 2025-10-15 Formulations d'arni de snca et leurs procédés d'utilisation Pending WO2026085164A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202463708119P 2024-10-16 2024-10-16
US63/708,119 2024-10-16

Publications (1)

Publication Number Publication Date
WO2026085164A2 true WO2026085164A2 (fr) 2026-04-23

Family

ID=97796786

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2025/050995 Pending WO2026085164A2 (fr) 2024-10-16 2025-10-15 Formulations d'arni de snca et leurs procédés d'utilisation

Country Status (1)

Country Link
WO (1) WO2026085164A2 (fr)

Citations (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
WO1994002595A1 (fr) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Procede et reactif pour le traitement de maladies chez les animaux
US5445934A (en) 1989-06-07 1995-08-29 Affymax Technologies N.V. Array of oligonucleotides on a solid substrate
US5677195A (en) 1991-11-22 1997-10-14 Affymax Technologies N.V. Combinatorial strategies for polymer synthesis
US5744305A (en) 1989-06-07 1998-04-28 Affymetrix, Inc. Arrays of materials attached to a substrate
US5770722A (en) 1994-10-24 1998-06-23 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US5874219A (en) 1995-06-07 1999-02-23 Affymetrix, Inc. Methods for concurrently processing multiple biological chip assays
WO2012177906A1 (fr) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Dosages et procédés de détermination de l'activité d'un agent thérapeutique chez un sujet
WO2015116658A1 (fr) 2014-01-31 2015-08-06 University Hospitals Systèmes et procédés pour l'administration intrathécale d'agent pharmaceutique
WO2019217459A1 (fr) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Administration extra-hépatique
WO2020132227A2 (fr) 2018-12-19 2020-06-25 Alnylam Pharmaceuticals, Inc. Compositions d'agent d'arni de précurseur de la protéine amyloïde (app) et leurs méthodes d'utilisation
WO2021108291A1 (fr) 2019-11-27 2021-06-03 Alnylam Pharmaceuticals, Inc. Synthèse d'oligonucléotides de 3'-arn
WO2022072447A1 (fr) 2020-10-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Compositions à base d'arni de snca et leurs procédés d'utilisation pour traiter ou prévenir des maladies neurodégénératives associées à snca
WO2023192977A2 (fr) 2022-04-01 2023-10-05 Alnylam Pharmaceuticals, Inc. Compositions d'arnsi ciblant snca pour le traitement d'une maladie associée au snca

Patent Citations (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683202B1 (fr) 1985-03-28 1990-11-27 Cetus Corp
US5445934A (en) 1989-06-07 1995-08-29 Affymax Technologies N.V. Array of oligonucleotides on a solid substrate
US5744305A (en) 1989-06-07 1998-04-28 Affymetrix, Inc. Arrays of materials attached to a substrate
US5677195A (en) 1991-11-22 1997-10-14 Affymax Technologies N.V. Combinatorial strategies for polymer synthesis
WO1994002595A1 (fr) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Procede et reactif pour le traitement de maladies chez les animaux
US5770722A (en) 1994-10-24 1998-06-23 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US5874219A (en) 1995-06-07 1999-02-23 Affymetrix, Inc. Methods for concurrently processing multiple biological chip assays
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
WO2012177906A1 (fr) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Dosages et procédés de détermination de l'activité d'un agent thérapeutique chez un sujet
WO2015116658A1 (fr) 2014-01-31 2015-08-06 University Hospitals Systèmes et procédés pour l'administration intrathécale d'agent pharmaceutique
WO2019217459A1 (fr) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Administration extra-hépatique
WO2020132227A2 (fr) 2018-12-19 2020-06-25 Alnylam Pharmaceuticals, Inc. Compositions d'agent d'arni de précurseur de la protéine amyloïde (app) et leurs méthodes d'utilisation
WO2021108291A1 (fr) 2019-11-27 2021-06-03 Alnylam Pharmaceuticals, Inc. Synthèse d'oligonucléotides de 3'-arn
WO2022072447A1 (fr) 2020-10-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Compositions à base d'arni de snca et leurs procédés d'utilisation pour traiter ou prévenir des maladies neurodégénératives associées à snca
WO2023192977A2 (fr) 2022-04-01 2023-10-05 Alnylam Pharmaceuticals, Inc. Compositions d'arnsi ciblant snca pour le traitement d'une maladie associée au snca

Non-Patent Citations (37)

* Cited by examiner, † Cited by third party
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AKHTAR S.JULIAN RL., TRENDS CELL. BIOL., vol. 2, no. 5, 1992, pages 139 - 144
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BERNSTEIN ET AL., NATURE, vol. 409, 2001, pages 363
BITKO, V. ET AL., NAT. MED., vol. 11, 2005, pages 50 - 55
CHOI ET AL., FEBS LETT., vol. 576, 2004, pages 363 - 368
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DOXAKIS, JBC, 2010
ELBASHIR ET AL., GENES DEV., vol. 15, no. XM_005555421, 2001, pages 188
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