CA3224868A1 - Compositions and methods for silencing myoc expression - Google Patents

Compositions and methods for silencing myoc expression Download PDF

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Publication number
CA3224868A1
CA3224868A1 CA3224868A CA3224868A CA3224868A1 CA 3224868 A1 CA3224868 A1 CA 3224868A1 CA 3224868 A CA3224868 A CA 3224868A CA 3224868 A CA3224868 A CA 3224868A CA 3224868 A1 CA3224868 A1 CA 3224868A1
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CA
Canada
Prior art keywords
nucleotide
myoc
nucleotides
strand
sense strand
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
CA3224868A
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English (en)
French (fr)
Inventor
Adam CASTORENO
Bhaumik A. Pandya
Elena CASTELLANOS-RIZALDOS
Mark K. SCHLEGEL
Vasant R. Jadhav
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
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Alnylam Pharmaceuticals Inc
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CA3224868A1 publication Critical patent/CA3224868A1/en
Pending legal-status Critical Current

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
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    • C12N2310/3513—Protein; Peptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3527—Other alkyl chain
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Chemical Kinetics & Catalysis (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
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CA3224868A 2021-06-28 2022-06-28 Compositions and methods for silencing myoc expression Pending CA3224868A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163215804P 2021-06-28 2021-06-28
US63/215,804 2021-06-28
US202163287404P 2021-12-08 2021-12-08
US63/287,404 2021-12-08
US202263351033P 2022-06-10 2022-06-10
US63/351,033 2022-06-10
PCT/US2022/035266 WO2023278406A1 (en) 2021-06-28 2022-06-28 Compositions and methods for silencing myoc expression

Publications (1)

Publication Number Publication Date
CA3224868A1 true CA3224868A1 (en) 2023-01-05

Family

ID=84692959

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3224868A Pending CA3224868A1 (en) 2021-06-28 2022-06-28 Compositions and methods for silencing myoc expression

Country Status (10)

Country Link
US (1) US20240318175A1 (https=)
EP (1) EP4363587A4 (https=)
JP (1) JP2024526234A (https=)
KR (1) KR20240026216A (https=)
AU (1) AU2022301058A1 (https=)
CA (1) CA3224868A1 (https=)
IL (1) IL309230A (https=)
MX (1) MX2023014976A (https=)
TW (1) TW202317148A (https=)
WO (1) WO2023278406A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240117571A (ko) * 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 돌연변이 마이오실린 질환 모델 및 이의 용도

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005117938A2 (en) * 2004-04-13 2005-12-15 Regents Of The University Of Minnesota Methods of treating ocular conditions
EP2313505A2 (en) * 2008-07-15 2011-04-27 F. Hoffmann-La Roche AG Compositions and methods for inhibiting expression of tgf-beta receptor genes
EP3540061A1 (en) * 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
SG11201702055PA (en) * 2014-09-16 2017-04-27 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
PL4012035T3 (pl) * 2014-09-16 2025-03-24 Genzyme Corporation Wektory wirusowe związane z adenowirusami do leczenia jaskry miocylinowej (myoc)
KR20230008729A (ko) * 2020-04-06 2023-01-16 알닐람 파마슈티칼스 인코포레이티드 Myoc 발현을 사일런싱하기 위한 조성물 및 방법

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Publication number Publication date
JP2024526234A (ja) 2024-07-17
KR20240026216A (ko) 2024-02-27
WO2023278406A1 (en) 2023-01-05
TW202317148A (zh) 2023-05-01
MX2023014976A (es) 2024-02-09
US20240318175A1 (en) 2024-09-26
AU2022301058A1 (en) 2024-01-04
EP4363587A1 (en) 2024-05-08
EP4363587A4 (en) 2025-08-06
IL309230A (en) 2024-02-01

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