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WO2025029913A3 - Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof - Google Patents

Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof Download PDF

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Publication number
WO2025029913A3
WO2025029913A3 PCT/US2024/040375 US2024040375W WO2025029913A3 WO 2025029913 A3 WO2025029913 A3 WO 2025029913A3 US 2024040375 W US2024040375 W US 2024040375W WO 2025029913 A3 WO2025029913 A3 WO 2025029913A3
Authority
WO
WIPO (PCT)
Prior art keywords
galnac
conjugates
oligonucleotides
galactosamine
acetyl
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/US2024/040375
Other languages
French (fr)
Other versions
WO2025029913A4 (en
WO2025029913A2 (en
Inventor
Saul MARTINEZ MONTERO
Kevin J. Kim
Jing Yang
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.)
Basecure Therapeutics LLC
Original Assignee
Basecure Therapeutics LLC
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
Priority claimed from PCT/US2024/019424 external-priority patent/WO2024187193A2/en
Application filed by Basecure Therapeutics LLC filed Critical Basecure Therapeutics LLC
Publication of WO2025029913A2 publication Critical patent/WO2025029913A2/en
Publication of WO2025029913A3 publication Critical patent/WO2025029913A3/en
Publication of WO2025029913A4 publication Critical patent/WO2025029913A4/en
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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • C07H15/08—Polyoxyalkylene derivatives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/26—Acyclic or carbocyclic radicals, substituted by hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical

Landscapes

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

Abstract

The present disclosure provides, in part, conjugates of N-actylgalactosamine (GalNAc) and oligonucleotides are their methods of use in inhibiting gene expression and the treatment of various disorders.
PCT/US2024/040375 2023-07-31 2024-07-31 Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof Pending WO2025029913A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202363516762P 2023-07-31 2023-07-31
US63/516,762 2023-07-31
USPCT/US2024/019424 2024-03-11
PCT/US2024/019424 WO2024187193A2 (en) 2023-03-09 2024-03-11 Compositions and methods for inhibition of expression of angiotensinogen (agt) genes

Publications (3)

Publication Number Publication Date
WO2025029913A2 WO2025029913A2 (en) 2025-02-06
WO2025029913A3 true WO2025029913A3 (en) 2025-04-10
WO2025029913A4 WO2025029913A4 (en) 2025-06-19

Family

ID=94395991

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/040375 Pending WO2025029913A2 (en) 2023-07-31 2024-07-31 Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof

Country Status (1)

Country Link
WO (1) WO2025029913A2 (en)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230016929A1 (en) * 2019-11-06 2023-01-19 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2023014765A1 (en) * 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
WO2023014677A1 (en) * 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
WO2023034983A2 (en) * 2021-09-03 2023-03-09 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
US20230159929A1 (en) * 2021-09-08 2023-05-25 Aligos Therapeutics, Inc. MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) MOLECULES AND USES THEREOF

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230016929A1 (en) * 2019-11-06 2023-01-19 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2023014677A1 (en) * 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
WO2023014765A1 (en) * 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
WO2023034983A2 (en) * 2021-09-03 2023-03-09 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
US20230159929A1 (en) * 2021-09-08 2023-05-25 Aligos Therapeutics, Inc. MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) MOLECULES AND USES THEREOF

Also Published As

Publication number Publication date
WO2025029913A4 (en) 2025-06-19
WO2025029913A2 (en) 2025-02-06

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