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WO2025151250A3 - Complement factor b-modulating compositions and methods of use thereof - Google Patents

Complement factor b-modulating compositions and methods of use thereof

Info

Publication number
WO2025151250A3
WO2025151250A3 PCT/US2024/060424 US2024060424W WO2025151250A3 WO 2025151250 A3 WO2025151250 A3 WO 2025151250A3 US 2024060424 W US2024060424 W US 2024060424W WO 2025151250 A3 WO2025151250 A3 WO 2025151250A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
complement factor
compositions
modulating compositions
expression
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/US2024/060424
Other languages
French (fr)
Other versions
WO2025151250A2 (en
Inventor
Zhen Li
Christopher CLAEBOE
Feriandas GREBLIKAS
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.)
AdaRx Pharmaceuticals Inc
Original Assignee
AdaRx 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 AdaRx Pharmaceuticals Inc filed Critical AdaRx Pharmaceuticals Inc
Publication of WO2025151250A2 publication Critical patent/WO2025151250A2/en
Publication of WO2025151250A3 publication Critical patent/WO2025151250A3/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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Aspects of the disclosure provide compounds, compositions, and methods for modulating the expression or activity of Complement Factor B (CFB). In some aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB mRNA in a cell or animal. In some aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB protein in a cell or animal.
PCT/US2024/060424 2024-01-09 2024-12-16 Complement factor b-modulating compositions and methods of use thereof Pending WO2025151250A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202463619249P 2024-01-09 2024-01-09
US63/619,249 2024-01-09

Publications (2)

Publication Number Publication Date
WO2025151250A2 WO2025151250A2 (en) 2025-07-17
WO2025151250A3 true WO2025151250A3 (en) 2025-08-21

Family

ID=96387426

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/060424 Pending WO2025151250A2 (en) 2024-01-09 2024-12-16 Complement factor b-modulating compositions and methods of use thereof

Country Status (1)

Country Link
WO (1) WO2025151250A2 (en)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220213486A1 (en) * 2013-12-12 2022-07-07 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2023031359A1 (en) * 2021-09-02 2023-03-09 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
WO2023097291A1 (en) * 2021-11-24 2023-06-01 Adarx Pharmaceuticals, Inc. Complement factor b-modulating compositions and methods of use thereof
WO2023141247A2 (en) * 2022-01-20 2023-07-27 Alexion Pharmaceuticals, Inc. Compositions and methods for inhibiting complement factor b
US20230357776A1 (en) * 2014-05-01 2023-11-09 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating complement factor b expression

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220213486A1 (en) * 2013-12-12 2022-07-07 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US20230357776A1 (en) * 2014-05-01 2023-11-09 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating complement factor b expression
WO2023031359A1 (en) * 2021-09-02 2023-03-09 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
WO2023097291A1 (en) * 2021-11-24 2023-06-01 Adarx Pharmaceuticals, Inc. Complement factor b-modulating compositions and methods of use thereof
WO2023141247A2 (en) * 2022-01-20 2023-07-27 Alexion Pharmaceuticals, Inc. Compositions and methods for inhibiting complement factor b

Also Published As

Publication number Publication date
WO2025151250A2 (en) 2025-07-17

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