WO2012006083A3 - Targeted receptor-mediated sirna - Google Patents

Targeted receptor-mediated sirna Download PDF

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Publication number
WO2012006083A3
WO2012006083A3 PCT/US2011/042170 US2011042170W WO2012006083A3 WO 2012006083 A3 WO2012006083 A3 WO 2012006083A3 US 2011042170 W US2011042170 W US 2011042170W WO 2012006083 A3 WO2012006083 A3 WO 2012006083A3
Authority
WO
WIPO (PCT)
Prior art keywords
cell
cells
ligand
sirna
molecular conjugate
Prior art date
Application number
PCT/US2011/042170
Other languages
French (fr)
Other versions
WO2012006083A2 (en
Inventor
Seunghee Cha
Kaleb Marie Pauley
Original Assignee
University Of Florida Research Foundation, 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 University Of Florida Research Foundation, Inc. filed Critical University Of Florida Research Foundation, Inc.
Publication of WO2012006083A2 publication Critical patent/WO2012006083A2/en
Publication of WO2012006083A3 publication Critical patent/WO2012006083A3/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders

Abstract

Embodiments of this disclosure encompass compositions and methods for the delivery of siRNAs to the cytoplasm of a cell by endocytosis. The compositions comprise molecular conjugates between a receptor ligand, most suitably a small molecule ligand, and an siRNA that when administered to an animal or human subject in need thereof, can modulate or relieve symptoms of diseases such as, but not limited to, Sjogren's Syndrome as well as inhibiting or reducing such cellular processes as apoptosis of glandular acinar cells that trigger or sustain Sjogren's Syndrome. The siRNA moiety can reduce caspase-3 levels when delivered to a salivary or lacrimal cell. The method for delivery of a molecular conjugate to a cell, comprises the steps of contacting with a cell with a molecular conjugate comprising a ligand characterized as having affinity for a surface receptor of the cell, and an siRNA moiety linked to the ligand; and maintaining the cell, or population of said cells, under conditions whereby the ligand specifically binds to a surface receptor of the cell or cells, whereupon the molecular conjugate enters the cell or cells by endocytosis, thereby delivering the siRNA moiety to the cytoplasm of the cell or cells.
PCT/US2011/042170 2010-07-09 2011-06-28 Targeted receptor-mediated sirna WO2012006083A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US36275110P 2010-07-09 2010-07-09
US61/362,751 2010-07-09

Publications (2)

Publication Number Publication Date
WO2012006083A2 WO2012006083A2 (en) 2012-01-12
WO2012006083A3 true WO2012006083A3 (en) 2012-04-12

Family

ID=45441729

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/042170 WO2012006083A2 (en) 2010-07-09 2011-06-28 Targeted receptor-mediated sirna

Country Status (1)

Country Link
WO (1) WO2012006083A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015149056A1 (en) 2014-03-28 2015-10-01 Board Of Regents, The University Of Texas System Inhibition of bruton's tyrosine kinase (btk) in the lung to treat severe lung inflammation and lung injury

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050255120A1 (en) * 2004-05-12 2005-11-17 Simon Michael R Composition and method for introduction of DNA directed RNA interference sequences into targeted cells and tissues
US20070258993A1 (en) * 2003-11-12 2007-11-08 The Austin Research Institute Dna-Carrier Conjugate
WO2008092081A2 (en) * 2007-01-26 2008-07-31 Immune Disease Institute, Inc. Targeted delivery of sirna
WO2010048352A2 (en) * 2008-10-22 2010-04-29 Quark Pharmaceuticals, Inc. Methods for treating eye disorders

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070258993A1 (en) * 2003-11-12 2007-11-08 The Austin Research Institute Dna-Carrier Conjugate
US20050255120A1 (en) * 2004-05-12 2005-11-17 Simon Michael R Composition and method for introduction of DNA directed RNA interference sequences into targeted cells and tissues
WO2008092081A2 (en) * 2007-01-26 2008-07-31 Immune Disease Institute, Inc. Targeted delivery of sirna
WO2010048352A2 (en) * 2008-10-22 2010-04-29 Quark Pharmaceuticals, Inc. Methods for treating eye disorders

Also Published As

Publication number Publication date
WO2012006083A2 (en) 2012-01-12

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