[go: up one dir, main page]

ES2142934T3 - Derivado del glicerol, dispositivo y composicion farmaceutica. - Google Patents

Derivado del glicerol, dispositivo y composicion farmaceutica.

Info

Publication number
ES2142934T3
ES2142934T3 ES94907060T ES94907060T ES2142934T3 ES 2142934 T3 ES2142934 T3 ES 2142934T3 ES 94907060 T ES94907060 T ES 94907060T ES 94907060 T ES94907060 T ES 94907060T ES 2142934 T3 ES2142934 T3 ES 2142934T3
Authority
ES
Spain
Prior art keywords
odioleilglicerol
carbamil
lipids
glycerol
derivative
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.)
Expired - Lifetime
Application number
ES94907060T
Other languages
English (en)
Inventor
Junichi Yano
Tadaaki Ohgi
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.)
Nippon Shinyaku Co Ltd
Original Assignee
Nippon Shinyaku Co Ltd
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 Nippon Shinyaku Co Ltd filed Critical Nippon Shinyaku Co Ltd
Application granted granted Critical
Publication of ES2142934T3 publication Critical patent/ES2142934T3/es
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/08Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C271/00Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C271/06Esters of carbamic acids
    • C07C271/08Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms
    • C07C271/10Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C271/16Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by singly-bound oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C271/00Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C271/06Esters of carbamic acids
    • C07C271/08Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms
    • C07C271/10Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C271/20Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms not being part of nitro or nitroso groups
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C279/00Derivatives of guanidine, i.e. compounds containing the group, the singly-bound nitrogen atoms not being part of nitro or nitroso groups
    • C07C279/20Derivatives of guanidine, i.e. compounds containing the group, the singly-bound nitrogen atoms not being part of nitro or nitroso groups containing any of the groups, X being a hetero atom, Y being any atom, e.g. acylguanidines
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02Monothiocarbamic acids; Derivatives thereof
    • C07C333/04Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D295/00Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/12Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
    • C07D295/125Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/13Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/28Phosphorus compounds with one or more P—C bonds
    • C07F9/38Phosphonic acids [RP(=O)(OH)2]; Thiophosphonic acids ; [RP(=X1)(X2H)2(X1, X2 are each independently O, S or Se)]
    • C07F9/40Esters thereof
    • C07F9/4071Esters thereof the ester moiety containing a substituent or a structure which is considered as characteristic
    • C07F9/4075Esters with hydroxyalkyl compounds

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

EL FIN DE LA INVENCION ES PRESENTAR UN DISPOSITIVO QUE CONTIENE LIPIDOS QUE ACTUAN COMO LOS DENOMINADOS LIPOSOMAS CATIONICOS Y QUE PRESENTAN UNA MENOR TOXICIDAD Y LOS LIPIDOS COMO EL CONSTITUYENTE DEL DISPOSITIVO. LOS COMPUESTOS DE LA INVENCION QUEDAN EJEMPLIFICADOS POR 3-O-(4-DIMETILAMINOBUTANOIL)-1,2-ODIOLEILGLICEROL, 3-O-(2-DIMETILAMINOETIL)CARBAMIL-1,2-ODIOLEILGLICEROL, 3-O-(2-DIETILAMINOETIL)CARBAMIL-1,2-ODIOLEILGLICEROL Y 2-O-(2-DIETILAMINOETIL)CARBAMIL-1,3-ODIOLEILGLICEROL. EL DISPOSITIVO CONTIENE ESTOS LIPIDOS Y FOSFOLIPIDOS. EL DISPOSITIVO PERMITE, CUANDO SE ADMINISTRA JUNTO CON, POR EJEMPLO, UN ARN DE DOBLE CEPA, QUE EL ARN MIGRE AL LUGAR DE ACCION DE FORMA SEGURA.
ES94907060T 1993-02-19 1994-02-17 Derivado del glicerol, dispositivo y composicion farmaceutica. Expired - Lifetime ES2142934T3 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP5493993 1993-02-19

Publications (1)

Publication Number Publication Date
ES2142934T3 true ES2142934T3 (es) 2000-05-01

Family

ID=12984615

Family Applications (1)

Application Number Title Priority Date Filing Date
ES94907060T Expired - Lifetime ES2142934T3 (es) 1993-02-19 1994-02-17 Derivado del glicerol, dispositivo y composicion farmaceutica.

Country Status (11)

Country Link
US (2) US6020317A (es)
EP (2) EP0685457B1 (es)
JP (1) JP3189279B2 (es)
KR (2) KR100334858B1 (es)
AT (2) ATE192657T1 (es)
AU (2) AU6044994A (es)
CA (2) CA2156288C (es)
DE (2) DE69424406T2 (es)
ES (1) ES2142934T3 (es)
RU (2) RU2143903C1 (es)
WO (2) WO1994019314A1 (es)

Families Citing this family (287)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996037194A1 (en) * 1995-05-26 1996-11-28 Somatix Therapy Corporation Delivery vehicles comprising stable lipid/nucleic acid complexes
EP0840744A4 (en) 1995-06-07 1999-03-10 Genta Inc CATIONIC LIPIDS BASED ON PHOSPHONIC ACID
AU701106B2 (en) * 1995-06-07 1999-01-21 Promega Biosciences, Inc. Novel carbamate-based cationic lipids
DE69737597D1 (de) * 1996-07-22 2007-05-24 Us Gov Health & Human Serv Vektoren zur inhibierung von viralem und tumorwachstum
FR2759367A1 (fr) * 1997-02-10 1998-08-14 Transgene Sa Glycerolipides cationiques et complexes de glycerolipides cationiques et de substances actives, notamment pour la transfection de cellules
FR2759382A1 (fr) * 1997-02-10 1998-08-14 Transgene Sa Nouveaux composes et compositions les contenant utilisables pour le transfert d'au moins une substance therapeutiquement active, notamment un polynucleotide, dans une cellule cible et utilisation en therapie genique
US6716625B1 (en) 1997-04-16 2004-04-06 Claude Selitrennikoff Histidine kinases of Aspergillus and other fungal species, related compositions, and methods of use
WO1999001579A1 (en) 1997-07-01 1999-01-14 Isis Pharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
TW524693B (en) * 1997-10-16 2003-03-21 Nippon Shinyaku Co Ltd Cancer cell nuclease activator
WO1999048531A1 (fr) * 1998-03-24 1999-09-30 Nippon Shinyaku Co., Ltd. Nouveaux remedes contre l'hepatite
US6043390A (en) * 1998-04-03 2000-03-28 The Regents Of The University Of California Pentaerythritol lipid derivatives and nucleic-acid complexes
EP1469009A2 (en) * 1998-05-21 2004-10-20 Isis Parmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
JP2002515514A (ja) * 1998-05-21 2002-05-28 アイシス・ファーマシューティカルス・インコーポレーテッド オリゴヌクレオチドの局所送逹のための組成物及び方法
WO1999061032A1 (fr) * 1998-05-25 1999-12-02 Nippon Shinyaku Co., Ltd. Procede de production d'une preparation composite contenant de l'acide nucleique
BR0008227A (pt) * 1999-02-15 2001-10-30 Nippon Shinyaku Co Ltd Polinucleotìdeos de cadeia curta e método para apreparação dos mesmos
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
AU2637500A (en) * 1999-12-10 2001-06-18 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US20020055479A1 (en) 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20030176385A1 (en) * 2000-02-15 2003-09-18 Jingfang Ju Antisense modulation of protein expression
US20030134420A1 (en) * 2000-02-18 2003-07-17 Lollo Charles Peter Methods and compositions for gene delivery
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US8044259B2 (en) 2000-08-03 2011-10-25 The Regents Of The University Of Michigan Determining the capability of a test compound to affect solid tumor stem cells
US20080194022A1 (en) * 2000-08-03 2008-08-14 Clarke Michael F Isolation and use of solid tumor stem cells
US6984522B2 (en) 2000-08-03 2006-01-10 Regents Of The University Of Michigan Isolation and use of solid tumor stem cells
US6998115B2 (en) 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US7427394B2 (en) * 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
USRE43612E1 (en) 2000-10-10 2012-08-28 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
WO2002059366A2 (en) * 2000-11-28 2002-08-01 Promega Corporation Rna polymers and uses thereof
GB0106041D0 (en) * 2001-03-12 2001-05-02 Cancer Res Ventures Ltd Lipids and liposomes
CA2451643C (en) 2001-06-21 2012-11-13 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US20030096772A1 (en) 2001-07-30 2003-05-22 Crooke Rosanne M. Antisense modulation of acyl CoA cholesterol acyltransferase-2 expression
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7229774B2 (en) 2001-08-02 2007-06-12 Regents Of The University Of Michigan Expression profile of prostate cancer
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
NZ566396A (en) 2001-10-09 2009-07-31 Isis Pharmaceuticals Inc Antisense modulation of insulin-like growth factor binding protein 5 expressions
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
EP1466016A2 (en) 2002-01-09 2004-10-13 Riken Institute Of Physical And Chemical Research Cancer profiles
DE10207177A1 (de) * 2002-02-19 2003-09-04 Novosom Ag Fakultativ kationische Lipide
US20060193905A1 (en) * 2002-05-14 2006-08-31 University Of Louisville Research Foundation, Inc. Direct cellular energy delivery system
US20050196382A1 (en) * 2002-09-13 2005-09-08 Replicor, Inc. Antiviral oligonucleotides targeting viral families
WO2004024919A1 (en) * 2002-09-13 2004-03-25 Replicor, Inc. Non-sequence complementary antiviral oligonucleotides
EP2272958A1 (en) 2002-09-26 2011-01-12 ISIS Pharmaceuticals, Inc. Modulation of forkhead box O1A expression
ES2417879T3 (es) 2002-11-13 2013-08-09 Genzyme Corporation Modulación antisentido de la expresión de la apolipoproteína B
WO2004044181A2 (en) 2002-11-13 2004-05-27 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein b expression
US8084432B2 (en) * 2003-02-13 2011-12-27 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of pouchitis
US20070042979A1 (en) * 2003-05-30 2007-02-22 Junichi Yano Oligonucleic acid-bearing composite and pharmaceutical composition containing the composite
US20080020990A1 (en) * 2003-05-30 2008-01-24 Nippon Shinyaku Co., Ltd. Oligo Double-Stranded Rna Inhibiting the Expression of Bcl-2 and Pharmaceutical Composition Containing the Same
WO2005005601A2 (en) 2003-06-09 2005-01-20 The Regents Of The University Of Michigan Compositions and methods for treating and diagnosing cancer
EP1648914A4 (en) 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US20070123480A1 (en) * 2003-09-11 2007-05-31 Replicor Inc. Oligonucleotides targeting prion diseases
US7960350B2 (en) 2003-10-24 2011-06-14 Ader Enterprises, Inc. Composition and method for the treatment of eye disease
CA2554779A1 (en) * 2004-02-03 2005-08-18 The Regents Of The University Of Michigan Compositions and methods for characterizing, regulating, diagnosing, and treating cancer
EP1735009A4 (en) * 2004-03-12 2011-03-30 Alnylam Pharmaceuticals Inc RNAI AGENTS TARGETING THE VASCULAR ENDOTHELIUM GROWTH FACTOR (VEGF)
AU2005222965B8 (en) 2004-03-15 2010-07-01 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US7807647B2 (en) * 2004-06-01 2010-10-05 Pronai Therapeutics, Inc. Methods and compositions for cancer therapy
US8815599B2 (en) * 2004-06-01 2014-08-26 Pronai Therapeutics, Inc. Methods and compositions for the inhibition of gene expression
US7498315B2 (en) * 2004-06-01 2009-03-03 Pronai Therapeutics, Inc. Methods and compositions for the inhibition of gene expression
US7297786B2 (en) 2004-07-09 2007-11-20 University Of Iowa Research Foundation RNA interference in respiratory epitheial cells
WO2006022325A1 (ja) * 2004-08-26 2006-03-02 Nippon Shinyaku Co., Ltd. ガラクトース誘導体、薬物担体及び医薬組成物
US7973084B2 (en) 2005-04-28 2011-07-05 Postech Academy-Industrial Foundation Molecular transporters based on alditol or inositol and processes for the preparation thereof
KR100699279B1 (ko) * 2005-04-28 2007-03-23 학교법인 포항공과대학교 당 또는 당 유사체를 골격으로 하는 분자 수송체 및 그의제조방법
US20090069260A1 (en) 2005-05-30 2009-03-12 Nippon Shinyaku Co., Ltd Method for producing a nucleic-acid-containing complex preparation
JP2008546387A (ja) * 2005-06-13 2008-12-25 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 癌を処置および診断するための組成物および方法
US20070099209A1 (en) * 2005-06-13 2007-05-03 The Regents Of The University Of Michigan Compositions and methods for treating and diagnosing cancer
KR100789323B1 (ko) * 2005-07-20 2007-12-28 주식회사 엔지켐 글리세롤 유도체의 위치 선택적 제조방법 및 그 중간체
DE06814528T1 (de) 2005-09-12 2012-01-05 The Regent Of The University Of Michigan Wiederkehrende genfusionen bei prostatakrebs
WO2007047907A2 (en) * 2005-10-14 2007-04-26 The Regents Of The University Of Michigan Dek protein compositions and methods of using the same
US7794951B2 (en) * 2005-10-18 2010-09-14 University Of Massachusetts Medical School SREBP2gc transcription factors and uses thereof
US7723477B2 (en) 2005-10-31 2010-05-25 Oncomed Pharmaceuticals, Inc. Compositions and methods for inhibiting Wnt-dependent solid tumor cell growth
EP2500360B1 (en) 2005-10-31 2015-08-05 Oncomed Pharmaceuticals, Inc. Compositions and methods for diagnosing and treating cancer
JP5129149B2 (ja) 2005-10-31 2013-01-23 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 癌を処置および診断するための組成物および方法
KR20080086440A (ko) * 2005-11-01 2008-09-25 알닐람 파마슈티칼스 인코포레이티드 인플루엔자 바이러스 복제의 RNAi 억제
JP5122474B2 (ja) 2005-12-01 2013-01-16 プロネイ・セラピューティクス・インコーポレイテッド 両性リポソーム製剤
US8229398B2 (en) * 2006-01-30 2012-07-24 Qualcomm Incorporated GSM authentication in a CDMA network
JP5093095B2 (ja) * 2006-03-01 2012-12-05 日本新薬株式会社 ガラクトース誘導体、薬物担体及び医薬組成物
KR101462874B1 (ko) 2006-03-31 2014-11-18 알닐람 파마슈티칼스 인코포레이티드 Eg5 유전자의 발현을 억제하는 이본쇄 리보핵산
JP4812874B2 (ja) * 2006-04-28 2011-11-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド Jcウイルス遺伝子の発現を抑制するための組成物および方法
CA2652770A1 (en) * 2006-05-19 2007-11-29 Alnylam Pharmaceuticals, Inc. Rnai modulation of aha and therapeutic uses thereof
EP2192200B1 (en) 2006-05-22 2012-10-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of IKK-B gene
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
CA2654302A1 (en) * 2006-06-05 2007-12-13 Massachusetts Institute Of Technology Crosslinked, degradable polymers and uses thereof
EP2046266A4 (en) 2006-07-21 2009-11-04 Massachusetts Inst Technology ENDMODICIFIED POLY (BETA AMINO ESTER) AND ITS USE
WO2008030605A2 (en) * 2006-09-08 2008-03-13 The Regents Of The University Of Michigan Herv group ii viruses in lymphoma and cancer
US20100136537A1 (en) * 2006-10-10 2010-06-03 The Regents Of The University Of Michigan Photoreceptor precursor cells
WO2008086807A2 (en) * 2007-01-19 2008-07-24 Exiqon A/S Mediated cellular delivery of lna oligonucleotides
EP2106439B1 (en) 2007-01-24 2014-11-12 The Regents of the University of Michigan Compositions and methods for treating and diagnosing pancreatic cancer
WO2008096690A1 (ja) 2007-02-05 2008-08-14 Nippon Shinyaku Co., Ltd. ポリエチレングリコール誘導体
WO2008109806A2 (en) * 2007-03-08 2008-09-12 Massachusetts Institute Of Technology Electrostatic coating of particles for drug delivery
EP2905336A1 (en) 2007-03-29 2015-08-12 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of a gene from the ebola
KR20100029079A (ko) * 2007-05-15 2010-03-15 헬리콘 테라퓨틱스 인코퍼레이티드 Gpr12의 저해로 인지 질환을 치료하는 방법
AR066984A1 (es) 2007-06-15 2009-09-23 Novartis Ag Inhibicion de la expresion de la subunidad alfa del canal epitelial de sodio (enac) por medio de arni (arn de interferencia)
US20090324596A1 (en) * 2008-06-30 2009-12-31 The Trustees Of Princeton University Methods of identifying and treating poor-prognosis cancers
US10745701B2 (en) 2007-06-28 2020-08-18 The Trustees Of Princeton University Methods of identifying and treating poor-prognosis cancers
PL3009148T3 (pl) 2007-07-02 2019-03-29 Oncomed Pharmaceuticals, Inc. Kompozycje oraz sposoby leczenia i diagnozowania nowotworu
JP5529732B2 (ja) 2007-07-06 2014-06-25 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 前立腺癌における再発性遺伝子融合
US8084057B2 (en) 2007-07-17 2011-12-27 University Of Massachusetts Compositions and methods for wound healing
US8865178B2 (en) 2007-07-17 2014-10-21 University Of Massachusetts Compositions and methods for wound healing
EP2597464B1 (en) 2007-08-16 2015-02-25 The Regents of the University of Michigan Metabolomic profiling of prostate cancer
JP5049713B2 (ja) * 2007-09-14 2012-10-17 株式会社コナミデジタルエンタテインメント ゲームシステム並びにこれを構成するゲーム装置及び課題報知装置
WO2009076400A2 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
EP3100718B1 (en) * 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
US20110118340A1 (en) * 2008-02-08 2011-05-19 Muthiah Manoharan Delivery of rnai constructs to oligodendrocytes
EP2265276A2 (en) * 2008-03-05 2010-12-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 and vegf genes
WO2009129387A2 (en) * 2008-04-16 2009-10-22 Abbott Laboratories Cationic lipids and uses thereof
WO2009129385A1 (en) * 2008-04-16 2009-10-22 Abbott Laboratories Cationic lipids and uses thereof
WO2009153888A1 (ja) 2008-06-19 2009-12-23 日本新薬株式会社 薬物担体
BRPI0912923A8 (pt) 2008-08-25 2017-12-12 Ionis Pharmaceuticals Inc Composto que consiste de um oligonucleotídeo modificadpo composto de 12-30 nucleosídeos ligados, compostos que compreende um oligonucleotídeo modificado que compreende pelo menos 12 nucleosídeos ligados, composto que compreende um oligonucleotídeo modificado que compreende nucleosídeos ligados, e, uso de um composto
EP3208337A1 (en) 2008-09-02 2017-08-23 Alnylam Pharmaceuticals, Inc. Compositions for combined inhibition of mutant egfr and il-6 expression
ES2740129T3 (es) 2008-09-25 2020-02-05 Alnylam Pharmaceuticals Inc Composiciones formuladas en lípidos y métodos de inhibición de la expresión del gen de suero amiloide A
SG190568A1 (en) 2008-09-26 2013-06-28 Oncomed Pharm Inc Frizzled-binding agents and uses thereof
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
CN102264357B (zh) * 2008-12-22 2015-04-01 宝丽化成工业有限公司 黑色素产生抑制剂
CN102639709A (zh) 2009-01-09 2012-08-15 密歇根大学董事会 癌症中的复现性基因融合体
US20120041051A1 (en) 2009-02-26 2012-02-16 Kevin Fitzgerald Compositions And Methods For Inhibiting Expression Of MIG-12 Gene
JP6032724B2 (ja) 2009-03-12 2016-11-30 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. 脂質製剤組成物およびEg5遺伝子とVEGF遺伝子の発現を阻害する方法
EP2756845B1 (en) 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
US20100272824A1 (en) * 2009-04-16 2010-10-28 The Texas A&M University System Compositions and methods for preventing and monitoring disease
EP2429530B1 (en) 2009-05-14 2018-11-07 The General Hospital Corporation (s)-meclizine for treating ischaemia-reperfusion injury
WO2010141630A1 (en) 2009-06-03 2010-12-09 University Of Southern California Compositions and methods for treatment of cancer by disrupting the lh/lhr signaling pathway
ES2527220T5 (es) 2009-07-09 2018-06-05 Marina Biotech, Inc. Liposomas anfóteros que comprenden lípidos imino
AP2015008874A0 (en) 2009-08-14 2015-11-30 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
WO2011025540A1 (en) 2009-08-28 2011-03-03 Bellweather Farms Chemical induction of lactation
US9364495B2 (en) 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
LT3338765T (lt) 2009-12-01 2019-06-25 Translate Bio, Inc. Steroidiniai dariniai, skirti mrnr tiekimui, esant genetinėms ligoms
TWI535445B (zh) 2010-01-12 2016-06-01 安可美德藥物股份有限公司 Wnt拮抗劑及治療和篩選方法
JP2013530929A (ja) 2010-04-01 2013-08-01 オンコメッド ファーマシューティカルズ インコーポレイテッド frizzled結合剤およびその使用
EP2555778A4 (en) 2010-04-06 2014-05-21 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING CD274 / PD-L1 GENE EXPRESSION
US9125931B2 (en) 2010-04-06 2015-09-08 Massachusetts Institute Of Technology Post-transcriptional regulation of RNA-related processes using encoded protein-binding RNA aptamers
CA3102008A1 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
DK2575764T3 (en) * 2010-06-03 2017-08-07 Alnylam Pharmaceuticals Inc BIODEGRADABLE LIPIDS FOR THE ACTIVATION OF ACTIVE AGENTS
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
CN103370054A (zh) 2010-11-09 2013-10-23 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因的表达的脂质配制的组合物和方法
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHOD FOR INCREASING AN ERYTHROPOIETIN (EPO) PREPARATION
AU2012212110A1 (en) 2011-02-02 2013-08-01 Excaliard Pharmaceuticals, Inc. Method of treating keloids or hypertrophic scars using antisense compounds targeting connective tissue growth factor (CTGF)
WO2012109495A1 (en) 2011-02-09 2012-08-16 Metabolic Solutions Development Company, Llc Cellular targets of thiazolidinediones
EP2691443B1 (en) 2011-03-28 2021-02-17 Massachusetts Institute of Technology Conjugated lipomers and uses thereof
CA2831284C (en) 2011-03-29 2023-12-12 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of tmprss6 gene
EP3460064B8 (en) 2011-04-03 2024-03-20 The General Hospital Corporation d/b/a Massachusetts General Hospital Efficient protein expression in vivo using modified rna (mod-rna)
EP4458350A3 (en) 2011-06-08 2025-01-22 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
WO2012177906A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
CN112961855B (zh) 2011-06-21 2024-08-09 阿尔尼拉姆医药品有限公司 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法
EP3597750B1 (en) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
MX363734B (es) 2011-10-27 2019-03-29 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco.
EP2780456A1 (en) 2011-11-17 2014-09-24 The U.S.A. as represented by the Secretary, Department of Health and Human Services Therapeutic rna switches compositions and methods of use
ES2921724T1 (es) 2011-12-07 2022-08-31 Alnylam Pharmaceuticals Inc Lípidos biodegradables para la administración de agentes activos
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
WO2014047232A2 (en) 2012-09-21 2014-03-27 Cornell University C-rel inhibitors and uses thereof
EP2911691B1 (en) 2012-10-23 2018-10-10 OncoMed Pharmaceuticals, Inc. Methods of treating neuroendocrine tumors using wnt pathway-binding agents
PL2929031T4 (pl) 2012-12-05 2022-06-27 Alnylam Pharmaceuticals, Inc. Kompozycje iRNA PCSK9 i sposoby ich zastosowania
JPWO2014112280A1 (ja) * 2013-01-17 2017-01-19 学校法人日本大学 油性組成物及び増粘又はゲル形成剤
US20140200261A1 (en) 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
JP2016510411A (ja) 2013-02-04 2016-04-07 オンコメッド ファーマシューティカルズ インコーポレイテッド Wnt経路インヒビターによる処置の方法およびモニタリング
WO2014184655A1 (en) 2013-02-07 2014-11-20 Olympic Seafood As Methods for using crustacean phospholipid-peptide-protein complexes
WO2014159813A1 (en) 2013-03-13 2014-10-02 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
US9168300B2 (en) 2013-03-14 2015-10-27 Oncomed Pharmaceuticals, Inc. MET-binding agents and uses thereof
SG11201507391VA (en) 2013-03-14 2015-10-29 Shire Human Genetic Therapies Cftr mrna compositions and related methods and uses
EP2970955B1 (en) 2013-03-14 2018-11-14 Translate Bio, Inc. Methods for purification of messenger rna
CN114015692B (zh) 2013-03-14 2024-12-31 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
US10246708B2 (en) 2013-05-06 2019-04-02 Alnylam Pharmaceuticals, Inc. Dosages and methods for delivering lipid formulated nucleic acid molecules
IL285780B (en) 2013-05-22 2022-07-01 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
AU2014268509B2 (en) 2013-05-22 2020-10-01 Alnylam Pharmaceuticals, Inc. Serpina1 iRNA compositions and methods of use thereof
US10126305B2 (en) 2013-06-25 2018-11-13 University of Pittsburg—Of the Commonwealth System of Higher Education Proteomic biomarkers of sepsis in elderly patients
CN105793423A (zh) 2013-10-02 2016-07-20 阿尔尼拉姆医药品有限公司 用于抑制lect2基因表达的组合物和方法
CR20160195A (es) 2013-10-04 2016-09-05 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen alas 1
US9629804B2 (en) 2013-10-22 2017-04-25 Shire Human Genetic Therapies, Inc. Lipid formulations for delivery of messenger RNA
US11224642B2 (en) 2013-10-22 2022-01-18 Translate Bio, Inc. MRNA therapy for argininosuccinate synthetase deficiency
US9522176B2 (en) 2013-10-22 2016-12-20 Shire Human Genetic Therapies, Inc. MRNA therapy for phenylketonuria
CA2931090A1 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
US10059655B2 (en) * 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
EP3960860A3 (en) 2014-02-11 2022-06-08 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
JP6571679B2 (ja) 2014-04-25 2019-09-04 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの精製方法
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療serpinc1相關疾患之方法和組成物
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EA201692370A1 (ru) 2014-05-22 2017-03-31 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ иРНК АНГИОТЕНЗИНОГЕНА (AGT) И СПОСОБЫ ИХ ИСПОЛЬЗОВАНИЯ
JP6557722B2 (ja) 2014-05-30 2019-08-07 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための生分解性脂質
SG11201610670WA (en) 2014-06-24 2017-01-27 Shire Human Genetic Therapies Stereochemically enriched compositions for delivery of nucleic acids
US9840479B2 (en) 2014-07-02 2017-12-12 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
JP6778175B2 (ja) 2014-07-16 2020-10-28 ノバルティス アーゲー 脂質ナノ粒子ホスト中に核酸を封入する方法
WO2016024205A1 (en) 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
WO2016033424A1 (en) 2014-08-29 2016-03-03 Genzyme Corporation Methods for the prevention and treatment of major adverse cardiovascular events using compounds that modulate apolipoprotein b
MA40498A (fr) 2014-09-02 2021-05-19 Texas A & M Univ Sys Méthode de traitement de l'intoxication par organophosphates
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2016069694A2 (en) 2014-10-30 2016-05-06 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
JP7105065B2 (ja) 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ, インコーポレイテッド リガンド修飾二本鎖核酸
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. パタチン様ホスホリパーゼドメイン含有3(PNPLA3)iRNA組成物およびその使用方法
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
MX392757B (es) 2015-04-13 2025-03-24 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico de interferencia (arni) de angiopoyetina-tipo 3 (angptl3) y método de uso de las mismas.
CN108271386B (zh) 2015-05-06 2022-07-15 阿尔尼拉姆医药品有限公司 因子XII(哈格曼因子)(F12)、激肽释放酶B、血浆(夫列契因子)1(KLKB1)和激肽原1(KNG1)iRNA组合物及其使用方法
US9861597B2 (en) 2015-05-19 2018-01-09 Penn State Research Foundation Gluconate-based compositions as a neonate anticonvulsant
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
CA2990172A1 (en) 2015-06-19 2016-12-22 Massachusetts Institute Of Technology Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell
EP3347464B1 (en) 2015-09-08 2024-01-24 University of Massachusetts Dnase h activity of neisseria meningitidis cas9
EP3350328A1 (en) 2015-09-14 2018-07-25 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
WO2017048843A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
WO2017067592A1 (en) 2015-10-21 2017-04-27 Biontech Ag Cytotoxic immunostimulating particles and uses thereof
CA3007014A1 (en) 2015-12-07 2017-06-15 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a serpinc1-associated disorder
EP3387129A1 (en) 2015-12-10 2018-10-17 Alnylam Pharmaceuticals, Inc. STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US10729690B2 (en) 2016-01-13 2020-08-04 University Of Massachusetts Antihelminthic medications for pathogenic nematodes
US10322171B2 (en) 2016-02-19 2019-06-18 The Board Of Regents Of The University Of Texas Systems Vaccine with reduced enhancement of viral infection
WO2017160689A1 (en) 2016-03-15 2017-09-21 University Of Massachusetts Anti-crispr compounds and methods of use
US10213503B2 (en) 2016-05-19 2019-02-26 The Board Of Trustees Of The University Of Texas System Attenuated Burkholderia mallei strain which protects against pathogenic Burkholderia infections, vaccine containing and use thereof
US20190256845A1 (en) 2016-06-10 2019-08-22 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
US11091795B2 (en) 2016-07-11 2021-08-17 Arizona Board Of Regents On Behalf Of The University Of Arizona Compositions and methods for diagnosing and treating arrhythmias
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
JP7058656B2 (ja) 2016-12-16 2022-04-22 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(TTR)iRNA組成物を用いてTTR関連疾患を治療または予防するための方法
US11241436B2 (en) 2017-01-25 2022-02-08 Northwestern University Autophagy inducers for treatment of CNS conditions
CA3054062A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Novel codon-optimized cftr mrna
EP3621645A4 (en) 2017-05-10 2021-01-27 University of Massachusetts BIVALENT VACCINES AGAINST DENGUE / HEPATITIS B
MA49138A (fr) 2017-05-16 2020-03-25 Translate Bio Inc Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr
KR20200024793A (ko) 2017-07-06 2020-03-09 애로우헤드 파마슈티컬스 인코포레이티드 알파-ENaC의 발현을 억제하기 위한 RNAi 작용제 및 사용 방법
JP7277432B2 (ja) 2017-07-13 2023-05-19 アルナイラム ファーマシューティカルズ, インコーポレイテッド 乳酸脱水素酵素A(LDHA)iRNA組成物及びその使用方法
WO2019067782A1 (en) 2017-09-28 2019-04-04 University Of Massachusetts COMBINED TREATMENT OF NEUROPSYCHIATRIC DISORDERS
EP3700965A1 (en) 2017-10-27 2020-09-02 Massachusetts Institute of Technology Poly (beta-amino esters) and uses thereof
WO2019099610A1 (en) 2017-11-16 2019-05-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019103967A1 (en) 2017-11-22 2019-05-31 The Regents Of The University Of Michigan Compositions and methods for treating cancer
CN111727252A (zh) 2017-12-18 2020-09-29 阿尔尼拉姆医药品有限公司 高速泳动族盒-1(HMGB1)iRNA组合物及其使用方法
WO2019136168A1 (en) 2018-01-03 2019-07-11 Penn State Research Foundation Glucose oxidase compositions as a neonate anticonvulsant
US20230132377A9 (en) 2018-05-11 2023-04-27 Alpha Anomeric Sas Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides
US20210154117A1 (en) * 2018-06-29 2021-05-27 Basf Se Esteramine salts
WO2020033791A1 (en) 2018-08-09 2020-02-13 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
US11987792B2 (en) 2018-08-16 2024-05-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
EP3841208A1 (en) 2018-08-24 2021-06-30 Translate Bio, Inc. Methods for purification of messenger rna
US11608412B2 (en) 2018-10-26 2023-03-21 Massachusetts Institute Of Technology Polymer-lipids and compositions
AU2019384557A1 (en) 2018-11-21 2021-06-10 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
US20220306734A1 (en) 2019-07-24 2022-09-29 H. Lundbeck A/S Anti-mglur5 antibodies and uses thereof
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN114616331B (zh) 2019-09-03 2024-10-22 阿尔尼拉姆医药品有限公司 抑制lect2基因表达的组合物和方法
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
KR20220094213A (ko) 2019-11-01 2022-07-05 알닐람 파마슈티칼스 인코포레이티드 헌팅틴 (HTT) iRNA 제제 조성물 및 이의 사용 방법
WO2021102373A1 (en) 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
AU2020402885A1 (en) 2019-12-13 2022-06-16 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (C9orf72) iRNA agent compositions and methods of use thereof
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
US20230136787A1 (en) 2020-02-10 2023-05-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
WO2021173674A1 (en) 2020-02-26 2021-09-02 A2 Biotherapeutics, Inc. Polypeptides targeting mage-a3 peptide-mhc complexes and methods of use thereof
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
TW202204615A (zh) 2020-03-26 2022-02-01 美商阿尼拉製藥公司 冠狀病毒iRNA組成物及其使用方法
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, Inc. Compositions and methods for silencing dnajc15 gene expression
AU2021252545A1 (en) 2020-04-06 2022-11-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CA3179678A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
WO2021222065A1 (en) 2020-04-27 2021-11-04 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
EP4153746A1 (en) 2020-05-21 2023-03-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
WO2021252557A1 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
EP4232581A1 (en) 2020-10-21 2023-08-30 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
TW202237150A (zh) 2020-12-01 2022-10-01 美商艾拉倫製藥股份有限公司 用於抑制hao1(羥基酸氧化酶1(乙醇酸氧化酶))基因表現的方法及組成物
KR20230146048A (ko) 2021-02-12 2023-10-18 알닐람 파마슈티칼스 인코포레이티드 슈퍼옥사이드 디스뮤타제 1(sod1) irna 조성물 및 슈퍼옥사이드 디스뮤타제 1- (sod1-) 관련 신경퇴행성 질환을 치료하거나 예방하기 위한 이의 사용 방법
JP2024509783A (ja) 2021-02-25 2024-03-05 アルナイラム ファーマシューティカルズ, インコーポレイテッド プリオンタンパク質(prnp)irna組成物およびその使用方法
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
CN117561334A (zh) 2021-06-04 2024-02-13 阿尔尼拉姆医药品有限公司 人染色体9开放阅读框72(C9ORF72)iRNA药剂组合物和其使用方法
US20240376499A1 (en) 2021-07-09 2024-11-14 University Of Massachusetts Modular prime editing systems for genome engineering
JP2024528659A (ja) 2021-07-19 2024-07-30 アルナイラム ファーマシューティカルズ, インコーポレイテッド 非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物
TW202334418A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 杭丁頓(HTT)iRNA劑組成物及其使用方法
JPWO2023085440A1 (es) 2021-11-10 2023-05-19
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
CN114907243B (zh) * 2022-02-28 2023-10-27 东南大学 一种可离子化脂质、其组合物及应用
CN116120198B (zh) * 2022-03-21 2024-03-15 苏州科锐迈德生物医药科技有限公司 具有甘油骨架的脂质化合物及基于其的脂质载体、核酸脂质纳米粒组合物和药物制剂
CN116354836A (zh) * 2022-03-25 2023-06-30 深圳市新合生物医疗科技有限公司 阳离子脂质化合物及其制备方法和应用、以及mRNA递送系统
WO2023235501A1 (en) 2022-06-02 2023-12-07 University Of Massachusetts High fidelity nucleotide polymerase chimeric prime editor systems
WO2024025824A2 (en) 2022-07-25 2024-02-01 University Of Massachusetts Nucleic acid antisense oligomer readthrough of nonsense codons
TW202424193A (zh) 2022-09-15 2024-06-16 美商艾拉倫製藥股份有限公司 第13型17β-羥基類固醇去氫酶(HSD17B13)iRNA組成物及其使用方法
WO2025038864A1 (en) * 2023-08-16 2025-02-20 Beam Therapeutics Inc. Ionizable lipids with linear head groups
CN117820149B (zh) * 2023-12-29 2025-01-07 北京剂泰医药科技有限公司 可电离脂质化合物

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) * 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
JPS5933222A (ja) * 1982-08-20 1984-02-23 Mitsui Toatsu Chem Inc 合成デオキシリボ核酸を有効成分とする抗腫瘍剤
JPS60104066A (ja) * 1983-11-10 1985-06-08 Ono Pharmaceut Co Ltd グリセリン誘導体
WO1985002118A1 (en) * 1983-11-21 1985-05-23 Regents Of The University Of Minnesota A buffered polyol-hormone mixture for use in chronic parenteral hormone administration
JPS61103824A (ja) * 1984-10-27 1986-05-22 Nippon Kayaku Co Ltd ポリリボイノシン酸・ポリリボシチヂル酸・ポリ−レ−リジン複合体の注射用製剤の新規調製法
US5049386A (en) * 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797479A (en) * 1985-04-15 1989-01-10 Toyo Jozo Kabushiki Kaisha Nucleoside-phospholipid conjugate
JP2527346B2 (ja) * 1987-01-27 1996-08-21 昭 梶 抗ウイルス剤
US4851232A (en) * 1987-02-13 1989-07-25 Alza Corporation Drug delivery system with means for obtaining desirable in vivo release rate pattern
US5030454A (en) * 1987-10-26 1991-07-09 Alza Corporation Method for delivering drug in tiny pills in liquid carrier
US4961932A (en) * 1987-10-26 1990-10-09 Alza Corporation Plurality of tiny pills in liquid dosage form
US4853229A (en) * 1987-10-26 1989-08-01 Alza Corporation Method for adminstering tiny pills
US4882147A (en) * 1987-12-16 1989-11-21 The Research Foundation Of State University Of New York Novel polynucleotide analogs, methods for inhibiting nucleic acid polymerases and methods for inducing synthesis of interferon
EP0367724B1 (en) * 1988-10-31 1993-02-10 Sandoz Nutrition Ltd. Improvements in or relating to organic compounds
US5231085A (en) * 1988-10-31 1993-07-27 Sandoz Ltd. Compositions and methods for the enhancement of host defense mechanisms
JPH03240795A (ja) * 1990-02-15 1991-10-28 Ajinomoto Co Inc 新規オリゴヌクレオチド誘導体及び抗ウイルス剤への使用
JP2604268B2 (ja) * 1990-04-09 1997-04-30 富士写真フイルム株式会社 ペプチド誘導体両親媒性化合物、その中間体、ペプチド誘導体両親媒性化合物を用いたリポソームおよび薄膜
EP0468520A3 (en) * 1990-07-27 1992-07-01 Mitsui Toatsu Chemicals, Inc. Immunostimulatory remedies containing palindromic dna sequences
JPH04352724A (ja) * 1990-07-27 1992-12-07 Mitsui Toatsu Chem Inc 免疫調節型治療剤
JPH04210921A (ja) * 1991-02-08 1992-08-03 Asahi Chem Ind Co Ltd 新規リン脂質・ヌクレオシド誘導体を有効成分とする抗腫瘍剤

Also Published As

Publication number Publication date
CA2156288A1 (en) 1994-09-01
DE69422163D1 (de) 2000-01-20
KR960700730A (ko) 1996-02-24
DE69422163T2 (de) 2000-06-15
CA2156289C (en) 2006-01-03
CA2156288C (en) 2005-10-18
EP0685457A4 (en) 1996-09-18
WO1994019314A1 (en) 1994-09-01
KR100334858B1 (ko) 2006-01-27
DE69424406T2 (de) 2000-10-26
EP0685234A4 (en) 1997-10-22
DE69424406D1 (de) 2000-06-15
RU2123492C1 (ru) 1998-12-20
CA2156289A1 (en) 1994-09-01
JP3189279B2 (ja) 2001-07-16
WO1994018987A1 (en) 1994-09-01
KR100283601B1 (ko) 2001-03-02
US6020317A (en) 2000-02-01
ATE187713T1 (de) 2000-01-15
EP0685234A1 (en) 1995-12-06
RU2143903C1 (ru) 2000-01-10
EP0685457A1 (en) 1995-12-06
ATE192657T1 (de) 2000-05-15
EP0685457B1 (en) 1999-12-15
KR960700997A (ko) 1996-02-24
US5705188A (en) 1998-01-06
EP0685234B1 (en) 2000-05-10
AU6045094A (en) 1994-09-14
AU6044994A (en) 1994-09-14

Similar Documents

Publication Publication Date Title
ES2142934T3 (es) Derivado del glicerol, dispositivo y composicion farmaceutica.
ES2113867T3 (es) Imino-derivados ciclicos, medicamentos que contienen estos compuestos y procedimiento para su preparacion.
ES2128552T3 (es) Combinacion de un inhibidor de la biosintesis del colesterol y un inhibidor de la absorcion del colesterol beta-lactama.
ES2192201T3 (es) Compuestos de piranona utiles para el tratamiento de infecciones retrovirales.
ES2118160T3 (es) Compuestos de azol, su produccion y su uso.
ES2059317T3 (es) Tolunitrilos alfa-heterociclo-sustituidos.
ES2043625T3 (es) Derivados de acido ariloxicarboxilico, su preparacion y empleo.
GT199700089A (es) Derivados 6.6 o 6.7 -biciclicos sustituidos que contienen pirido o pirimido.
GT199700033A (es) Derivados de 6-fenilpiridil-2-amina.
MX9401315A (es) Derivados ciclicos, medicamentos que contienen estos compuestos y procedimiento para su preparacion.
ES2084168T3 (es) Composiciones antimalaricas.
ES2119325T3 (es) Compuestos polienicos, composiciones farmaceuticas y cosmeticas que los contienen y utilizaciones.
ES2061462T3 (es) Compuestos cefemo, procedimientos para su preparacion y composiciones farmaceuticas que los contienen.
ES2036221T3 (es) Procedimiento para preparar una composicion de l-dopa de dosis unitaria multiple.
ES2093732T3 (es) Nuevos derivados de erucilo, brasidilo y nervonilo.
AR006401A1 (es) Compuestos de pirrolopirrolona, una composicion farmaceutica que los contiene, su uso en la fabricacion de un medicamento y en terapia, procesospara su preparación y compuestos intermediarios para dichos procesos.
CO5050328A1 (es) Sistema solvente para la mejor penetracion de compuestos farmaceuticos
ES461415A2 (es) Procedimiento para la preparacion de vacunas acelulares.
ES2179476T3 (es) Procedimiento para preparar derivados de pirazol.
GT199800068A (es) Procedimiento para la preparacion de nuevos derivados de triazole y los medicamentos que los contienen.
ES2063815T3 (es) 2-alquil-3-benzoilbenzofuranos y composiciones farmaceuticas que los contienen.
ES2172222T3 (es) Derivados de tetrahidropiridopirimidona 3-substituidos, su obtencion y empleo.
ES2166098T3 (es) Composiciones farmaceuticas que contienen acidos 4-oxo-butanoicos.
ES2105923B1 (es) Composiciones para el tratamiento de una infeccion y una enfermedad causadas por el virus de la hepatitis b (hbv).
ES2091283T3 (es) Derivados de isocromano.

Legal Events

Date Code Title Description
FG2A Definitive protection

Ref document number: 685457

Country of ref document: ES