[go: up one dir, main page]

WO1998039359B1 - Dimeric cationic lipids on dicystine basis - Google Patents

Dimeric cationic lipids on dicystine basis

Info

Publication number
WO1998039359B1
WO1998039359B1 PCT/US1998/004349 US9804349W WO9839359B1 WO 1998039359 B1 WO1998039359 B1 WO 1998039359B1 US 9804349 W US9804349 W US 9804349W WO 9839359 B1 WO9839359 B1 WO 9839359B1
Authority
WO
WIPO (PCT)
Prior art keywords
cationic lipids
dicystine
basis
dimeric cationic
alk
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.)
Ceased
Application number
PCT/US1998/004349
Other languages
French (fr)
Other versions
WO1998039359A1 (en
Filing date
Publication date
Priority claimed from US08/812,771 external-priority patent/US6034135A/en
Application filed filed Critical
Priority to CA002281915A priority Critical patent/CA2281915A1/en
Priority to DE69815075T priority patent/DE69815075T2/en
Priority to EP98910189A priority patent/EP0968227B1/en
Priority to AU64488/98A priority patent/AU732333B2/en
Publication of WO1998039359A1 publication Critical patent/WO1998039359A1/en
Publication of WO1998039359B1 publication Critical patent/WO1998039359B1/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Abstract

The present invention provides novel dimeric cationic lipids. The present invention further provides compositions of these lipids with anionic or polyanionic macromolecules, methods for interfering with protein expression in a cell utilizing these compositions and a kit for preparing the same.

Claims

AMENDED CLAIMS[received by the International Bureau on 25 September 1998 (25.09.98); original claim 1 amended; remaining claims unchanged (1 page)]
1. A compound of the formula
Z - S - S - Z wherein Z is
Figure imgf000003_0001
wherein (a) n is 0, 1 or 2; (b) R- is hydroxy, a glyceryl moiety or a lipophilic moiety;
!c) R2 is
(i) -NH- [alk-- H]nl- wherein nl is an integer from 0 to 2 and alk: is an alkylene group of 2 to S carbon atoms;
(ϋ) -[W n2- wherein n2 is an integer from 0 to 3 and each W: is an independently selected amino acid residue;
Figure imgf000003_0002
wherein R4 is hydrogen, alkyl of 1 to 18 carbon atoms optionally mono-, di- or tri-substituted with Y1# Y2 and/or Y3 ;
PCT/US1998/004349 1997-03-06 1998-03-05 Dimeric cationic lipids on dicystine basis Ceased WO1998039359A1 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002281915A CA2281915A1 (en) 1997-03-06 1998-03-05 Dimeric cationic lipids on dicystine basis
DE69815075T DE69815075T2 (en) 1997-03-06 1998-03-05 DICYSTINE-BASED DIMERATION CATIONIC LIPIDS
EP98910189A EP0968227B1 (en) 1997-03-06 1998-03-05 Dimeric cationic lipids on dicystine basis
AU64488/98A AU732333B2 (en) 1997-03-06 1998-03-05 Dimeric cationic lipids on dicystine basis

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/812,771 US6034135A (en) 1997-03-06 1997-03-06 Dimeric cationic lipids
US08/812,771 1997-03-06

Publications (2)

Publication Number Publication Date
WO1998039359A1 WO1998039359A1 (en) 1998-09-11
WO1998039359B1 true WO1998039359B1 (en) 1998-11-26

Family

ID=25210580

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/004349 Ceased WO1998039359A1 (en) 1997-03-06 1998-03-05 Dimeric cationic lipids on dicystine basis

Country Status (6)

Country Link
US (1) US6034135A (en)
EP (1) EP0968227B1 (en)
AU (1) AU732333B2 (en)
CA (1) CA2281915A1 (en)
DE (1) DE69815075T2 (en)
WO (1) WO1998039359A1 (en)

Families Citing this family (142)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000053722A2 (en) * 1999-03-10 2000-09-14 Phogen Limited Delivery of nucleic acids and proteins to cells
IL144828A0 (en) * 1999-03-11 2002-06-30 Fujisawa Pharmaceutical Co A pipecolic acid derivative containing liposome composition
AU6912500A (en) * 1999-08-25 2001-03-19 Gmp Companies, Inc. Enhanced oxygen delivery in mammals, methods and reagents related thereto
WO2001070685A2 (en) * 2000-03-22 2001-09-27 Solulink, Incorporated Hydrazine-based and carbonyl-based bifunctional crosslinking reagents
US6686461B1 (en) * 2000-03-22 2004-02-03 Solulink Bioscience, Inc. Triphosphate oligonucleotide modification reagents and uses thereof
US7102024B1 (en) 2000-08-01 2006-09-05 Schwartz David A Functional biopolymer modification reagents and uses thereof
AU2003245160B2 (en) 2002-06-28 2009-09-24 Arbutus Biopharma Corporation Method and apparatus for producing liposomes
WO2004080406A2 (en) 2003-03-07 2004-09-23 Alnylam Pharmaceuticals Therapeutic compositions
ES2702942T3 (en) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Modified RNAi agents
NZ581166A (en) * 2003-09-15 2011-06-30 Protiva Biotherapeutics Inc Polyethyleneglycol-modified lipid compounds and uses thereof
US7158353B2 (en) * 2003-11-06 2007-01-02 Seagate Technology Llc Magnetoresistive sensor having specular sidewall layers
WO2005121348A1 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP2199298A1 (en) * 2004-11-17 2010-06-23 Protiva Biotherapeutics Inc. Sirna silencing of Apolipoprotein B
EP2395012B8 (en) 2005-11-02 2018-06-06 Arbutus Biopharma Corporation Modified siRNA molecules and uses thereof
EP2231195B1 (en) 2007-12-04 2017-03-29 Arbutus Biopharma Corporation Targeting lipids
ES2535419T3 (en) * 2007-12-27 2015-05-11 Protiva Biotherapeutics Inc. Polo kinase expression silencing using interfering RNA
CA3044134A1 (en) * 2008-01-02 2009-07-09 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
EP2274425A2 (en) 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
CN102119217B (en) 2008-04-15 2015-06-03 普洛体维生物治疗公司 Novel lipid formulations for nucleic acid delivery
EP2743265B1 (en) 2008-10-09 2017-03-15 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
US9023820B2 (en) 2009-01-26 2015-05-05 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein C-III expression
CA2753975C (en) 2009-03-02 2017-09-26 Alnylam Pharmaceuticals, Inc. Nucleic acid chemical modifications
JP5766188B2 (en) 2009-07-01 2015-08-19 プロチバ バイオセラピューティクス インコーポレイティッド Lipid formulations for delivering therapeutic agents to solid tumors
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
WO2011109427A2 (en) 2010-03-01 2011-09-09 Alnylam Pharmaceuticals, Inc. Improving the biological activity of sirna through modulation of its thermodynamic profile
US8455455B1 (en) 2010-03-31 2013-06-04 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes involved in hemorrhagic fever
WO2011123621A2 (en) 2010-04-01 2011-10-06 Alnylam Pharmaceuticals Inc. 2' and 5' modified monomers and oligonucleotides
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
US20130260460A1 (en) 2010-04-22 2013-10-03 Isis Pharmaceuticals Inc Conformationally restricted dinucleotide monomers and oligonucleotides
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
BR112013033260B1 (en) 2011-06-21 2022-06-21 Alnylam Pharmaceuticals Double-stranded ribonucleic acid (dsrna) for inhibiting angptl3 expression, pharmaceutical composition and in vitro method for inhibiting angptl3 expression in a cell
MX385869B (en) 2011-11-18 2025-03-18 Alnylam Pharmaceuticals Inc MODIFIED RNA AGENTS.
SG10201912170WA (en) 2011-11-18 2020-02-27 Alnylam Pharmaceuticals Inc Rnai Agents, Compositions And Methods Of Use Thereof For Treating Transthyretin (TTR) Associated Diseases
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
CN104582732A (en) 2012-06-15 2015-04-29 布里格姆及妇女医院股份有限公司 Compositions for treating cancer and methods for making the same
EP2880162B1 (en) 2012-08-03 2017-07-05 Alnylam Pharmaceuticals, Inc. Modified rnai agents
NZ709013A (en) 2012-12-05 2020-01-31 Alnylam Pharmaceuticals Inc Pcsk9 irna compositions and methods of use thereof
US9428453B2 (en) 2013-01-02 2016-08-30 Rutgers, The State University Of New Jersey Cystine diamide analogs for the prevention of cystine stone formation in cystinuria
KR102905197B1 (en) 2013-03-14 2025-12-31 알닐람 파마슈티칼스 인코포레이티드 Complement component c5 irna compositions and methods of use thereof
KR102486617B1 (en) 2013-05-22 2023-01-12 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 compositions and methods of use thereof
DK2999785T3 (en) 2013-05-22 2018-07-23 Alnylam Pharmaceuticals Inc SERPINA1-IRNA COMPOSITIONS AND PROCEDURES FOR USE THEREOF
AR097738A1 (en) 2013-09-23 2016-04-13 Alnylam Pharmaceuticals Inc METHODS TO TREAT OR PREVENT DISEASES ASSOCIATED WITH TRANSTIRETINE (TTR)
IL314045A (en) 2013-12-12 2024-09-01 Alnylam Pharmaceuticals Inc Complementary component iRNA compositions and methods for using them
US10119136B2 (en) 2014-01-09 2018-11-06 Alnylam Pharmaceuticals, Inc. RNAi agents modified at the 4′-C position
KR20230152154A (en) 2014-02-11 2023-11-02 알닐람 파마슈티칼스 인코포레이티드 KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2015153345A1 (en) 2014-04-03 2015-10-08 Invictus Oncology Pvt. Ltd. Supramolecular combinatorial therapeutics
BR122020023687B1 (en) 2014-05-22 2023-03-07 Alnylam Pharmaceuticals, Inc DOUBLE-STRANDED RIBONUCLEIC ACID (RNAI) AGENT, ITS USES AND PHARMACEUTICAL COMPOSITION
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents and uses thereof
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
JOP20200115A1 (en) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc Compositions And Methods For Inhibition Of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) Gene Expression
WO2016057693A1 (en) 2014-10-10 2016-04-14 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhalation delivery of conjugated oligonucleotide
EP3207138B1 (en) 2014-10-17 2020-07-15 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 (en) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc HEPATITIS B VIRUS (HBV) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP3221451A1 (en) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
EP3256587A2 (en) 2015-02-13 2017-12-20 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CN115322991A (en) 2015-05-06 2022-11-11 阿尔尼拉姆医药品有限公司 Factor XII, kallikrein B, plasma Freuey factor 1 and kininogen 1 iRNA compositions and methods of use thereof
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
CN114525280A (en) 2015-09-02 2022-05-24 阿尔尼拉姆医药品有限公司 iRNA compositions of programmed cell death 1 ligand 1(PD-L1) and methods of use thereof
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
WO2017100542A1 (en) 2015-12-10 2017-06-15 Alnylam Pharmaceuticals, Inc. Sterol regulatory element binding protein (srebp) chaperone (scap) irna compositions and methods of use thereof
JP2019518028A (en) 2016-06-10 2019-06-27 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. Complement component C5i RNA composition and its use for treating paroxysmal nocturnal hemoglobinuria (PNH)
TWI788312B (en) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
AU2017376950B2 (en) 2016-12-16 2024-02-22 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing TTR-associated diseases using transthyretin (TTR) iRNA compositions
MA50278A (en) 2017-04-18 2020-02-26 Alnylam Pharmaceuticals Inc METHODS FOR THE TREATMENT OF SUBJECTS INFECTED WITH THE HEPATITIS B VIRUS (HBV) INFECTION
WO2018209130A1 (en) 2017-05-11 2018-11-15 Tc1 Llc Thermal interconnect for implantable blood pump
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
EP3728281A1 (en) 2017-12-21 2020-10-28 Alnylam Pharmaceuticals Inc. Chirally-enriched double-stranded rna agents
JP2022500003A (en) 2018-09-18 2022-01-04 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. Ketohexokinase (KHK) iRNA composition and its usage
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. THERAPEUTIC METHODS FOR TRINUCLEOTIDE REPEAT EXPANSION DISEASES ASSOCIATED WITH MLH3 ACTIVITY
WO2020117840A2 (en) 2018-12-05 2020-06-11 Empirico Inc. Process to inhibit or eliminate eosinophilic diseases of the airway and related conditions
JP2022515744A (en) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ, インコーポレイテッド Compositions and Methods for the Treatment of KCNT1-Related Disorders
EP3911632A1 (en) 2019-01-15 2021-11-24 Empirico Inc. Prodrugs of alox-15 inhibitors and methods of using the same
JP2022532652A (en) 2019-05-17 2022-07-15 アルニラム ファーマスーティカルズ インコーポレイテッド Oral delivery of oligonucleotides
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
TW202132567A (en) 2019-11-01 2021-09-01 美商阿尼拉製藥公司 Huntingtin (htt) irna agent compositions and methods of use thereof
WO2021102373A1 (en) 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
BR112022011417A2 (en) 2019-12-13 2022-08-30 Alnylam Pharmaceuticals Inc COMPOSITIONS OF THE IRNA AGENT OF THE OPEN READING PHASE 72 OF HUMAN CHROMOSOME 9 (C9ORF72) AND METHODS OF USE THEREOF
BR112022011572A2 (en) 2019-12-13 2022-10-04 Univ Rutgers CYSTINE DIAMIDE ANALOGS FOR CYSTINURIA
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)
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
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
US12534731B2 (en) 2020-04-01 2026-01-27 Alnylam Pharmaceuticals, Inc. Alpha-2A adrenergic receptor (ADRA2A) iRNA agent 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
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
CA3181400A1 (en) 2020-04-27 2021-11-04 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
US20230203496A1 (en) 2020-06-09 2023-06-29 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
CN116113697A (en) 2020-07-10 2023-05-12 国家健康与医学研究院 Methods and compositions for treating epilepsy
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
TW202229552A (en) 2020-10-05 2022-08-01 美商艾拉倫製藥股份有限公司 G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
US11591544B2 (en) 2020-11-25 2023-02-28 Akagera Medicines, Inc. Ionizable cationic lipids
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
KR20230146048A (en) 2021-02-12 2023-10-18 알닐람 파마슈티칼스 인코포레이티드 Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-)-related neurodegenerative diseases
EP4298220A1 (en) 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods of use thereof
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna 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
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
JP2024521907A (en) 2021-06-04 2024-06-04 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 Human chromosome 9 open reading frame 72 (C9ORF72) iRNA agent compositions and methods of use thereof
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
EP4363574A1 (en) 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
CA3226878A1 (en) 2021-07-23 2023-01-26 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
CA3233101A1 (en) 2021-09-24 2023-03-30 Alnylam Pharmaceuticals, Inc. Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof
US20250352667A1 (en) 2021-10-22 2025-11-20 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
CA3242118A1 (en) 2021-12-22 2023-06-29 Alfica Sehgal Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
CN119562806A (en) 2022-05-25 2025-03-04 阿卡格拉医药公司 Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
JP2025522380A (en) 2022-06-10 2025-07-15 キャンプ4 セラピューティクス コーポレイション Methods for modulating progranulin expression using antisense oligonucleotides targeted to regulatory RNA
MA71735A (en) 2022-09-15 2025-05-30 Regeneron Pharmaceuticals, Inc. 17B-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) RNAI COMPOSITIONS AND METHODS OF USE THEREOF
AU2023403208A1 (en) 2022-12-01 2025-06-19 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025255388A1 (en) 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4897355A (en) * 1985-01-07 1990-01-30 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
FR2645866B1 (en) * 1989-04-17 1991-07-05 Centre Nat Rech Scient NEW LIPOPOLYAMINES, THEIR PREPARATION AND THEIR USE
ATE154246T1 (en) * 1990-07-27 1997-06-15 Isis Pharmaceuticals Inc NUCLEASE RESISTANT PYRIMIDINE MODIFIED OLIGONUCLEOTIDES THAT DETECTE AND MODULATE GENE EXPRESSION
WO1992009705A1 (en) * 1990-11-23 1992-06-11 Gilead Sciences, Inc. Triplex-forming oligomers containing modified bases
US5777153A (en) * 1994-07-08 1998-07-07 Gilead Sciences, Inc. Cationic lipids
JPH11507352A (en) * 1995-06-07 1999-06-29 ジンタ・インコーポレイテッド New carbamate-based cationic lipids
JPH11510489A (en) * 1995-07-21 1999-09-14 ジンタ・インコーポレイテッド New amide-based cationic lipid
CA2263134A1 (en) * 1996-09-27 1998-04-02 Biomolecular Research Institute Ltd. Cytotoxic peptides

Similar Documents

Publication Publication Date Title
WO1998039359B1 (en) Dimeric cationic lipids on dicystine basis
WO1998039358B1 (en) Oligomeric cationic lipids
WO2000012509B1 (en) Collections of compounds
Safer et al. Undecagold clusters for site-specific labeling of biological macromolecules: simplified preparation and model applications
EP1815859A3 (en) Halimide, a cytotoxic marine natural product, and derivatives thereof
CA2105014A1 (en) Imidazolidine derivatives
RU94040390A (en) Derivatives of vinyleneazaindole, method of their synthesis, pharmaceutical composition
NZ326238A (en) Compositons and methods for inhibiting nonenzymatic cross-linking and glycosylation (protein aging) using N-acylaminoalkylhydrazine carboximidamides
CA2153979A1 (en) Compounds having both potent calcium antagonist and antioxidant activity and use thereof as cytoprotective agents
CA2180205A1 (en) Triazinyl or pyrimidinyl group-containing amino-silicones
WO1994020463B1 (en) Novel distamycin analogues
CA2446622A1 (en) Isostearic acid salts as permeation enhancers
CA2299429A1 (en) New lipopolyamines,their preparation and the use thereof
KR910016771A (en) Lipopeptide Derivatives
CA2489576A1 (en) Compositions and methods for darkening the skin
AU9479098A (en) Enhanced efficacy stable antiperspirant active solution and method of making same
RU2205186C2 (en) Labeled analogues of glutamine and lysine
ZA858169B (en) New adamantanamine derivatives,processes for their preparation and drugs in which they are present
CA2334821A1 (en) Chemical synthesis of morpholine derivatives
TW284765B (en)
TR199800403A2 (en) I won during the preparation of a-Bromo-lactam tablets.
TR200504091T2 (en) (7alpha, 17alpha) -17-hydroxy-7-methyl-19-nor-17-pregn-5 (10) -en 20-yn-3-one.
KR910000775A (en) Muramil Dipeptide Derivatives
EP1057825A4 (en) Isoflavane derivatives and immunopotentiating compositions containing the same
CA2217026A1 (en) New antiviral substituted pyrimidinedione homocarbocyclic nucleoside derivatives and methods for the preparation thereof and compositions containing the same as active ingredients