AU2018348195B2 - Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation - Google Patents

Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation Download PDF

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AU2018348195B2
AU2018348195B2 AU2018348195A AU2018348195A AU2018348195B2 AU 2018348195 B2 AU2018348195 B2 AU 2018348195B2 AU 2018348195 A AU2018348195 A AU 2018348195A AU 2018348195 A AU2018348195 A AU 2018348195A AU 2018348195 B2 AU2018348195 B2 AU 2018348195B2
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seq
hsd17b13
isoform
protein
missense
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AU2018348195A1 (en
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Noura ABUL-HUSN
Aris BARAS
Xiping CHENG
Frederick Dewey
Jesper Gromada
Claudia SCHURMANN
Tanya Teslovich Dostal
Yurong XIN
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Regeneron Pharmaceuticals Inc
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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2018348195A 2017-10-11 2018-10-11 Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation Active AU2018348195B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025203267A AU2025203267A1 (en) 2017-10-11 2025-05-07 Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762570985P 2017-10-11 2017-10-11
US62/570,985 2017-10-11
PCT/US2018/055377 WO2019075181A1 (en) 2017-10-11 2018-10-11 INHIBITION OF HSD17B13 IN THE TREATMENT OF HEPATIC DISEASE IN PATIENTS EXPRESSING PNPLA3 I148M VARIATION

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AU2025203267A Division AU2025203267A1 (en) 2017-10-11 2025-05-07 Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation

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AU2018348195A1 AU2018348195A1 (en) 2020-04-23
AU2018348195B2 true AU2018348195B2 (en) 2025-05-15

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AU2025203267A Pending AU2025203267A1 (en) 2017-10-11 2025-05-07 Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation

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US (4) US10961583B2 (enExample)
EP (2) EP4234719B1 (enExample)
JP (2) JP7434151B2 (enExample)
KR (2) KR102694809B1 (enExample)
CN (1) CN111183234A (enExample)
AU (2) AU2018348195B2 (enExample)
CA (1) CA3078883A1 (enExample)
IL (2) IL273550B2 (enExample)
MX (1) MX2020003561A (enExample)
RU (1) RU2020115526A (enExample)
SG (1) SG11202003254SA (enExample)
WO (1) WO2019075181A1 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10208081B2 (en) 2014-11-26 2019-02-19 Enanta Pharmaceuticals, Inc. Bile acid derivatives as FXR/TGR5 agonists and methods of use thereof
CN118325899A (zh) 2017-01-23 2024-07-12 瑞泽恩制药公司 Hsd17b13变体及其应用
EP4234719B1 (en) 2017-10-11 2025-11-26 Regeneron Pharmaceuticals, Inc. Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
AU2019403447B2 (en) * 2018-12-21 2023-07-27 Ionis Pharmaceuticals, Inc. Modulators of HSD17B13 expression
WO2021003295A1 (en) 2019-07-02 2021-01-07 Regeneron Pharmaceuticals, Inc. Modulators of hsd17b13 and methods of use thereof
WO2021211959A2 (en) * 2020-04-18 2021-10-21 Inipharm, Inc. Methods and compositions for treating drug induced steatohepatitis
WO2021211981A1 (en) * 2020-04-18 2021-10-21 Inipharm, Inc. Substrate selective hsd17b13 inhibitors and uses thereof
JP7626423B6 (ja) 2020-10-16 2025-03-03 公立大学法人大阪 線維症治療薬
US12018259B2 (en) * 2020-12-23 2024-06-25 Regeneron Pharmaceuticals, Inc. Treatment of liver diseases with cell death inducing DFFA like effector B (CIDEB) inhibitors
CN112626199A (zh) * 2021-01-04 2021-04-09 杭州艾迪康医学检验中心有限公司 检测慢性肝炎转化为肝硬化的风险预测位点的引物和方法
CN118103038A (zh) 2021-08-20 2024-05-28 英安塔制药有限公司 17β-羟基类固醇脱氢酶13型抑制剂及其使用方法
MX2024007463A (es) 2021-12-20 2024-09-02 Regeneron Pharma Metodos para identificar y evaluar la inflamacion hepatica y la fibrosis hepatica en un sujeto mediante la determinacion de una puntuacion estratificada basada en la expresion genetica.
CN114262738B (zh) * 2021-12-31 2023-03-31 吉林大学第一医院 Hsd17b13基因多态性在中重度mafld诊断中的应用
WO2023213284A1 (zh) * 2022-05-06 2023-11-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
IL319486A (en) * 2022-10-07 2025-05-01 Pfizer HSD17B13 Inhibitors and/or Retarders
WO2024173343A1 (en) 2023-02-14 2024-08-22 Regeneron Pharmaceuticals, Inc. Compounds targeting hydroxysteroid 17-beta dehydrogenase (hsd17b) and uses thereof
WO2025012038A1 (en) 2023-07-13 2025-01-16 Enyo Pharma L-lysine salt of 4-chloro-5-[4-(2,6-dichlorophenyl)sulfonylpiperazin-1-yl]-1-benzofuran-2-carboxylic acid and various forms thereof
WO2025012425A1 (en) 2023-07-13 2025-01-16 Enyo Pharma Tromethamine salt of 4-chloro-5-[4-(2,6-dichlorophenyl)sulfonylpiperazin-1-yl]-1-benzofuran-2-carboxylic acid and various forms thereof
WO2025012422A1 (en) 2023-07-13 2025-01-16 Enyo Pharma L-arginine salt of 4-chloro-5-[4-(2,6-dichlorophenyl)sulfonylpiperazin-1-yl]-1-benzofuran-2-carboxylic acid and various forms thereof
WO2025098440A1 (zh) * 2023-11-08 2025-05-15 浙江扬厉医药技术有限公司 抑制PNPLA3表达的双链siRNA以及其缀合物
CN118813778B (zh) * 2024-07-01 2025-10-21 北京泛生子基因科技有限公司 一种试剂盒、使用方法及存储介质

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103520724A (zh) * 2013-10-23 2014-01-22 北京美森生物医药科技有限公司 Hsd17b13蛋白或其编码基因的抑制剂的新用途
WO2017048620A1 (en) * 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof

Family Cites Families (133)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US38170A (en) 1863-04-14 Improvement in lamps
US5294533A (en) 1988-07-05 1994-03-15 Baylor College Of Medicine Antisense oligonucleotide antibiotics complementary to the macromolecular synthesis operon, methods of treating bacterial infections and methods for identification of bacteria
US5135917A (en) 1990-07-12 1992-08-04 Nova Pharmaceutical Corporation Interleukin receptor expression inhibiting antisense oligonucleotides
US5271941A (en) 1990-11-02 1993-12-21 Cho Chung Yoon S Antisense oligonucleotides of human regulatory subunit RI.sub.α of cAMP-dependent protein kinases
US5786138A (en) 1993-01-29 1998-07-28 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Hyperstabilizing antisense nucleic acid binding agents
AU693097B2 (en) 1993-06-04 1998-06-25 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Method for treating kaposi's sarcoma with antisense oligonucleotides
US5578716A (en) 1993-12-01 1996-11-26 Mcgill University DNA methyltransferase antisense oligonucleotides
US5641754A (en) 1994-01-10 1997-06-24 The Board Of Regents Of The University Of Nebraska Antisense oligonucleotide compositions for selectively killing cancer cells
US5624813A (en) 1994-04-21 1997-04-29 Mahant; Vijay K. NAD(P)+ /NAD(P)H based chemiluminescent diagnostics
EP0775204A1 (en) 1994-08-09 1997-05-28 Novartis AG Antitumor antisense oligonucleotides
US5856103A (en) 1994-10-07 1999-01-05 Board Of Regents The University Of Texas Method for selectively ranking sequences for antisense targeting
US5994320A (en) 1995-02-06 1999-11-30 Regents Of The University Of Minnesota Antisense oligonucleotides and methods for treating central nervous system tumors
IT1275862B1 (it) 1995-03-03 1997-10-24 Consiglio Nazionale Ricerche Trascritto antisenso associato ad alcuni tipi di cellule tumorali ed oligodeossinucleotidi sintetici utili nella diagnosi e nel trattamento
US6040296A (en) 1995-06-07 2000-03-21 East Carolina University Specific antisense oligonucleotide composition & method for treatment of disorders associated with bronchoconstriction and lung inflammation
FR2739029B1 (fr) 1995-09-21 1997-11-21 Roussel Uclaf Nouvelle application therapeutique des composes antimineralo-corticoides
WO1997014709A1 (en) 1995-10-13 1997-04-24 F. Hoffmann-La Roche Ag Antisense oligomers
JPH11507245A (ja) 1995-11-21 1999-06-29 アイシーエヌ・ファーマシューティカルズ・インコーポレイテッド Il―8およびil―8受容体に対するアンチセンスオリゴヌクレオチドによる睡瘍増殖の阻害
WO1997020942A1 (en) 1995-12-05 1997-06-12 Piao Yun Shang Hsd17b1 promoter, enhancer, silencer and use thereof
AU708535B2 (en) 1996-02-15 1999-08-05 Cleveland Clinic Foundation, The RNase L activators and antisense oligonucleotides effective to treat RSV infections
US5955590A (en) 1996-07-15 1999-09-21 Worcester Foundation For Biomedical Research Conjugates of minor groove DNA binders with antisense oligonucleotides
US6046004A (en) 1997-02-27 2000-04-04 Lorne Park Research, Inc. Solution hybridization of nucleic acids with antisense probes having modified backbones
JPH1142091A (ja) 1997-07-25 1999-02-16 Toagosei Co Ltd アンチセンス核酸化合物
US6391311B1 (en) 1998-03-17 2002-05-21 Genentech, Inc. Polypeptides having homology to vascular endothelial cell growth factor and bone morphogenetic protein 1
US6046319A (en) 1997-10-22 2000-04-04 University Technologies International, Inc. Antisense oligodeoxynucleotides regulating expression of TNF-α
NZ507435A (en) 1998-03-10 2003-12-19 Genentech Inc Novel polypeptides and nucleic acids with homology to cornichon
RU2000125533A (ru) 1998-03-11 2002-10-20 Эндорешерш, Инк. (Ca) Ингибиторы 17-бета-гидроксистероиддегидрогеназ 5 и 3 типа и способы их применения
US6007995A (en) 1998-06-26 1999-12-28 Isis Pharmaceuticals Inc. Antisense inhibition of TNFR1 expression
US6013522A (en) 1999-02-23 2000-01-11 Isis Pharmaceuticals Inc. Antisense inhibition of human Smad1 expression
US6025198A (en) 1999-06-25 2000-02-15 Isis Pharmaceuticals Inc. Antisense modulation of Ship-2 expression
US6033910A (en) 1999-07-19 2000-03-07 Isis Pharmaceuticals Inc. Antisense inhibition of MAP kinase kinase 6 expression
FR2801218B1 (fr) 1999-11-23 2001-12-28 Hoechst Marion Roussel Inc Compositions pharmaceutiques comprenant de la trimegestone, leurs procedes de preparation ainsi que le conditionnement primaire les renfermant
US7754709B2 (en) 2003-06-10 2010-07-13 Solvay Pharmaceuticals Bv Tetracyclic thiophenepyrimidinone compounds as inhibitors of 17β hydroxysteroid dehydrogenase compounds
US20090169585A1 (en) 2003-10-23 2009-07-02 Resveratrol Partners, Llc Resveratrol-Containing Compositions And Their Use In Modulating Gene Product Concentration Or Activity
US20050158376A1 (en) 2003-10-23 2005-07-21 Sardi William F. Dietary supplement and method of processing same
US20080299042A1 (en) 2004-04-30 2008-12-04 Biogen Idec Ma Inc. Membrane Associated Molecules
EP2423326B1 (en) 2004-05-07 2014-01-08 Celera Corporation Genetic polymorphism associated with liver fibrosis methods of detection and uses thereof
FR2904000A1 (fr) 2006-07-19 2008-01-25 Galderma Res & Dev S N C Snc Modulateurs de hsd17b7 dans le traitement de l'acne ou de l'hyperseborrhee
US20090203602A1 (en) 2006-09-01 2009-08-13 Cohava Gelber Compositions and methods for diagnosis and treatment of type 2 diabetes
US20080300170A1 (en) 2006-09-01 2008-12-04 Cohava Gelber Compositions and methods for diagnosis and treatment for type 2 diabetes
US7951776B2 (en) 2006-09-01 2011-05-31 American Type Culture Collection Methods for treatment of type 1 diabetes
US20100267052A1 (en) 2006-09-01 2010-10-21 American Type Culture Collection Compositions and methods for diagnosis and treatment of type 2 diabetes
WO2008091873A2 (en) 2007-01-24 2008-07-31 Dana-Farber Cancer Institute, Inc. Compositions and methods for the identification, assessment, prevention and therapy of thymic lymphoma or hamartomatous tumours
EP2197434A4 (en) 2007-09-20 2011-08-10 Resveratrol Partners Llc Compositions Containing Resveratrol for Modulating Genetic Product Concentration or Activity
JPWO2009148137A1 (ja) 2008-06-04 2011-11-04 協和発酵キリン株式会社 肥満細胞の脱顆粒を制御する核酸
WO2010028110A2 (en) 2008-09-04 2010-03-11 Board Of Regents, The University Of Texas System Sequence variations in pnpla3 associated with hepatic steatosis
US20100209427A1 (en) 2008-09-24 2010-08-19 Yu Li Lysine acetylation sites
EP2177615A1 (en) 2008-10-10 2010-04-21 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Method for a genome wide identification of expression regulatory sequences and use of genes and molecules derived thereof for the diagnosis and therapy of metabolic and/or tumorous diseases
JPWO2010064702A1 (ja) 2008-12-05 2012-05-10 国立大学法人 東京大学 癌の予後を予測するためのバイオマーカー
US20100266618A1 (en) 2009-03-18 2010-10-21 Children's Hospital Of Eastern Ontario Research Institute Compositions and methods for augmenting activity of oncolytic viruses
WO2010120526A2 (en) 2009-03-31 2010-10-21 Emory University Methods and systems for screening for and diagnosing dna methylation associated with autism spectrum disorders
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
WO2011008348A2 (en) 2009-07-15 2011-01-20 Calimmune Inc. Dual vector for inhibition of human immunodeficiency virus
WO2011006214A1 (en) 2009-07-16 2011-01-20 Peter Maccallum Cancer Institute Method of detecting radiation exposure and adverse toxicity thereto
AU2010324735A1 (en) 2009-11-25 2012-07-12 Nestec S.A. Novel genomic biomarkers for irritable bowel syndrome diagnosis
WO2011084747A2 (en) 2009-12-21 2011-07-14 The Johns Hopkins University Compositions and methods for somatic tissue induced pluripotent stem cells having an endoderm origin
WO2011127561A1 (en) 2010-04-12 2011-10-20 University Health Network Methods and compositions for diagnosing pulmonary fibrosis subtypes and assessing the risk of primary graft dysfunction after lung transplantation
WO2011128096A1 (en) 2010-04-16 2011-10-20 Roche Diagnostics Gmbh Polymorphism markers for predicting response to interleukin-6 receptor-inhibiting monoclonal antibody drug treatment
DK2569446T3 (en) 2010-05-12 2018-10-29 Steven E Schutzer DIAGNOSTIC MARKERS FOR NEUROPsychiatric Disease
US8945847B2 (en) 2010-05-24 2015-02-03 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Methods and kits for ascertaining biosafety of an agent
US20120058088A1 (en) 2010-06-28 2012-03-08 Resveratrol Partners, Llc Resveratrol-Containing Compositions And Methods Of Use
US20120015904A1 (en) 2010-07-14 2012-01-19 Regents Of The University Of California Biomarkers for diagnosis of transient ischemic attacks
WO2012031008A2 (en) 2010-08-31 2012-03-08 The General Hospital Corporation Cancer-related biological materials in microvesicles
WO2012052953A1 (en) 2010-10-20 2012-04-26 Fondazione Centro San Raffaele Del Monte Tabor MiRNA
US9617514B2 (en) 2010-10-26 2017-04-11 Buck Institute For Research On Aging Downregulation of SINE/ALU retrotransposon transcription to induce or restore proliferative capacity and/or pluripotency to a stem cell
CA2817256A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012070014A2 (en) 2010-11-26 2012-05-31 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Identification of novel cell surface markers for pancreatic progenitor cells and definite endodermal cells
EP2652149A2 (en) 2010-12-16 2013-10-23 Norwegian University of Science and Technology (NTNU) Single nucleotide polymorphism associated with risk of insulin resistance development
CA2822462A1 (en) 2010-12-20 2012-06-28 The General Hospital Corporation Polycomb-associated non-coding rnas
EP2704728A4 (en) 2011-05-03 2015-04-22 Dermachip Inc EXPRESSION SIGNATURES OF AGENTS ASSOCIATED WITH SKIN AGING AND GEN NETWORKS
GB201112246D0 (en) 2011-07-15 2011-08-31 Univ Birmingham Diagnosis of alzheimer's disease
US20130079241A1 (en) 2011-09-15 2013-03-28 Jianhua Luo Methods for Diagnosing Prostate Cancer and Predicting Prostate Cancer Relapse
WO2013126565A1 (en) 2012-02-24 2013-08-29 Lunyak Victoria V Downregulation of sine/alu retrotransposon transcription to induce or restore proliferative capacity and/or pluripotency to a stem cell
WO2013166264A2 (en) 2012-05-02 2013-11-07 University Of Georgia Research Foundation, Inc. Methods for altering virus replication
WO2013177060A2 (en) 2012-05-20 2013-11-28 Trustees Of Boston University Methods and systems for monitoring, diagnosing, and treating chronic obstructive polmonary disease
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US20150147761A1 (en) 2012-06-20 2015-05-28 Helmut E. Meyer Specific biomarkers for hepatocellular carcinoma (hcc)
US20140004153A1 (en) 2012-07-02 2014-01-02 Lancell, L.L.C. Use of somatic mutations in the extracellular domain of transmembrane proteins in a patient's tumor as immunogens for active humoral immunotherapy of cancer
US9526720B2 (en) 2012-08-17 2016-12-27 The Broad Institute, Inc. Modulators of hepatic lipoprotein metabolism
US9375433B2 (en) 2012-09-26 2016-06-28 Tangent Reprofiling Limited Modulators of androgen synthesis
BR112015006727B1 (pt) 2012-09-26 2022-08-16 Tangent Reprofiling Limited Uso de uma composição farmacêutica
CN104854242B (zh) 2012-12-05 2018-03-16 阿尔尼拉姆医药品有限公司 PCSK9 iRNA组合物及其使用方法
WO2014089486A1 (en) 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
US20160032395A1 (en) 2013-03-14 2016-02-04 Elai Davicioni Cancer biomarkers and classifiers and uses thereof
EP3431592A1 (en) 2013-03-14 2019-01-23 Translate Bio, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
WO2014144564A2 (en) 2013-03-15 2014-09-18 Veracyte, Inc. Biomarkers for diagnosis of lung diseases and methods of use thereof
US20140329704A1 (en) 2013-03-28 2014-11-06 President And Fellows Of Harvard College Markers for mature beta-cells and methods of using the same
GB201306536D0 (en) 2013-04-10 2013-05-22 Gt Biolog Ltd Polypeptide and immune modulation
US9574241B2 (en) 2013-06-03 2017-02-21 The Trustees Of Columbia University In The City Of New York Recurrent mutations in epigenetic regulators, RHOA and FYN kinase in peripheral T-cell lymphomas
EP3004376B1 (en) 2013-06-03 2018-05-09 Nestec S.A. Methods for diagnosing chronic valvular disease
EP3825406A1 (en) 2013-06-17 2021-05-26 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
US20150079062A1 (en) 2013-07-12 2015-03-19 Patrick J. Casey Method for harvesting, processing, and storage of proteins from the mammalian feto-placental unit and use of such proteins in compositions and medical treatment
US20150079061A1 (en) 2013-07-12 2015-03-19 Patrick J. Casey Method for the harvesting, processing, and storage of proteins from the mammalian feto-placental unit and use of such proteins in compositions and medical treatment
RU2545990C2 (ru) 2013-07-16 2015-04-10 Государственное бюджетное учреждение здравоохранения города Москвы Московский клинический научно-практический центр Департамента здравоохранения города Москвы Способ дифференциальной диагностики стеатоза печени и стеатогепатита
EP3871696B1 (en) 2013-10-22 2025-03-19 Translate Bio, Inc. Lipid formulations for delivery of messenger rna
GB201320061D0 (en) 2013-11-13 2013-12-25 Electrophoretics Ltd Materials nad methods for diagnosis and prognosis of liver cancer
EP3139923B1 (en) 2014-05-09 2023-09-27 Tangent Reprofiling Limited Composition for treating pancreatic adenocarcinoma
EP3155116A4 (en) 2014-06-10 2017-12-27 Massachusetts Institute Of Technology Method for gene editing
US10588980B2 (en) 2014-06-23 2020-03-17 Novartis Ag Fatty acids and their use in conjugation to biomolecules
WO2016004387A1 (en) 2014-07-02 2016-01-07 H. Lee Moffitt Cancer Center And Research Institute, Inc. Gene expression signature for cancer prognosis
WO2016009246A1 (en) 2014-07-14 2016-01-21 Casey Patrick J Method for the harvesting, processing, and storage of proteins from the mammalian feto-placental unit and use of such proteins in compositions and medical treatment
WO2016028638A1 (en) 2014-08-18 2016-02-25 Drexel University Methods, computer-readable media, and systems for assessing samples and wounds, predicting whether a wound will heal, and monitoring effectiveness of a treatment
JP7356788B2 (ja) 2014-11-05 2023-10-05 ベラサイト インコーポレイテッド 機械学習および高次元転写データを使用して経気管支生検における特発性肺線維症を診断するシステムおよび方法
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
CN104698108B (zh) 2015-03-26 2016-11-09 中国药科大学 一种利用固定化酶筛选I型17β羟类固醇脱氢酶抑制剂的方法
EP3159407A1 (en) 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
JP2018536689A (ja) 2015-12-10 2018-12-13 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. ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法
EP3390635A4 (en) 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
EP3390642B1 (en) 2015-12-14 2021-11-24 Cold Spring Harbor Laboratory Compositions for treatment of retinitis pigmentosa 13
EP3389672A4 (en) 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
WO2017106292A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of kidney diseases
CA3005128A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
EP4104867A3 (en) 2015-12-14 2023-03-01 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
WO2017156310A1 (en) 2016-03-09 2017-09-14 Molecular Stethoscope, Inc. Methods and systems for detecting tissue conditions
AR107895A1 (es) 2016-03-16 2018-06-28 Spogen Biotech Inc Métodos para promover la salud de las plantas usando enzimas libres y microorganismos que sobreexpresan enzimas
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
US10036024B2 (en) 2016-06-03 2018-07-31 Purdue Research Foundation siRNA compositions that specifically downregulate expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD)
GB201609977D0 (en) 2016-06-08 2016-07-20 Cancer Rec Tech Ltd Chemosensitivity predictive biomarkers
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
IL267024B2 (en) 2016-12-08 2023-12-01 Intellia Therapeutics Inc Adapted leader RNAs for genomic editing
JP2020500539A (ja) 2016-12-09 2020-01-16 サンガモ セラピューティクス, インコーポレイテッド 標的特異的ヌクレアーゼの送達
CN110582301A (zh) 2016-12-14 2019-12-17 利甘达尔股份有限公司 用于核酸和蛋白质有效负载递送的方法和组合物
CN118325899A (zh) 2017-01-23 2024-07-12 瑞泽恩制药公司 Hsd17b13变体及其应用
WO2018220211A1 (en) 2017-06-02 2018-12-06 Institut National De La Sante Et De La Recherche Medicale (Inserm) Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
EP4234719B1 (en) * 2017-10-11 2025-11-26 Regeneron Pharmaceuticals, Inc. Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
WO2019183329A1 (en) 2018-03-21 2019-09-26 Ionis Pharmaceuticals, Inc. Modulation of hsd17b13 expression
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
EP3802828A4 (en) 2018-06-08 2022-10-26 Intellia Therapeutics, Inc. RNA MODIFIED GUIDES FOR GENE EDITING
WO2019246203A1 (en) 2018-06-19 2019-12-26 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mrna and long nucleic acids
WO2021003295A1 (en) 2019-07-02 2021-01-07 Regeneron Pharmaceuticals, Inc. Modulators of hsd17b13 and methods of use thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103520724A (zh) * 2013-10-23 2014-01-22 北京美森生物医药科技有限公司 Hsd17b13蛋白或其编码基因的抑制剂的新用途
WO2017048620A1 (en) * 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
JIAN-HUA SHEN ET AL: "The rs738409 (I148M) variant of the PNPLA3 gene and cirrhosis: a meta-analysis", JOURNAL OF LIPID RESEARCH, vol. 56, no. 1, 5 November 2014 (2014-11-05), US, pages 167-175, ISSN: 0022-2275, DOI: 10.1194/jlr.M048777 *
KENTARO ONIKI ET AL: "Influence of the PNPLA3 rs738409 Polymorphism on Non-Alcoholic Fatty Liver Disease and Renal Function among Normal Weight Subjects", PLOS ONE, vol. 10, no. 7, 22 July 2015, p0132640, DOI: 10.1371/journal.pone.0132640 *
KIKUKO HOTTA ET AL: "R Association of the rs738409 polymorphism in PNPLA3 with liver damage and the development of nonalcoholic fatty liver disease", 22 December 2010, URL:https://www.ncbi.nlm.nih.gov/pubmed/21176169 *

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