SG11202100928QA - Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy - Google Patents

Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

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Publication number
SG11202100928QA
SG11202100928QA SG11202100928QA SG11202100928QA SG11202100928QA SG 11202100928Q A SG11202100928Q A SG 11202100928QA SG 11202100928Q A SG11202100928Q A SG 11202100928QA SG 11202100928Q A SG11202100928Q A SG 11202100928QA SG 11202100928Q A SG11202100928Q A SG 11202100928QA
Authority
SG
Singapore
Prior art keywords
muscular dystrophy
facioscapulohumeral muscular
muscle targeting
targeting complexes
treating facioscapulohumeral
Prior art date
Application number
SG11202100928QA
Other languages
English (en)
Inventor
Romesh Subramanian
Mohammed Qatanani
Timothy Weeden
Original Assignee
Dyne Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Dyne Therapeutics Inc filed Critical Dyne Therapeutics Inc
Publication of SG11202100928QA publication Critical patent/SG11202100928QA/en

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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
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SG11202100928QA 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy SG11202100928QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862713933P 2018-08-02 2018-08-02
PCT/US2019/044990 WO2020028864A1 (fr) 2018-08-02 2019-08-02 Complexes de ciblage musculaire et leurs utilisations pour le traitement de la dystrophie musculaire facio-scapulo-humérale

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US (9) US20220193250A1 (fr)
EP (1) EP3830259A4 (fr)
JP (1) JP2021533200A (fr)
KR (1) KR20210081324A (fr)
CN (1) CN112912499A (fr)
AU (1) AU2019316103A1 (fr)
CA (1) CA3108289A1 (fr)
EA (1) EA202190416A1 (fr)
IL (1) IL280426A (fr)
MX (1) MX2021001284A (fr)
SG (1) SG11202100928QA (fr)
WO (1) WO2020028864A1 (fr)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3127241A1 (fr) * 2019-01-22 2020-07-30 Korro Bio, Inc. Oligonucleotides d'edition d'arn et leurs utilisations
WO2020154344A1 (fr) 2019-01-22 2020-07-30 Korro Bio, Inc. Oligonucléotides d'édition d'arn et leurs utilisations
AU2021237465A1 (en) * 2020-03-19 2022-10-13 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
CN116096890A (zh) 2020-04-02 2023-05-09 米雷楚莱股份有限公司 使用工程化寡核苷酸的靶向抑制
JP2023541404A (ja) 2020-09-11 2023-10-02 アローヘッド ファーマシューティカルズ インコーポレイテッド ＤＵＸ４の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用方法
WO2022147207A1 (fr) * 2020-12-31 2022-07-07 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour traiter la myopathie facio-scapulo-humérale
EP4359534A1 (fr) * 2021-06-21 2024-05-01 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de la maladie de pompe
WO2023283629A1 (fr) * 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs formulations pour traiter la dystrophie musculaire facio-scapulo-humérale
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2023006985A1 (fr) * 2021-07-30 2023-02-02 4Basebio Uk Ltd Nanoparticules et peptides pour l'administration de charges à des cellules musculaires
EP4124345A1 (fr) * 2021-07-30 2023-02-01 4basebio UK Ltd Nanoparticules pour l'administration musculaire
IL311175A (en) 2021-09-01 2024-04-01 Biogen Ma Inc Anti-transferrin receptor antibodies and their uses
US11912779B2 (en) * 2021-09-16 2024-02-27 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2023086864A1 (fr) * 2021-11-12 2023-05-19 Dyne Therapeutics, Inc. Complexes de ciblage musculaire pour traiter la dystrophie musculaire facio-scapulo-humérale
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy

Family Cites Families (231)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2631173A (en) 1949-01-04 1953-03-10 Phillips Petroleum Co Production of cyclooctadiene
IL54795A (en) 1978-05-26 1980-10-26 Yeda Res & Dev Analgesic compositions comprising substituted bicyclic and spiroaliphatic amines and certain novel compounds of these types
ATE108692T1 (de) 1988-03-02 1994-08-15 Uab Research Foundation Reversibel mechanisch-chemisch arbeitende maschinen, die aus bioelastomeren bestehen, die zu regelbaren inversen temperaturübergängen fähig sind, zur umwandlung von chemischer in mechanische arbeit.
WO1991004753A1 (fr) 1989-10-02 1991-04-18 Cetus Corporation Conjugues d'oligonucleotides non codants et leurs emplois therapeutiques
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US6214345B1 (en) 1993-05-14 2001-04-10 Bristol-Myers Squibb Co. Lysosomal enzyme-cleavable antitumor drug conjugates
US6255455B1 (en) 1996-10-11 2001-07-03 The Trustees Of The University Of Pennsylvania Rh(D)-binding proteins and magnetically activated cell sorting method for production thereof
ATE292119T1 (de) 2000-02-22 2005-04-15 Banyu Pharma Co Ltd Imidazolin-verbindungen
CA2408011A1 (fr) 2000-05-04 2001-11-08 Avi Biopharma, Inc. Composition anti-sens a region d'epissage et methode associee
EP1191097A1 (fr) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction du "exon-skipping" dans des cellules eukaryotes
US20040063654A1 (en) 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
US7241444B2 (en) 2002-01-18 2007-07-10 Pierre Fabre Medicament Anti-IGF-IR antibodies and uses thereof
JP4828795B2 (ja) 2002-03-11 2011-11-30 モメンタ ファーマシューティカルズ インコーポレイテッド 硫酸化多糖類の分析
EP2314691A3 (fr) 2002-11-14 2012-01-18 Dharmacon, Inc. SIRNA fonctionnel et hyperfonctionnel
EP1569661B1 (fr) 2002-11-18 2009-09-09 Santaris Pharma A/S Conception antisens
CA2796924C (fr) 2002-11-25 2016-12-13 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy Produits pharmaceutiques a base d'acide nucleique ena capables de modifier l'epissage de precurseurs d'arnm
EP1572660B1 (fr) 2002-12-20 2011-01-26 X-Ceptor Therapeutics, Inc. Dérives d'isoquinolinone et leur utilisation comme agents thérapeutiques
AU2004209598B2 (en) 2003-02-10 2009-08-06 Enzon Pharmaceuticals, Inc. Oligomeric compounds for the modulation of survivin expression
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
CA2522700A1 (fr) 2003-04-18 2004-11-04 Cytovia, Inc. Procedes pour traiter des maladies entrainant l'induction d'apoptose et essais de criblage
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
WO2005087217A1 (fr) 2004-03-05 2005-09-22 The General Hospital Corporation Compositions et procedes pour moduler l'interaction entre des polypeptides
WO2005113571A2 (fr) 2004-05-13 2005-12-01 The University Of North Carolina At Chapel Hill Methodes de delivrance de composes d'oligomeres
AU2005243410B2 (en) 2004-05-14 2010-04-22 Rosetta Genomics Ltd. Micronas and uses thereof
US7807816B2 (en) 2004-06-28 2010-10-05 University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
US8431558B2 (en) 2004-11-01 2013-04-30 The Regents Of The University Of California Compositions and methods for modification of biomolecules
DK1812031T3 (en) 2004-11-01 2015-09-14 Univ California Compositions and methods for the modification of biomolecules
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20060252107A1 (en) 2005-02-22 2006-11-09 Acucela, Inc. Compositions and methods for diagnosing and treating retinal diseases
US7807648B2 (en) 2005-10-06 2010-10-05 University Of Delaware G-rich polynucleotides for the treatment of Huntington's Disease
JP2007104971A (ja) 2005-10-14 2007-04-26 New Industry Research Organization ミオスタチンのスプライシング制御アンチセンスオリゴヌクレオチド
US20090214531A1 (en) 2006-01-30 2009-08-27 John Mekalanos Methods and Compositions for Treating and Preventing Bacterial Infections
WO2008029281A2 (fr) 2006-02-10 2008-03-13 Invitrogen Corporation Marquage et détection de protéines modifiées post-traductionnellement
US8859752B2 (en) 2006-04-18 2014-10-14 The Trustees Of The University Of Pennsylvania SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP)
WO2007134014A2 (fr) 2006-05-05 2007-11-22 Isis Pharmaceuticals, Inc. Composés et procédés de modulation de l'expression du gcgr
EP1857548A1 (fr) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Procédé et moyen permettant d'induire un saut d'exon
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
ES2620472T5 (es) 2006-10-18 2020-07-09 Ionis Pharmaceuticals Inc Compuestos antisentido
WO2008067480A2 (fr) 2006-11-29 2008-06-05 Nationwide Children's Hospital Inhibition de la myostatine destinée à améliorer le muscle et/ou à améliorer la fonction musculaire
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
EP2149605B1 (fr) 2007-03-22 2013-07-03 Santaris Pharma A/S Composés ARN antagonistes courts pour la modulation de l'ARNm cible
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
US8846639B2 (en) 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
WO2009144481A2 (fr) 2008-05-30 2009-12-03 Isis Innovation Limited Conjugués pour la délivrance de composés biologiquement actifs
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2350281B1 (fr) 2008-10-24 2014-05-14 Sarepta Therapeutics, Inc. Compositions à sauts d'exons multiples pour la dmd
CA2741629C (fr) 2008-10-27 2022-07-05 Academisch Ziekenhuis Leiden Procedes et moyens d'induction du saut de l'exon 45 dans l'arn pre-messager du gene de la dystrophie musculaire de duchenne
US20120046342A1 (en) 2009-04-24 2012-02-23 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US9012139B2 (en) * 2009-05-08 2015-04-21 Curna, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to DMD family
FR2947269B1 (fr) 2009-06-29 2013-01-18 Sanofi Aventis Nouveaux composes anticancereux
WO2011036640A2 (fr) 2009-09-24 2011-03-31 Cellectis Réactifs à base de méganucléase et leurs utilisations pour traiter des maladies génétiques provoquées par des mutations de changement de phase/ non sens
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
WO2011078797A2 (fr) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Oligonucléotides antisens et utilisations de ceux-ci
US8734799B2 (en) 2010-04-13 2014-05-27 The Regents Of The University Of California Unconjugated anti-TfR antibodies and compositions thereof for the treatment of cancer
EP2563753B9 (fr) 2010-04-27 2016-07-06 SynAffix B.V. Composés de cyclooctyne condensés et leur utilisation dans des click-réactions non métalliques
CA2799501C (fr) 2010-05-28 2022-02-15 Sarepta Therapeutics, Inc. Analogues oligonucleotidiques ayant des liaisons modifiees entre sous-unites et/ou des groupes terminaux modifies
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
AR082205A1 (es) 2010-07-12 2012-11-21 Covx Technologies Ireland Ltd Conjugados de anticuerpos multifuncionales
EP3633038A3 (fr) 2010-07-19 2020-07-29 Ionis Pharmaceuticals, Inc. Modulation de l'expression d'une dystrophia myotonica-protéine kinase
EP2606152B1 (fr) 2010-08-18 2016-10-05 Fred Hutchinson Cancer Research Center Procédés de détermination de la présence ou du risque de développement de la dystrophie facio-scapulo-humérale (fshd)
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20120225034A1 (en) 2010-09-02 2012-09-06 Universite De Mons Agents useful in treating facioscapulohumeral muscular dystrophy
EP2673361B1 (fr) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Composés oligomères comprenant des nucléotides bicycliques et leurs utilisations
WO2012109296A1 (fr) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Oligonucléotides antisens
JP2014513946A (ja) 2011-04-22 2014-06-19 プロセンサ テクノロジーズ ビー．ブイ． 筋強直性ジストロフィー１型（ｄｍ１）などのヒト遺伝性疾患の治療、遅延及び／又は予防のための新規の化合物
CN103732259A (zh) 2011-06-24 2014-04-16 森彻斯有限公司 细胞外靶向药物缀合物
AU2012286994B2 (en) * 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
US20140242093A1 (en) 2011-07-29 2014-08-28 Fred Hutchinson Cancer Research Center Methods and compositions for modulating the innate immune response and/or myogenesis in a mammalian subject
US20130066063A1 (en) 2011-09-09 2013-03-14 John Cooke Hodges Bicyclo[6.1.0]non-4-yne regents for chemical modification of oligonucleotides
EP2788026A4 (fr) 2011-12-05 2015-08-05 Univ Duke Immunogènes v1v2
US9725716B2 (en) 2011-12-06 2017-08-08 Ohio State Innovation Foundation and Research Institute at Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
JP6141770B2 (ja) 2011-12-28 2017-06-07 日本新薬株式会社 アンチセンス核酸
WO2013103800A1 (fr) 2012-01-06 2013-07-11 Bioalliance C.V. Anticorps de récepteur d'anti-transferrine et leurs procédés
EP3093293A1 (fr) 2012-02-24 2016-11-16 Stemcentrx, Inc. Anticorps anti dll3 et procédés d'utilisation
WO2013138662A1 (fr) 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Conjugués antisens destinés à diminuer l'expression du dmpk
CN104271741A (zh) 2012-04-23 2015-01-07 普罗森萨科技有限公司 用于治疗神经肌肉障碍的具有改善特性的rna调节寡核苷酸
JP6460983B2 (ja) 2012-07-03 2019-01-30 バイオマリン テクノロジーズ ベー．フェー． 筋ジストロフィー患者の治療のためのオリゴヌクレオチド
EP2684892A1 (fr) 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions et procédés pour la thérapie génique de la dystrophie musculaire de Duchenne
US9695418B2 (en) 2012-10-11 2017-07-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
CN110201186A (zh) 2012-10-23 2019-09-06 西纳福克斯股份有限公司 经修饰的抗体、抗体-缀合物及其制备方法
JP6133431B2 (ja) 2012-11-24 2017-05-24 ハンジョウ ディーエーシー バイオテック シーオー．，エルティディ．Ｈａｎｇｚｈｏｕ Ｄａｃ Ｂｉｏｔｅｃｈ Ｃｏ．，Ｌｔｄ． 親水性連結体及び薬物分子と細胞結合分子との共役反応における親水性連結体の使用
CN110066793A (zh) 2013-03-14 2019-07-30 萨勒普塔医疗公司 用于治疗肌肉萎缩的外显子跳跃组合物
CA2906812A1 (fr) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Compositions ameliorees pour le traitement de la dystrophie musculaire
WO2014189973A2 (fr) 2013-05-20 2014-11-27 Genentech, Inc. Anticorps anti-récepteur de transferrine et procédés d'utilisation
WO2014189370A1 (fr) 2013-05-24 2014-11-27 Stichting Katholieke Universiteit Composés azadibenzocyclooctyne substitués et leur utilisation dans des réactions « click » sans métal
EP3417880A1 (fr) 2013-06-05 2018-12-26 Duke University Édition et régulation géniques à guidage arn
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US20160201064A1 (en) 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
SG11201601375VA (en) 2013-08-28 2016-03-30 Stemcentrx Inc Engineered anti-dll3 conjugates and methods of use
EP3057618B1 (fr) 2013-10-14 2022-12-14 SynAffix B.V. Anticorps glycosynthétisée, anticorps/conjugué et procédés pour leur préparation
US9987373B2 (en) 2013-10-14 2018-06-05 Synaffix B.V. Modified glycoprotein, protein-conjugate and process for the preparation thereof
EP2862928A1 (fr) 2013-10-18 2015-04-22 Université de Strasbourg Inhibiteur de dynamine 2 pour le traitement de myopathies centronucléaires
US10004814B2 (en) 2013-11-11 2018-06-26 Sirna Therapeutics, Inc. Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety
US10344284B2 (en) 2013-11-14 2019-07-09 Lina A. Shehadeh Methods and compositions employing an osteopontin aptamer to deliver nucleic acids into smooth muscle, endothelial, cardiac and progenitor/stem cells
WO2015112016A1 (fr) 2014-01-24 2015-07-30 Synaffix B.V. Procédé de cycloaddition d'un composé 1,3-dippole halogéné avec un (hétéro)cycloalkyne
JP2017505770A (ja) 2014-01-24 2017-02-23 シンアフィックス ビー．ブイ． （ヘテロ）アリール１，３−双極子化合物と（ヘテロ）シクロアルキンとの環化付加のためのプロセス
CN110951732A (zh) 2014-03-12 2020-04-03 日本新药株式会社 反义核酸
WO2015143062A1 (fr) 2014-03-18 2015-09-24 Fred Hutchinson Cancer Research Center Expression génique induite par dux4 en dystrophie musculaire facio-scapulo-humérale (fshd)
JP6750148B2 (ja) 2014-04-25 2020-09-02 公益財団法人野口研究所 糖鎖切断抗体の製造方法及び均一糖鎖抗体
WO2015179741A1 (fr) 2014-05-23 2015-11-26 Genzyme Corporation Inhibition ou régulation à la baisse de la glycogène synthase par la création de codons d'arrêt prématurés à l'aide d'oligonucléotides antisens
CA2951221A1 (fr) 2014-06-17 2015-12-23 Nippon Shinyaku Co., Ltd. Acide nucleique antisens
CA2957618A1 (fr) 2014-08-04 2016-02-11 MiRagen Therapeutics, Inc. Inhibiteurs de myh7b et utilisations associees
JP2017528124A (ja) 2014-08-04 2017-09-28 シンアフィックス ビー．ブイ． ベータ−（１，４）−ｎ−アセチルガラクトサミニルトランスフェラーゼ又はその突然変異体を用いる糖タンパク質の改変方法
US20160075772A1 (en) 2014-09-12 2016-03-17 Regeneron Pharmaceuticals, Inc. Treatment of Fibrodysplasia Ossificans Progressiva
EP3733209A1 (fr) 2014-10-03 2020-11-04 Synaffix B.V. Liaison de sulfamide, leurs conjugués et procédés de préparation
EP3218411B1 (fr) 2014-11-14 2022-01-12 Ossianix, Inc. Récepteurs d'antigène nouveaux variables (vnars) dirigé contre récepteur de transferrine et leur utilisation
JP6779876B2 (ja) 2014-11-19 2020-11-04 ジェネンテック， インコーポレイテッド 抗トランスフェリン受容体抗体及びその使用方法
ES2946247T3 (es) 2014-11-21 2023-07-14 Merck Sharp & Dohme Llc Agonistas parciales del receptor de insulina
WO2016094374A1 (fr) 2014-12-09 2016-06-16 The Board Of Regents Of The University Of Texas System Compositions et méthodes de traitement de l'ataxie de friedreich
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
WO2016138534A2 (fr) 2015-02-27 2016-09-01 Sarepta Therapeutics, Inc. Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide
JP6966162B2 (ja) 2015-04-23 2021-11-10 シンアフィックス ビー．ブイ． β（１，４）−Ｎ−アセチルガラクトサミニルトランスフェラーゼであるか又はそれに由来するグリコシルトランスフェラーゼを用いる糖タンパク質の改変方法
BR112017023862A2 (pt) 2015-05-04 2018-07-17 Cytomx Therapeutics Inc anticorpos anti-cd71, anticorpos anti-cd71 ativáveis, e métodos de uso destes
KR20240035901A (ko) 2015-05-19 2024-03-18 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
HUE053296T2 (hu) 2015-07-22 2021-06-28 Inatherys TFR-elleni antitestek és alkalmazásuk proliferatív és gyulladásos rendellenességek kezelése során
UA123359C2 (uk) 2015-09-15 2021-03-24 Ніппон Шин'Яку Ко., Лтд. Антисенсова нуклеїнова кислота
US20180237775A1 (en) 2015-09-21 2018-08-23 Association Institut De Myologie Antisense oligonucleotides and uses thereof
WO2017049407A1 (fr) 2015-09-23 2017-03-30 UNIVERSITé LAVAL Modification du gène de la dystrophine et ses utilisations
TW201722439A (zh) 2015-10-09 2017-07-01 波濤生命科學有限公司 寡核苷酸組合物及其方法
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
AU2016365834B2 (en) 2015-12-08 2023-09-28 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
EP3413922A1 (fr) 2016-02-08 2018-12-19 SynAffix B.V. Lieurs à sulfamide améliorés pour une utilisation dans des bioconjugués
US20210213138A1 (en) 2016-02-19 2021-07-15 Genisphere Llc Nucleic Acid Carriers And Therapeutic Methods of Use
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
FR3049951A1 (fr) 2016-04-12 2017-10-13 Univ Rabelais Francois Nouvelles constructions peptidiques et leur utilisation dans le traitement de la toxoplasmose
JP7033547B2 (ja) 2016-04-18 2022-03-10 サレプタ セラピューティクス， インコーポレイテッド 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
EP3922260A3 (fr) 2016-05-24 2022-06-15 Merck Sharp & Dohme Corp. Agonistes partiels du récepteur de l'insuline et analogues du glp-1
EP3463386A4 (fr) 2016-06-03 2020-03-04 Wave Life Sciences Ltd. Oligonucléotides, compositions et méthodes associées
EP4212176A1 (fr) 2016-06-20 2023-07-19 Genahead Bio, Inc. Conjugué anticorps-médicament
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
WO2018007475A1 (fr) 2016-07-05 2018-01-11 Biomarin Technologies B.V. Oligonucléotides de commutation ou de modulation d'épissage de pré-arnm comprenant des fragments d'échafaudage bicycliques, présentant des caractéristiques améliorées pour le traitement des troubles d'origine génétique
US20190330626A1 (en) 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
EP3487523B1 (fr) 2016-07-19 2023-09-06 Duke University Applications thérapeutiques de l'édition du génome fondée sur cpf1
EP3516062A1 (fr) 2016-09-21 2019-07-31 Alnylam Pharmaceuticals, Inc. Compositions d'arni de myostatine et procédés d'utilisation associés
WO2018091544A1 (fr) 2016-11-16 2018-05-24 Biomarin Pharmaceutical, Inc. Substances pour le ciblage de divers organes ou tissus sélectionnés
US10947540B2 (en) 2016-11-29 2021-03-16 Association Institut De Myologie Allele-specific silencing therapy for Dynamin 2-related diseases
US20200046854A1 (en) 2016-11-28 2020-02-13 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
US20190364862A1 (en) 2016-12-08 2019-12-05 The Board Of Regents Of The University Of Texas System Dmd reporter models containing humanized duchenne muscular dystrophy mutations
JOP20190166A1 (ar) 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
JP2020505330A (ja) 2017-01-06 2020-02-20 アビディティー バイオサイエンシーズ エルエルシー エクソンスキッピングを誘導する核酸ポリペプチド組成物および方法
NZ760232A (en) 2017-06-07 2023-05-26 Regeneron Pharma Compositions and methods for internalizing enzymes
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CA3099522A1 (fr) 2017-09-19 2019-03-28 Children's National Medical Center Gapmeres et procedes d'utilisation de ces derniers pour le traitement de la dystrophie musculaire
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
AU2018335880A1 (en) 2017-09-22 2020-04-16 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
WO2019067975A1 (fr) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. Polythérapies pour traiter une dystrophie musculaire
EP3668997A4 (fr) 2017-10-02 2021-05-19 Research Institute at Nationwide Children's Hospital Système de déciblage de miarn pour interférence spécifique d'un tissu
CA3077213A1 (fr) 2017-10-04 2019-04-11 Avidity Biosciences, Inc. Compositions d'acide nucleique-polypeptide et utilisations de celles-ci
MA50578A (fr) 2017-11-09 2021-09-15 Vertex Pharma Systèmes crispr/cas pour le traitement de dmd
WO2019113393A1 (fr) 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique
EP3720504A1 (fr) 2017-12-06 2020-10-14 Synaffix B.V. Conjugués d'énédiyne
WO2019126641A2 (fr) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation de l'expression de la frataxine
WO2019136180A2 (fr) 2018-01-04 2019-07-11 Avidity Biosciences Llc Molécules d'acide nucléique hétéroduplex et leurs utilisations
WO2019136216A1 (fr) 2018-01-05 2019-07-11 The Board Of Regents Of The University Of Texas System Compositions crispr/cas9 thérapeutiques et méthodes d'utilisation
US20200370042A1 (en) 2018-01-31 2020-11-26 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes
EP3746082A4 (fr) 2018-02-02 2021-12-08 Fred Hutchinson Cancer Research Center Méthodes permettant de traiter la dystrophie musculaire facio-scapulo-humérale
BR112020016005A2 (pt) 2018-02-07 2020-12-15 Regeneron Pharmaceuticals, Inc. Método de administração de uma proteína terapêutica ao sistema nervoso central, proteína terapêutica multidomínio, polinucleotídeo, vetor de terapia gênica, e, uso de um nucleotídeo que codifica a proteína terapêutica multidomínio
JP2021521140A (ja) 2018-04-12 2021-08-26 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
CA3098266A1 (fr) 2018-05-11 2019-11-14 Alpha Anomeric Sas Conjugues d'oligonucleotides comprenant des nucleosides de glucide 7'-5 '-alpha-anomeriques-bicycliques
AR126019A1 (es) 2018-05-30 2023-09-06 Novartis Ag Anticuerpos frente a entpd2, terapias de combinación y métodos de uso de los anticuerpos y las terapias de combinación
WO2019241385A2 (fr) 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Oligomères induisant un saut d'exon pour la dystrophie musculaire
EP3810775A1 (fr) 2018-06-21 2021-04-28 The Board Of Regents Of The University Of Texas System Correction des délétions de l'exon 43, de l'exon 45 ou de l'exon 52 de la dystrophine dans la dystrophie musculaire de duchenne
EA202190417A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комплексы и их применение для лечения болезни помпе
WO2020028831A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de la fibrodysplasie ossifiante progressive
WO2020028844A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de la myopathie centronucléaire
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3829594A4 (fr) 2018-08-02 2022-05-11 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement d'une cardiomyopathie hypertrophique
EP3829635A4 (fr) 2018-08-02 2022-05-11 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de l'atrophie musculaire
EA202190419A1 (ru) 2018-08-02 2021-06-24 Дайн Терапьютикс, Инк. Мышечно-специфические комплексы и их применение для лечения атаксии фридрейха
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3829596A4 (fr) 2018-08-02 2022-05-11 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et utilisations associées pour le traitement de la dystrophie myotonique
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
JP2021533198A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 筋標的化複合体およびそれらの使用
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
IT201800009682A1 (it) 2018-10-22 2020-04-22 Ice Spa Coniugati di acidi biliari e loro derivati per la veicolazione di molecole attive
EP3876998A1 (fr) 2018-11-05 2021-09-15 Synaffix B.V. Anticorps-conjugués pour le ciblage de tumeurs exprimant trop -2
TW202039573A (zh) 2018-12-21 2020-11-01 美商亞維代堤生物科學公司 抗轉鐵蛋白受體抗體及其用途
CA3137715A1 (fr) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Compositions d'acide nucleique et methodes de saut multi-exon
AU2020287880A1 (en) 2019-06-06 2022-01-20 Avidity Biosciences, Inc. UNA amidites and uses thereof
KR20220024419A (ko) 2019-06-06 2022-03-03 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 이의 용도
EP3980073A4 (fr) 2019-06-07 2023-11-01 Dyne Therapeutics, Inc. Procédés de préparation de complexes protéine-oligonucléotide
WO2021003573A1 (fr) 2019-07-09 2021-01-14 The Governors Of The University Of Alberta Saut des exons 45 à 55 à l'aide de cocktails adaptés aux mutations, de morpholinos antisens, dans le gène dmd
US20240148891A1 (en) 2019-10-18 2024-05-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4087924A4 (fr) 2020-01-10 2024-01-17 Dyne Therapeutics Inc Complexes de ciblage de muscle et leurs utilisations pour traiter la dystrophie musculaire facio-scapulo-humérale
WO2021142260A1 (fr) 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et utilisations associées pour la modulation d'acvr1
WO2021142269A1 (fr) 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Complexes de ciblage de muscle et leurs utilisations pour la modulation de gènes associés à l'atrophie musculaire
US20230203180A1 (en) 2020-01-10 2023-06-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health
JP2023509793A (ja) 2020-01-10 2023-03-09 ダイン セラピューティクス，インコーポレーテッド 筋緊張性ジストロフィーを処置するための筋標的化複合体およびその使用
JP2023510350A (ja) 2020-01-10 2023-03-13 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用
WO2021142331A1 (fr) 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Complexes de ciblage de muscle et leurs utilisations pour la modulation de gènes associés à une maladie du muscle cardiaque
EP4087586A4 (fr) 2020-01-10 2024-05-08 Dyne Therapeutics Inc Complexes de ciblage musculaire et leurs utilisations pour la modulation de gènes associés à la santé musculaire
EP4087876A4 (fr) 2020-01-10 2024-03-27 Dyne Therapeutics Inc Complexes de ciblage de muscle et leurs utilisation
WO2021142217A1 (fr) 2020-01-10 2021-07-15 Dyne Thereapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour la modulation de milck1
WO2021150382A1 (fr) 2020-01-24 2021-07-29 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations dans le traitement de l'atrophie musculaire
BR112022014771A2 (pt) 2020-01-31 2022-10-11 Dyne Therapeutics Inc Anticorpo anti-receptor de transferrina (tfr) e usos do mesmo
WO2021154477A1 (fr) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anticorps anti-récepteur de la transferrine (tfr) et utilisations associées
KR20230046297A (ko) 2020-07-23 2023-04-05 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
AU2021318851A1 (en) 2020-07-23 2023-03-16 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4185330A1 (fr) 2020-07-23 2023-05-31 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations dans le traitement de dystrophinopathies
AU2021312708A1 (en) 2020-07-23 2023-03-16 Dyne Therapeutics, Inc. Anti-transferrin receptor (TFR) antibody and uses thereof
EP4185320A1 (fr) 2020-07-23 2023-05-31 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations dans le traitement de l'atrophie musculaire
AU2021313058A1 (en) 2020-07-23 2023-03-30 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
AR123420A1 (es) 2020-09-01 2022-11-30 Ultragenyx Pharmaceutical Inc Inhibidores de dux4 y métodos de uso de estos
EP4208173A1 (fr) 2020-09-03 2023-07-12 Dyne Therapeutics, Inc. Procédés de préparation de complexes protéine-oligonucléotide
WO2022115745A1 (fr) 2020-11-30 2022-06-02 Research Institute At Nationwide Children's Hospital Compositions et méthodes de traitement de la dystrophie musculaire facio-scapulo-humérale (fshd)
IL303332A (en) 2020-12-04 2023-07-01 Dyne Therapeutics Inc Antibody-oligonucleotide complexes and their uses
JP2024503609A (ja) 2020-12-31 2024-01-26 ダイン セラピューティクス，インコーポレーテッド 筋緊張性ジストロフィーを処置するための筋標的化複合体およびその使用
WO2022147207A1 (fr) 2020-12-31 2022-07-07 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour traiter la myopathie facio-scapulo-humérale
WO2022159712A1 (fr) 2021-01-22 2022-07-28 Ionis Pharmaceuticals, Inc. Composés et méthodes pour réduire l'expression de dux4
EP4359534A1 (fr) 2021-06-21 2024-05-01 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de la maladie de pompe
CA3222816A1 (fr) 2021-06-21 2022-12-29 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leur utilisation pour traiter l'ataxie de friedreich
CA3226365A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
WO2023283629A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs formulations pour traiter la dystrophie musculaire facio-scapulo-humérale
AU2022306307A1 (en) 2021-07-09 2024-02-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4366784A1 (fr) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Complexes ciblant le muscle et formulations de traitement de dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CA3226298A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations dans le traitement de dystrophinopathies
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
CA3226307A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3226367A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations dans le traitement de dystrophinopathies
CA3226361A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour la modulation de genes associes a la sante musculaire
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2023283619A2 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
WO2023044398A1 (fr) 2021-09-16 2023-03-23 Dyne Therapeutics, Inc. Dosage de complexes de ciblage de muscle pour traiter des dystrophinopathies
WO2023077120A1 (fr) 2021-11-01 2023-05-04 Dyne Therapeutics, Inc. Complexes de ciblage musculaire pour traiter des dystrophinopathies
WO2023086864A1 (fr) 2021-11-12 2023-05-19 Dyne Therapeutics, Inc. Complexes de ciblage musculaire pour traiter la dystrophie musculaire facio-scapulo-humérale

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US11787869B2 (en) 2023-10-17
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