CA3091789A1 - Camk2d antisense oligonucleotides and uses thereof - Google Patents

Camk2d antisense oligonucleotides and uses thereof Download PDF

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Publication number
CA3091789A1
CA3091789A1 CA3091789A CA3091789A CA3091789A1 CA 3091789 A1 CA3091789 A1 CA 3091789A1 CA 3091789 A CA3091789 A CA 3091789A CA 3091789 A CA3091789 A CA 3091789A CA 3091789 A1 CA3091789 A1 CA 3091789A1
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CA
Canada
Prior art keywords
seq
aso
camk2d
nucleotides
cell
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.)
Pending
Application number
CA3091789A
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English (en)
French (fr)
Inventor
Richard E. Olson
Brian R. Anderson
Peter Hagedorn
Marianne Lerbech Jensen
Ivar M. Mcdonald
Stephen E. Mercer
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Roche Innovation Center Copenhagen AS
Bristol Myers Squibb Co
Original Assignee
Roche Innovation Center Copenhagen AS
Bristol Myers Squibb Co
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Filing date
Publication date
Application filed by Roche Innovation Center Copenhagen AS, Bristol Myers Squibb Co filed Critical Roche Innovation Center Copenhagen AS
Publication of CA3091789A1 publication Critical patent/CA3091789A1/en
Pending legal-status Critical Current

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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0607—Non-embryonic pluripotent stem cells, e.g. MASC
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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CA3091789A 2018-02-21 2019-02-21 Camk2d antisense oligonucleotides and uses thereof Pending CA3091789A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862633502P 2018-02-21 2018-02-21
US62/633,502 2018-02-21
US201862635954P 2018-02-27 2018-02-27
US62/635,954 2018-02-27
US201862665998P 2018-05-02 2018-05-02
US62/665,998 2018-05-02
US201862778679P 2018-12-12 2018-12-12
US62/778,679 2018-12-12
PCT/US2019/018947 WO2019165067A1 (en) 2018-02-21 2019-02-21 Camk2d antisense oligonucleotides and uses thereof

Publications (1)

Publication Number Publication Date
CA3091789A1 true CA3091789A1 (en) 2019-08-29

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CA3091789A Pending CA3091789A1 (en) 2018-02-21 2019-02-21 Camk2d antisense oligonucleotides and uses thereof

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Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE524121T1 (de) 2004-11-24 2011-09-15 Abdou Samy Vorrichtungen zur platzierung eines orthopädischen intervertebralen implantats
US8764806B2 (en) 2009-12-07 2014-07-01 Samy Abdou Devices and methods for minimally invasive spinal stabilization and instrumentation
US11998184B2 (en) * 2010-03-11 2024-06-04 Globus Medical, Inc Tissue retractor and methods of use
US8845728B1 (en) 2011-09-23 2014-09-30 Samy Abdou Spinal fixation devices and methods of use
US20130226240A1 (en) 2012-02-22 2013-08-29 Samy Abdou Spinous process fixation devices and methods of use
US9198767B2 (en) 2012-08-28 2015-12-01 Samy Abdou Devices and methods for spinal stabilization and instrumentation
US9320617B2 (en) 2012-10-22 2016-04-26 Cogent Spine, LLC Devices and methods for spinal stabilization and instrumentation
US10857003B1 (en) 2015-10-14 2020-12-08 Samy Abdou Devices and methods for vertebral stabilization
US10898175B2 (en) 2016-10-04 2021-01-26 Jgmg Bengochea, Llc Retractor extension clip systems
US10744000B1 (en) 2016-10-25 2020-08-18 Samy Abdou Devices and methods for vertebral bone realignment
US10973648B1 (en) 2016-10-25 2021-04-13 Samy Abdou Devices and methods for vertebral bone realignment
KR20200140805A (ko) 2018-02-21 2020-12-16 브리스톨-마이어스 스큅 컴퍼니 Camk2d 안티센스 올리고뉴클레오티드 및 그의 용도
US11806250B2 (en) 2018-02-22 2023-11-07 Warsaw Orthopedic, Inc. Expandable spinal implant system and method of using same
US11179248B2 (en) 2018-10-02 2021-11-23 Samy Abdou Devices and methods for spinal implantation
KR20200071198A (ko) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Nrf2 발현 조절 기반 T 세포 항암면역치료법
US11602337B2 (en) 2019-04-24 2023-03-14 Nuvasive, Inc. Transmission assembly
US11547395B2 (en) 2019-06-18 2023-01-10 Nuvasive, Inc. Tissue retraction system
US11253243B2 (en) 2019-06-18 2022-02-22 Nuvasive, Inc. Tissue retraction system
CN110731803B (zh) * 2019-11-20 2021-05-25 吕振乾 一种心外科手术辅助器
US11944286B2 (en) * 2020-01-24 2024-04-02 Lsi Solutions, Inc. Surgical rib retractor
WO2021158810A1 (en) 2020-02-05 2021-08-12 Bristol-Myers Squibb Company Oligonucleotides for splice modulation of camk2d
US11224415B1 (en) * 2020-07-10 2022-01-18 Warsaw Orthopedic, Inc. Tissue retractor
US12349889B2 (en) * 2020-07-10 2025-07-08 Warsaw Orthopedic, Inc. Tissue retractor, retraction modules, and associated methods
US12096923B2 (en) * 2020-07-10 2024-09-24 Warsaw Orthopedic, Inc. Tissue retractor, retraction modules, and associated methods
US12383249B2 (en) * 2020-07-10 2025-08-12 Warsaw Orthopedic, Inc. Tissue retractor, retraction modules, and associated methods
US11833059B2 (en) 2020-11-05 2023-12-05 Warsaw Orthopedic, Inc. Expandable inter-body device, expandable plate system, and associated methods
US11285014B1 (en) 2020-11-05 2022-03-29 Warsaw Orthopedic, Inc. Expandable inter-body device, system, and method
US11638653B2 (en) 2020-11-05 2023-05-02 Warsaw Orthopedic, Inc. Surgery instruments with a movable handle
US12171439B2 (en) 2020-11-05 2024-12-24 Warsaw Orthopedic, Inc. Protected drill
US12121453B2 (en) 2020-11-05 2024-10-22 Warsaw Orthopedic, Inc. Dual wedge expandable implant with eyelets, system, and method of use
US11517443B2 (en) 2020-11-05 2022-12-06 Warsaw Orthopedic, Inc. Dual wedge expandable implant, system and method of use
US11376134B1 (en) 2020-11-05 2022-07-05 Warsaw Orthopedic, Inc. Dual expanding spinal implant, system, and method of use
US11963881B2 (en) 2020-11-05 2024-04-23 Warsaw Orthopedic, Inc. Expandable inter-body device, system, and method
US12318308B2 (en) 2020-11-05 2025-06-03 Warsaw Orthopedic, Inc. Dual expandable inter-body device
US12239544B2 (en) 2020-11-05 2025-03-04 Warsaw Orthopedic, Inc. Rhomboid shaped implants
US11291554B1 (en) 2021-05-03 2022-04-05 Medtronic, Inc. Unibody dual expanding interbody implant
US11564724B2 (en) 2020-11-05 2023-01-31 Warsaw Orthopedic, Inc. Expandable inter-body device, system and method
US11395743B1 (en) 2021-05-04 2022-07-26 Warsaw Orthopedic, Inc. Externally driven expandable interbody and related methods
WO2022251644A1 (en) 2021-05-28 2022-12-01 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
US12268614B2 (en) 2021-06-24 2025-04-08 Warsaw Orthopedic, Inc. Interbody implant with adjusting shims
WO2022271280A1 (en) 2021-06-24 2022-12-29 Warsaw Orthopedic, Inc. Expandable interbody implant and corresponding surgical tool
US12440349B2 (en) 2021-11-01 2025-10-14 Warsaw Orthopedic, Inc. Expandable interbody implant and breakoff screw
US12485019B2 (en) 2021-06-24 2025-12-02 Warsaw Orthopedic, Inc. Parallel jaw inserter
US12295865B2 (en) 2021-06-24 2025-05-13 Warsaw Orthopedic, Inc. Expandable interbody implant and corresponding inserter
US11612499B2 (en) 2021-06-24 2023-03-28 Warsaw Orthopedic, Inc. Expandable interbody implant
US11730608B2 (en) 2021-07-13 2023-08-22 Warsaw Orthopedic, Inc. Monoblock expandable interbody implant
US20240336896A1 (en) 2021-10-14 2024-10-10 Lonza Sales Ag Modified producer cells for extracellular vesicle production
US11850163B2 (en) 2022-02-01 2023-12-26 Warsaw Orthopedic, Inc. Interbody implant with adjusting shims
KR20250027283A (ko) 2022-05-19 2025-02-25 라이엘 이뮤노파마, 인크. nr4a3을 표적하는 폴리뉴클레오타이드 및 이의 용도
WO2024064952A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells overexpressing c-jun
WO2024077174A1 (en) 2022-10-05 2024-04-11 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
CN118853661A (zh) * 2023-04-28 2024-10-29 时夕(广州)生物科技有限公司 编辑rna的方法、组合物及其应用
US12533241B2 (en) 2023-05-16 2026-01-27 Warsaw Orthopedic, Inc. Distracting and angling expandable interbody device
WO2025217398A1 (en) 2024-04-10 2025-10-16 Lyell Immunopharma, Inc. Methods for culturing cells with improved culture medium

Family Cites Families (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3749088A (en) 1971-06-23 1973-07-31 W Kohlmann Surgical retractor device
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
WO1993007883A1 (en) 1991-10-24 1993-04-29 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
WO1998021366A1 (en) 1996-11-13 1998-05-22 Qbi Enterprises, Ltd. Gene identification method
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6139493A (en) * 1998-07-08 2000-10-31 Koros; Tibor B. Retractor with adjustable length blades and light pipe guides
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
GB2375172A (en) 2000-02-07 2002-11-06 Quark Biotech Inc Fas pathway genes
WO2002060330A1 (en) * 2001-01-29 2002-08-08 Stephen Ritland Retractor and method for spinal pedicle screw placement
WO2005060667A2 (en) 2003-12-19 2005-07-07 The General Hospital Corporation Nucleic acid and amino acid sequences involved in pain
WO2003016475A2 (en) 2001-08-14 2003-02-27 The General Hospital Corporation Nucleic acid and amino acid sequences involved in pain
AU2002351828A1 (en) 2001-11-05 2003-05-19 Deutsches Krebsforschungszentrum Novel genetic markers for leukemias
US20030157082A1 (en) 2002-01-31 2003-08-21 Millennium Pharmaceuticals, Inc. Methods and compositions for treating cancer using 140, 1470, 1686, 2089, 2427, 3702, 5891, 6428, 7181, 7660, 25641, 69583, 49863, 8897, 1682, 17667, 9235, 3703, 14171, 10359, 1660, 1450, 18894, 2088, 32427, 2160, 9252, 9389, 1642, 85269, 10297, 1584, 9525, 14124, 4469, 8990, 2100, 9288, 64698, 10480,20893, 33230,1586, 9943, 16334, 68862, 9011, 14031, 6178, 21225, 1420, 32236, 2099, 2150, 26583, 2784, 8941, 9811, 27444, 50566 or 66428 molecules
US20040101855A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of PPAR binding protein expression
US9259144B2 (en) 2002-07-11 2016-02-16 Nuvasive, Inc. Surgical access system and related methods
WO2004012817A2 (en) 2002-07-31 2004-02-12 Kylix B.V. Use of genes identified to be involved in tumor development for the development of anti-cancer drugs
JP2006513701A (ja) 2002-10-11 2006-04-27 アーラム バイオシステムズ インコーポレイテッド 核酸ベースのシグナルトランスデューサーを含む立体構造的に敏感なプローブを有する標的検出システム
US7850608B2 (en) 2002-10-25 2010-12-14 K2M, Inc. Minimal incision maximal access MIS spine instrumentation and method
US9045518B2 (en) * 2002-11-18 2015-06-02 Santaris Pharma A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
US7691057B2 (en) 2003-01-16 2010-04-06 Nuvasive, Inc. Surgical access system and related methods
US7256200B2 (en) 2003-02-10 2007-08-14 The Board Of Trustees Of The University Of Illinois, A Body Corporate And Politic Of The State Of Illinois Method and composition for potentiating an oplate analgesic
US7819801B2 (en) 2003-02-27 2010-10-26 Nuvasive, Inc. Surgical access system and related methods
US20050085436A1 (en) 2003-07-08 2005-04-21 Hao Li Method to treat conditions associated with insulin signalling dysregulation
US7481766B2 (en) 2003-08-14 2009-01-27 Synthes (U.S.A.) Multiple-blade retractor
US20050137461A1 (en) * 2003-12-18 2005-06-23 Depuy Spine, Inc. Telescoping blade assembly and instruments for adjusting an adjustable blade
US7374927B2 (en) * 2004-05-03 2008-05-20 Affymetrix, Inc. Methods of analysis of degraded nucleic acid samples
US9622732B2 (en) * 2004-10-08 2017-04-18 Nuvasive, Inc. Surgical access system and related methods
JP2006223228A (ja) 2005-02-18 2006-08-31 Dainippon Sumitomo Pharma Co Ltd 自己免疫疾患治療剤のスクリーニング方法
US20060287584A1 (en) * 2005-06-16 2006-12-21 Javier Garcia-Bengochia Surgical retractor extensions
CN1904900A (zh) * 2005-07-28 2007-01-31 中国科学院生物物理研究所 人类的内源性siRNA序列及其应用和筛选方法
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
WO2007059533A2 (en) * 2005-11-18 2007-05-24 Myogen, Inc. Uses for camki i and hdacs in the treatment of heart conditions
JP5342881B2 (ja) 2006-01-27 2013-11-13 アイシス ファーマシューティカルズ， インコーポレーテッド ６−修飾された二環式核酸類似体
US20090156540A1 (en) * 2006-03-07 2009-06-18 Mckenna Katherine Method for diagnosing and/or predicting the development of an allergic disorder and agents for treating and/or preventing same
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
WO2008020435A2 (en) 2006-08-15 2008-02-21 Quark Pharmaceuticals, Inc Compositions and methods for treatment of mood disorders
ES2534430T3 (es) 2006-08-28 2015-04-22 The University Of Western Australia Método de modulación de la expresión del receptor del factor de crecimiento epidérmico (EGFR) que implica miARN
GB0617222D0 (en) 2006-08-31 2006-10-11 Vereniging Het Nl Kanker I Antibiotics
US8062217B2 (en) * 2007-01-26 2011-11-22 Theken Spine, Llc Surgical retractor with removable blades and method of use
WO2008113832A2 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S SHORT RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF TARGET mRNA
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
WO2008152636A2 (en) * 2007-06-15 2008-12-18 Quark Pharmaceuticals, Inc. Compositions and methods for inhibiting nadph oxidase expression
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
DE202007012284U1 (de) 2007-09-01 2007-10-25 Aesculap Ag & Co. Kg Chirurgischer Retraktor
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
US8841423B2 (en) 2008-04-28 2014-09-23 University Of Iowa Research Foundation Monoclonal antibodies specific for oxidized calcium/calmodulin dependent protein kinase II
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
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
US8226554B2 (en) 2008-10-30 2012-07-24 Warsaw Orthopedic, Inc. Retractor assemblies for surgery in a patient
WO2010120969A1 (en) 2009-04-15 2010-10-21 Board Of Regents, The University Of Texas System Targeting of the mir-30 family and let-7 family as a treatment for heart disease
ES2555057T3 (es) * 2009-06-12 2015-12-28 Roche Innovation Center Copenhagen A/S Nuevos potentes compuestos antisentido anti-ApoB
US8563528B2 (en) * 2009-07-21 2013-10-22 Santaris Pharma A/S Antisense oligomers targeting PCSK9
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2490699A1 (en) 2009-10-20 2012-08-29 Santaris Pharma A/S Oral delivery of therapeutically effective lna oligonucleotides
US9737288B2 (en) * 2010-03-11 2017-08-22 Globus Medical, Inc Tissue retractor and methods of use
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2012024255A2 (en) 2010-08-16 2012-02-23 Duke University Camkk-beta as a target for treating cancer
EP3778896A1 (en) 2011-08-09 2021-02-17 Fred Hutchinson Cancer Research Center Methods and compositions for inhibiting the growth and/or proliferation of myc-driven tumor cells
EP3453761A1 (en) 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
WO2013033436A1 (en) * 2011-09-01 2013-03-07 University Of Iowa Research Foundation Oligonucleotide-based probes for detection of bacterial nucleases
CA2857374A1 (en) 2011-09-02 2013-03-07 The Trustees Of Columbia University In The City Of New York Camkii, ip3r, calcineurin, p38 and mk2/3 inhibitors to treat metabolic disturbances of obesity
CN104136451A (zh) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 具有构象限制的单体的核酸化合物的合成和用途
WO2013151999A1 (en) 2012-04-02 2013-10-10 President And Fellows Of Harvard College Cancer treatment and immune system regulation through fat10 pathway inhibition
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
WO2014077693A1 (en) 2012-11-16 2014-05-22 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Means and methods for reducing an effect of aging in a mammalian cell
AU2014225889B2 (en) 2013-03-06 2018-12-06 The Johns Hopkins University CaMKII inhibitors and uses thereof
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
US20140329755A1 (en) 2013-05-01 2014-11-06 Gilead Sciences, Inc. Combination therapy for the treatment of arrhythmias or heart failure
ES2770667T3 (es) 2013-06-27 2020-07-02 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados que se dirigen a PCSK9
WO2016037106A1 (en) 2014-09-05 2016-03-10 Allosteros Therapeutics, Inc CaMKII INHIBITORS AND USES THEREOF
PL3265563T3 (pl) 2015-02-02 2021-09-13 Meiragtx Uk Ii Limited Regulacja ekspresji genów poprzez modulację alternatywnego splicingu za pośrednictwem aptamerów
EP3286318A2 (en) 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
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
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3353306A4 (en) 2015-09-25 2019-06-05 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
CA3005090A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
AU2017292173B2 (en) * 2016-07-06 2022-01-13 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
SMT202400091T1 (it) 2016-08-03 2024-05-14 Meiragtx Uk Ii Ltd Screening ad alta produzione basato su cellule per 5 aptameri
WO2019049148A1 (en) 2017-09-10 2019-03-14 Carmel Haifa University Economic Corporation Ltd. METHOD OF TREATING DEPRESSION AND MAJOR DEPRESSIVE DISORDER
US10717981B2 (en) 2018-01-18 2020-07-21 Advanced ReGen Medical Technologies, LLC Therapeutic compositions and methods of making and using the same
KR20200140805A (ko) 2018-02-21 2020-12-16 브리스톨-마이어스 스큅 컴퍼니 Camk2d 안티센스 올리고뉴클레오티드 및 그의 용도
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery

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