ES2935606T3 - Análogos de oligonucleótidos dirigidos a LMNA humana - Google Patents

Análogos de oligonucleótidos dirigidos a LMNA humana Download PDF

Info

Publication number: ES2935606T3
Authority: ES; Spain
Prior art keywords: alkyl; sequence; oligomer; bonds; antisense
Prior art date: 2011-12-08
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.): Active

Application number

ES18159070T

Other languages

English (en)

Spanish (es)

Inventor

Ryszard Kole

Richard Keith Bestwick

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.)

Sarepta Therapeutics Inc

Original Assignee

Sarepta 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.)

2011-12-08

Filing date

2012-12-07

Publication date

2023-03-08

2012-12-07 Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc

2023-03-08 Application granted granted Critical

2023-03-08 Publication of ES2935606T3 publication Critical patent/ES2935606T3/es

Status Active legal-status Critical Current

2032-12-07 Anticipated expiration legal-status Critical

Links

101001003584 Homo sapiens Prelamin-A/C Proteins 0.000 title claims abstract description 13
108091034117 Oligonucleotide Proteins 0.000 title claims description 83
230000008685 targeting Effects 0.000 title claims description 37
102000057754 human LMNA Human genes 0.000 title claims description 11
239000000074 antisense oligonucleotide Substances 0.000 claims abstract description 39
238000012230 antisense oligonucleotides Methods 0.000 claims abstract description 39
108020004999 messenger RNA Proteins 0.000 claims abstract description 32
230000001594 aberrant effect Effects 0.000 claims description 8
WROTXLSEMHAZEA-UHFFFAOYSA-N 4-diaminophosphoryloxymorpholine Chemical compound NP(N)(=O)ON1CCOCC1 WROTXLSEMHAZEA-UHFFFAOYSA-N 0.000 claims description 4
108020000948 Antisense Oligonucleotides Proteins 0.000 abstract description 22
102000001708 Protein Isoforms Human genes 0.000 abstract description 3
108010029485 Protein Isoforms Proteins 0.000 abstract description 3
238000013519 translation Methods 0.000 abstract description 2
102100026531 Prelamin-A/C Human genes 0.000 abstract 2
125000000217 alkyl group Chemical group 0.000 description 118
-1 piperazino group Chemical group 0.000 description 108
229910052739 hydrogen Inorganic materials 0.000 description 98
239000001257 hydrogen Substances 0.000 description 95
125000003545 alkoxy group Chemical group 0.000 description 52
125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 52
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- A61K47/62—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 a protein, peptide or polyamino acid
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- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing

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Proteomics, Peptides & Aminoacids (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
General Chemical & Material Sciences (AREA)
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Heart & Thoracic Surgery (AREA)
Cardiology (AREA)
Vascular Medicine (AREA)
Urology & Nephrology (AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Medicinal Preparation (AREA)
Peptides Or Proteins (AREA)
Micro-Organisms Or Cultivation Processes Thereof (AREA)

ES18159070T 2011-12-08 2012-12-07 Análogos de oligonucleótidos dirigidos a LMNA humana Active ES2935606T3 (es)

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EP (3)	EP2788487B1 (2)
JP (7)	JP6132848B2 (2)
CY (1)	CY1120495T1 (2)
DK (1)	DK2788487T3 (2)
ES (2)	ES2935606T3 (2)
HR (1)	HRP20181022T1 (2)
HU (1)	HUE038369T2 (2)
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SI (1)	SI2788487T1 (2)
SM (1)	SMT201800337T1 (2)
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US10017763B2 (en)	2010-09-03	2018-07-10	Sarepta Therapeutics, Inc.	dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
US10323236B2 (en)	2011-07-22	2019-06-18	President And Fellows Of Harvard College	Evaluation and improvement of nuclease cleavage specificity
HK1201514A1 (en)	2011-11-18	2015-09-04	Sarepta Therapeutics, Inc.	Functionally-modified oligonucleotides and subunits thereof
ES2935606T3 (es)	2011-12-08	2023-03-08	Sarepta Therapeutics Inc	Análogos de oligonucleótidos dirigidos a LMNA humana
US9163284B2 (en)	2013-08-09	2015-10-20	President And Fellows Of Harvard College	Methods for identifying a target site of a Cas9 nuclease
US11236338B2 (en) *	2013-09-05	2022-02-01	Sarepta Therapeutics, Inc.	Antisense-induced exon2 inclusion in acid alpha-glucosidase
US9322037B2 (en)	2013-09-06	2016-04-26	President And Fellows Of Harvard College	Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en)	2013-09-06	2016-01-05	President And Fellows Of Harvard College	Switchable gRNAs comprising aptamers
US20150165054A1 (en)	2013-12-12	2015-06-18	President And Fellows Of Harvard College	Methods for correcting caspase-9 point mutations
EA030615B1 (ru)	2013-12-24	2018-08-31	Сентисс Фарма Прайвет Лимитед	Офтальмологический раствор бримонидина для местного применения
US10077453B2 (en)	2014-07-30	2018-09-18	President And Fellows Of Harvard College	CAS9 proteins including ligand-dependent inteins
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EP3399038A3 (en)	2019-02-13
US11071749B2 (en)	2021-07-27
JP6581132B2 (ja)	2019-09-25
HUE038369T2 (hu)	2018-10-29
US20200147119A1 (en)	2020-05-14
JP2017099416A (ja)	2017-06-08
SMT201800337T1 (it)	2018-09-13
JP2019129848A (ja)	2019-08-08
JP6132849B2 (ja)	2017-05-31
JP7277503B2 (ja)	2023-05-19
PT2788487T (pt)	2018-07-03
EP3399038B1 (en)	2022-11-16
US20170051278A1 (en)	2017-02-23
SI2788487T1 (sl)	2018-09-28
EP2788488A2 (en)	2014-10-15
US9682097B2 (en)	2017-06-20
EP2788488B1 (en)	2018-03-21
HRP20181022T1 (hr)	2018-08-24
JP6132848B2 (ja)	2017-05-31
US9833468B2 (en)	2017-12-05
EP3399038A2 (en)	2018-11-07
US9326992B2 (en)	2016-05-03
JP2015501817A (ja)	2015-01-19
EP2788487A2 (en)	2014-10-15
US20140024698A1 (en)	2014-01-23
LT2788487T (lt)	2018-09-10
RS57467B1 (sr)	2018-09-28
US20140024821A1 (en)	2014-01-23
US20160102307A1 (en)	2016-04-14
US10398721B2 (en)	2019-09-03
WO2013086441A2 (en)	2013-06-13
JP2019172691A (ja)	2019-10-10
JP2021104052A (ja)	2021-07-26
WO2013086444A2 (en)	2013-06-13
JP6596032B2 (ja)	2019-10-23
JP2015505839A (ja)	2015-02-26
PL2788487T3 (pl)	2018-10-31
EP2788487B1 (en)	2018-04-04
US20180092938A1 (en)	2018-04-05
CY1120495T1 (el)	2019-07-10
DK2788487T3 (en)	2018-07-23
US20180271893A1 (en)	2018-09-27
WO2013086444A3 (en)	2013-08-01
ES2674929T3 (es)	2018-07-05
US9066967B2 (en)	2015-06-30
US10076536B2 (en)	2018-09-18
JP2017101080A (ja)	2017-06-08
WO2013086441A3 (en)	2013-08-01
TR201809173T4 (tr)	2018-07-23

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