JP2015504650A5 - - Google Patents
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- Publication number
- JP2015504650A5 JP2015504650A5 JP2014544962A JP2014544962A JP2015504650A5 JP 2015504650 A5 JP2015504650 A5 JP 2015504650A5 JP 2014544962 A JP2014544962 A JP 2014544962A JP 2014544962 A JP2014544962 A JP 2014544962A JP 2015504650 A5 JP2015504650 A5 JP 2015504650A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- antisense oligonucleotide
- exon
- group
- alkyl
- 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.)
- Granted
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- 108091034117 Oligonucleotide Proteins 0.000 claims description 33
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 31
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 31
- 239000002773 nucleotide Substances 0.000 claims description 18
- 125000003729 nucleotide group Chemical group 0.000 claims description 18
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims description 12
- 102100021947 Survival motor neuron protein Human genes 0.000 claims description 12
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 12
- 108020004999 messenger RNA Proteins 0.000 claims description 12
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 12
- 239000001257 hydrogen Substances 0.000 claims description 10
- 229910052739 hydrogen Inorganic materials 0.000 claims description 10
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 10
- 101150015954 SMN2 gene Proteins 0.000 claims description 9
- 125000000217 alkyl group Chemical group 0.000 claims description 8
- 150000002431 hydrogen Chemical class 0.000 claims description 8
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 6
- 208000002320 spinal muscular atrophy Diseases 0.000 claims description 6
- 229940113082 thymine Drugs 0.000 claims description 6
- 229940035893 uracil Drugs 0.000 claims description 6
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical group N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims description 4
- 125000002252 acyl group Chemical group 0.000 claims description 4
- 230000000295 complement effect Effects 0.000 claims description 4
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 4
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 4
- 229930024421 Adenine Natural products 0.000 claims description 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 2
- 239000004475 Arginine Substances 0.000 claims description 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 2
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims description 2
- 229960000643 adenine Drugs 0.000 claims description 2
- 150000001413 amino acids Chemical class 0.000 claims description 2
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 2
- 125000003118 aryl group Chemical group 0.000 claims description 2
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical group [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 2
- 230000004700 cellular uptake Effects 0.000 claims description 2
- 229940104302 cytosine Drugs 0.000 claims description 2
- 230000001747 exhibiting effect Effects 0.000 claims description 2
- 229910052736 halogen Inorganic materials 0.000 claims description 2
- 150000002367 halogens Chemical group 0.000 claims description 2
- 125000000623 heterocyclic group Chemical group 0.000 claims description 2
- 239000012528 membrane Substances 0.000 claims description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 2
- 229910052757 nitrogen Inorganic materials 0.000 claims description 2
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims description 2
- 125000004193 piperazinyl group Chemical group 0.000 claims description 2
- 150000003839 salts Chemical class 0.000 claims description 2
- 125000001424 substituent group Chemical group 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 20
- 230000037430 deletion Effects 0.000 claims 2
- 238000012217 deletion Methods 0.000 claims 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 1
- -1 C 1 -C 6 alkyl Substances 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 230000003834 intracellular effect Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 21
- 230000002708 enhancing effect Effects 0.000 description 3
- 239000002777 nucleoside Substances 0.000 description 2
- 150000003833 nucleoside derivatives Chemical class 0.000 description 2
- 230000002950 deficient Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161565499P | 2011-11-30 | 2011-11-30 | |
| US61/565,499 | 2011-11-30 | ||
| PCT/US2012/067475 WO2013082551A1 (en) | 2011-11-30 | 2012-11-30 | Induced exon inclusion in spinal muscle atrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018232429A Division JP2019059769A (ja) | 2011-11-30 | 2018-12-12 | 脊髄性筋萎縮症における誘発されたエクソン包含 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015504650A JP2015504650A (ja) | 2015-02-16 |
| JP2015504650A5 true JP2015504650A5 (cg-RX-API-DMAC7.html) | 2016-01-21 |
| JP6496549B2 JP6496549B2 (ja) | 2019-04-03 |
Family
ID=47520246
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014544962A Expired - Fee Related JP6496549B2 (ja) | 2011-11-30 | 2012-11-30 | 脊髄性筋萎縮症における誘発されたエクソン包含 |
| JP2018232429A Pending JP2019059769A (ja) | 2011-11-30 | 2018-12-12 | 脊髄性筋萎縮症における誘発されたエクソン包含 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018232429A Pending JP2019059769A (ja) | 2011-11-30 | 2018-12-12 | 脊髄性筋萎縮症における誘発されたエクソン包含 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US9944926B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2785840B8 (cg-RX-API-DMAC7.html) |
| JP (2) | JP6496549B2 (cg-RX-API-DMAC7.html) |
| AU (1) | AU2012345638C1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2857664A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2727481T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2013082551A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| US9856474B2 (en) | 2013-01-16 | 2018-01-02 | Iowa State University Research Foundation, Inc. | Deep intronic target for splicing correction on spinal muscular atrophy gene |
| AU2014317961B2 (en) * | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| JP6987041B2 (ja) * | 2015-08-28 | 2021-12-22 | サレプタ セラピューティクス，インコーポレイテッド | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
| EP3377073A4 (en) * | 2015-11-16 | 2019-06-26 | Ohio State Innovation Foundation | METHOD AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES WITH THE SURVIVAL MOTOR NEURON (SMN) PROTEIN |
| 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 |
| SG11201809494VA (en) | 2016-05-24 | 2018-12-28 | Sarepta Therapeutics Inc | Pharmaceutical composition comprising eteplirsen |
| MA45362A (fr) * | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| HRP20241428T1 (hr) * | 2016-05-24 | 2025-01-03 | Sarepta Therapeutics, Inc. | Postupci za pripremu oligomera |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| SG10202101836TA (en) * | 2016-05-24 | 2021-03-30 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| AU2017270598B2 (en) * | 2016-05-24 | 2022-12-01 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| BR112018074270B1 (pt) | 2016-05-24 | 2021-02-02 | Sarepta Therapeutics, Inc | processo para preparar compostos oligoméricos bem como os ditos compostos |
| EP3471779A4 (en) | 2016-06-16 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | COMBINATIONS FOR MODULATING SMN EXPRESSION |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| CN116804031A (zh) * | 2016-09-20 | 2023-09-26 | 科罗拉多州立大学董事会法人团体 | 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体 |
| JP7160807B2 (ja) | 2016-12-19 | 2022-10-25 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| FI4122497T3 (fi) | 2016-12-19 | 2024-06-04 | Sarepta Therapeutics Inc | Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan |
| LT3554553T (lt) | 2016-12-19 | 2022-08-25 | Sarepta Therapeutics, Inc. | Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos |
| US12370261B2 (en) | 2018-03-16 | 2025-07-29 | Sarepta Therapeutics, Inc. | Chimeric peptides for antisense delivery |
| JP7576835B2 (ja) * | 2018-03-22 | 2024-11-01 | ボード・オブ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・テキサス・システム | 自己免疫疾患および癌を治療するための可溶性インターロイキン７受容体（ｓＩＬ７Ｒ）調節療法 |
| EP4219717A3 (en) | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| TW202449155A (zh) | 2018-07-27 | 2024-12-16 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| WO2020123574A1 (en) | 2018-12-13 | 2020-06-18 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| BR112022016238A2 (pt) * | 2020-02-28 | 2022-10-11 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular smn2 |
| US12378267B2 (en) | 2021-06-17 | 2025-08-05 | Entrada Therapeutics, Inc. | Synthesis of FMOC-protected morpholino monomers and oligomers |
| CA3249036A1 (en) | 2022-04-22 | 2023-10-26 | Entrada Therapeutics, Inc. | CYCLIC PEPTIDES FOR ADMINISTERING THERAPEUTIC AGENTS |
| WO2024138018A1 (en) * | 2022-12-23 | 2024-06-27 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Treatment of gemin5-mediated disorders |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| ATE124999T1 (de) | 1985-03-15 | 1995-07-15 | Antivirals Inc | Immunotestmittel für polynukleotid und verfahren. |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| JPH07501204A (ja) | 1991-06-28 | 1995-02-09 | マサチューセッツ インスティテュート オブ テクノロジー | 局所的オリゴヌクレオチド療法 |
| PT876165E (pt) | 1995-12-18 | 2006-10-31 | Angiotech Biomaterials Corp | Composicoes de polimeros reticulados e processos para a sua utilizacao |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
| UA67760C2 (uk) | 1997-12-11 | 2004-07-15 | Міннесота Майнінг Енд Мануфакчурінг Компані | Імідазонафтиридин та тетрагідроімідазонафтиридин, фармацевтична композиція, спосіб індукування біосинтезу цитокінів та спосіб лікування вірусної інфекції, проміжні сполуки |
| FR2802206B1 (fr) | 1999-12-14 | 2005-04-22 | Sod Conseils Rech Applic | Derives de 4-aminopiperidine et leur utilisation en tant que medicament |
| WO2001083740A2 (en) * | 2000-05-04 | 2001-11-08 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
| US6965025B2 (en) | 2001-12-10 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of connective tissue growth factor expression |
| US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| EP3470072A1 (en) | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| CA2651881A1 (en) * | 2006-05-10 | 2008-03-27 | Avi Biopharma, Inc. | Oligonucleotide analogs having cationic intersubunit linkages |
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| KR101617472B1 (ko) | 2007-11-15 | 2016-05-02 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 모르폴리노 올리고머의 합성 방법 |
| WO2010120820A1 (en) | 2009-04-13 | 2010-10-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| JP5707396B2 (ja) | 2009-06-17 | 2015-04-30 | 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 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| JP5585822B2 (ja) * | 2010-05-11 | 2014-09-10 | 東レ・ファインケミカル株式会社 | 光学活性ニペコチン酸誘導体の製造方法 |
| KR102182663B1 (ko) | 2010-05-28 | 2020-11-25 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| CA2834128A1 (en) | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
-
2012
- 2012-11-30 US US14/360,895 patent/US9944926B2/en active Active
- 2012-11-30 JP JP2014544962A patent/JP6496549B2/ja not_active Expired - Fee Related
- 2012-11-30 ES ES12812432T patent/ES2727481T3/es active Active
- 2012-11-30 WO PCT/US2012/067475 patent/WO2013082551A1/en not_active Ceased
- 2012-11-30 AU AU2012345638A patent/AU2012345638C1/en not_active Ceased
- 2012-11-30 CA CA2857664A patent/CA2857664A1/en not_active Abandoned
- 2012-11-30 EP EP12812432.8A patent/EP2785840B8/en not_active Not-in-force
-
2018
- 2018-03-14 US US15/921,518 patent/US10577605B2/en active Active
- 2018-12-12 JP JP2018232429A patent/JP2019059769A/ja active Pending
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