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
Links
- 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 (enExample) | 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 (enExample) |
| EP (1) | EP2785840B8 (enExample) |
| JP (2) | JP6496549B2 (enExample) |
| AU (1) | AU2012345638C1 (enExample) |
| CA (1) | CA2857664A1 (enExample) |
| ES (1) | ES2727481T3 (enExample) |
| WO (1) | WO2013082551A1 (enExample) |
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 |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| 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 |
| CN105793422B (zh) * | 2013-09-05 | 2020-03-03 | 萨罗塔治疗公司(美国) | 酸性α-葡糖苷酶中反义诱导的外显子2纳入 |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| AU2016317667A1 (en) | 2015-08-28 | 2018-03-22 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| US10357543B2 (en) | 2015-11-16 | 2019-07-23 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein |
| EP3445405A4 (en) | 2016-04-18 | 2019-12-18 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERS AND METHOD FOR USE THEREOF TO TREAT DISEASES RELATING TO THE ACID ALPHA GLUCOSIDASE GENE |
| AU2017270598B2 (en) * | 2016-05-24 | 2022-12-01 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| IL263044B2 (en) * | 2016-05-24 | 2024-06-01 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| EP3464305B1 (en) * | 2016-05-24 | 2024-08-21 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| BR112018074299A2 (pt) | 2016-05-24 | 2019-03-12 | Sarepta Therapeutics, Inc. | composições farmacêuticas compreendendo eteplirsen |
| HRP20240583T1 (hr) | 2016-05-24 | 2024-07-19 | Sarepta Therapeutics, Inc. | Postupci za pripremu fosforodiamidat morfolino oligomera |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| WO2017218884A1 (en) | 2016-06-16 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of smn expression |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| EP3515445A4 (en) * | 2016-09-20 | 2020-05-27 | The Regents of The University of Colorado, A Body Corporate | SYNTHESIS OF BACKBONE-MODIFIED MORPHOLINO-OLIGONUCLEOTIDES AND CHIMERES WITH THE AID OF PHOSPHORAMIDITE CHEMISTRY |
| MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| WO2018118627A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| KR20240006057A (ko) | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| JP7596148B2 (ja) * | 2018-03-16 | 2024-12-09 | サレプタ セラピューティクス， インコーポレイテッド | アンチセンス送達用キメラペプチド |
| CN112166118B (zh) * | 2018-03-22 | 2025-07-11 | 德克萨斯大学体系董事会 | 治疗自身免疫性疾病和癌症的可溶性白介素-7受体(sIL7R)调节疗法 |
| EP3806868A4 (en) | 2018-06-13 | 2022-06-22 | 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 |
| IL318474A (en) | 2018-12-13 | 2025-03-01 | Sarepta Therapeutics Inc | Axon-skipping oligomer complexes for muscular dystrophy |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| AR121446A1 (es) * | 2020-02-28 | 2022-06-08 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| US12378267B2 (en) | 2021-06-17 | 2025-08-05 | Entrada Therapeutics, Inc. | Synthesis of FMOC-protected morpholino monomers and oligomers |
| WO2023205451A1 (en) | 2022-04-22 | 2023-10-26 | Entrada Therapeutics, Inc. | Cyclic peptides for delivering therapeutics |
| 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 |
|---|---|---|---|---|
| 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 |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| DE3687030T2 (de) | 1985-03-15 | 1993-03-11 | Eugene Stirchak | Stereoregulare polynukleotiden bindende polymere. |
| 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 |
| EP1704878B1 (en) | 1995-12-18 | 2013-04-10 | AngioDevice International GmbH | Crosslinked polymer compositions and methods for their use |
| 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 |
| JP2003533986A (ja) | 2000-05-04 | 2003-11-18 | エイブイアイ バイオファーマ， インコーポレイテッド | スプライス領域アンチセンス組成物および方法 |
| 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 |
| DK2548560T3 (en) | 2005-06-23 | 2015-07-13 | Cold Spring Harbor Lab | Compositions and Methods for Modulating SMN2 Splicing |
| US8067571B2 (en) * | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| ES2645410T3 (es) * | 2006-05-10 | 2017-12-05 | Sarepta Therapeutics, Inc. | Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos |
| WO2008018795A1 (en) | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| WO2009064471A1 (en) | 2007-11-15 | 2009-05-22 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| SI3449926T1 (sl) | 2009-06-17 | 2020-04-30 | Biogen Ma Inc. | Sestave in metode za modulacijo združevanja SMN2 pri subjektu |
| 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 |
| EP2576574A2 (en) * | 2010-05-28 | 2013-04-10 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| KR102183273B1 (ko) | 2011-05-05 | 2020-11-27 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
-
2012
- 2012-11-30 ES ES12812432T patent/ES2727481T3/es active Active
- 2012-11-30 US US14/360,895 patent/US9944926B2/en active Active
- 2012-11-30 WO PCT/US2012/067475 patent/WO2013082551A1/en not_active Ceased
- 2012-11-30 EP EP12812432.8A patent/EP2785840B8/en not_active Not-in-force
- 2012-11-30 JP JP2014544962A patent/JP6496549B2/ja not_active Expired - Fee Related
- 2012-11-30 AU AU2012345638A patent/AU2012345638C1/en not_active Ceased
- 2012-11-30 CA CA2857664A patent/CA2857664A1/en not_active Abandoned
-
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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