CA2834128A1 - Peptide oligonucleotide conjugates - Google Patents
Peptide oligonucleotide conjugates Download PDFInfo
- Publication number
- CA2834128A1 CA2834128A1 CA2834128A CA2834128A CA2834128A1 CA 2834128 A1 CA2834128 A1 CA 2834128A1 CA 2834128 A CA2834128 A CA 2834128A CA 2834128 A CA2834128 A CA 2834128A CA 2834128 A1 CA2834128 A1 CA 2834128A1
- Authority
- CA
- Canada
- Prior art keywords
- conjugate
- occurrence
- alkyl
- independently
- carrier peptide
- 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
Links
- 108090000765 processed proteins & peptides Proteins 0.000 title claims abstract description 183
- 108091034117 Oligonucleotide Proteins 0.000 title abstract description 35
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims abstract description 17
- 201000010099 disease Diseases 0.000 claims abstract description 13
- -1 glycine amino acid Chemical class 0.000 claims description 145
- 229940024606 amino acid Drugs 0.000 claims description 133
- 235000001014 amino acid Nutrition 0.000 claims description 129
- 150000001413 amino acids Chemical class 0.000 claims description 112
- 125000000217 alkyl group Chemical group 0.000 claims description 94
- 229910052739 hydrogen Inorganic materials 0.000 claims description 88
- 239000001257 hydrogen Substances 0.000 claims description 87
- 150000007523 nucleic acids Chemical class 0.000 claims description 76
- 102000039446 nucleic acids Human genes 0.000 claims description 74
- 108020004707 nucleic acids Proteins 0.000 claims description 74
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 70
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims description 69
- 238000000034 method Methods 0.000 claims description 69
- 125000000623 heterocyclic group Chemical group 0.000 claims description 52
- 125000003118 aryl group Chemical group 0.000 claims description 43
- 125000003545 alkoxy group Chemical group 0.000 claims description 42
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 42
- 239000004471 Glycine Substances 0.000 claims description 41
- 230000008685 targeting Effects 0.000 claims description 41
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 40
- 210000004027 cell Anatomy 0.000 claims description 38
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 37
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 35
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- 210000004899 c-terminal region Anatomy 0.000 claims description 31
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims description 30
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- 125000004432 carbon atom Chemical group C* 0.000 claims description 18
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- MTCFGRXMJLQNBG-REOHCLBHSA-N (2S)-2-Amino-3-hydroxypropansäure Chemical compound OC[C@H](N)C(O)=O MTCFGRXMJLQNBG-REOHCLBHSA-N 0.000 claims description 4
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Landscapes
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Priority Applications (1)
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| CA3092114A CA3092114A1 (en) | 2011-05-05 | 2011-11-17 | Peptide oligonucleotide conjugates |
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| US13/101,942 | 2011-05-05 | ||
| US13/101,942 US20110269665A1 (en) | 2009-06-26 | 2011-05-05 | Compound and method for treating myotonic dystrophy |
| US13/107,528 | 2011-05-13 | ||
| US13/107,528 US9238042B2 (en) | 2010-05-13 | 2011-05-13 | Antisense modulation of interleukins 17 and 23 signaling |
| PCT/US2011/061282 WO2012150960A1 (en) | 2011-05-05 | 2011-11-17 | Peptide oligonucleotide conjugates |
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| CA3092114A Division CA3092114A1 (en) | 2011-05-05 | 2011-11-17 | Peptide oligonucleotide conjugates |
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| JP (6) | JP6478632B2 (hr) |
| KR (3) | KR102229650B1 (hr) |
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| CA (2) | CA3092114A1 (hr) |
| IL (2) | IL273838B (hr) |
| WO (1) | WO2012150960A1 (hr) |
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| US11236329B2 (en) | 2007-06-29 | 2022-02-01 | Sarepta Therapeutics, Inc. | Compound and method for treating myotonic dystrophy |
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| PT1766010E (pt) | 2004-06-28 | 2011-05-25 | Univ Western Australia | Oligonucle?tidos antisense para a indu??o de skipping de ex?es e seus m?todos de utiliza??o |
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| CA2660523C (en) | 2006-08-11 | 2019-03-19 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| CN105641700B (zh) | 2007-10-26 | 2021-01-01 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| BRPI0920276B1 (pt) | 2008-10-24 | 2021-06-08 | Sarepta Therapeutics, Inc | oligonucleotídeo antissenso, composição que compreende o mesmo e uso do dito oligonucleotídeo para tratar distrofia muscular |
| RS58079B1 (sr) | 2009-11-12 | 2019-02-28 | Univ Western Australia | Antisens molekuli i postupci za tretman patologija |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| CN111808135A (zh) * | 2011-11-18 | 2020-10-23 | 萨勒普塔医疗公司 | 功能改性的寡核苷酸及其亚单元 |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| EP2785729B1 (en) | 2011-11-30 | 2020-11-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| RS57467B1 (sr) | 2011-12-08 | 2018-09-28 | Sarepta Therapeutics Inc | Oligonukleotidni analozi koji ciljaju humani lmna |
| CN104203289B (zh) | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
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| KR20210032545A (ko) | 2021-03-24 |
| CN103619356A (zh) | 2014-03-05 |
| JP6478632B2 (ja) | 2019-03-06 |
| IL229227A0 (en) | 2014-01-30 |
| IL229227B (en) | 2020-07-30 |
| KR102229650B1 (ko) | 2021-03-19 |
| JP2014515762A (ja) | 2014-07-03 |
| CN107693797A (zh) | 2018-02-16 |
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