JPWO2020198697A5 - - Google Patents
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- JPWO2020198697A5 JPWO2020198697A5 JP2021557397A JP2021557397A JPWO2020198697A5 JP WO2020198697 A5 JPWO2020198697 A5 JP WO2020198697A5 JP 2021557397 A JP2021557397 A JP 2021557397A JP 2021557397 A JP2021557397 A JP 2021557397A JP WO2020198697 A5 JPWO2020198697 A5 JP WO2020198697A5
- Authority
- JP
- Japan
- Prior art keywords
- lipid
- composition
- mol
- seq
- lnp
- 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.)
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- 150000002632 lipids Chemical class 0.000 claims description 183
- 239000000203 mixture Substances 0.000 claims description 137
- 108700026244 Open Reading Frames Proteins 0.000 claims description 45
- 108020005004 Guide RNA Proteins 0.000 claims description 39
- 125000003729 nucleotide group Chemical group 0.000 claims description 39
- 229930024421 Adenine Natural products 0.000 claims description 36
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 36
- 229960000643 adenine Drugs 0.000 claims description 36
- -1 PEG lipid) Chemical class 0.000 claims description 35
- 230000007935 neutral effect Effects 0.000 claims description 32
- 239000002773 nucleotide Substances 0.000 claims description 29
- 108020004705 Codon Proteins 0.000 claims description 19
- GZQKNULLWNGMCW-PWQABINMSA-N lipid A (E. coli) Chemical compound O1[C@H](CO)[C@@H](OP(O)(O)=O)[C@H](OC(=O)C[C@@H](CCCCCCCCCCC)OC(=O)CCCCCCCCCCCCC)[C@@H](NC(=O)C[C@@H](CCCCCCCCCCC)OC(=O)CCCCCCCCCCC)[C@@H]1OC[C@@H]1[C@@H](O)[C@H](OC(=O)C[C@H](O)CCCCCCCCCCC)[C@@H](NC(=O)C[C@H](O)CCCCCCCCCCC)[C@@H](OP(O)(O)=O)O1 GZQKNULLWNGMCW-PWQABINMSA-N 0.000 claims description 17
- 239000011230 binding agent Substances 0.000 claims description 16
- 101000772194 Homo sapiens Transthyretin Proteins 0.000 claims description 15
- 102100029290 Transthyretin Human genes 0.000 claims description 15
- 230000004568 DNA-binding Effects 0.000 claims description 14
- 101150091380 TTR gene Proteins 0.000 claims description 14
- 108020004707 nucleic acids Proteins 0.000 claims description 13
- 102000039446 nucleic acids Human genes 0.000 claims description 13
- 150000007523 nucleic acids Chemical class 0.000 claims description 13
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 12
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 12
- 239000013598 vector Substances 0.000 claims description 12
- 238000012986 modification Methods 0.000 claims description 10
- 230000004048 modification Effects 0.000 claims description 10
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 9
- 210000002966 serum Anatomy 0.000 claims description 9
- 230000005782 double-strand break Effects 0.000 claims description 8
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims description 6
- 235000012000 cholesterol Nutrition 0.000 claims description 6
- 102000001049 Amyloid Human genes 0.000 claims description 5
- 108010094108 Amyloid Proteins 0.000 claims description 5
- 238000009825 accumulation Methods 0.000 claims description 5
- 206010002022 amyloidosis Diseases 0.000 claims description 5
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 4
- 239000002105 nanoparticle Substances 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 230000001939 inductive effect Effects 0.000 claims description 3
- RKSLVDIXBGWPIS-UAKXSSHOSA-N 1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-iodopyrimidine-2,4-dione Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(I)=C1 RKSLVDIXBGWPIS-UAKXSSHOSA-N 0.000 claims description 2
- UVBYMVOUBXYSFV-XUTVFYLZSA-N 1-methylpseudouridine Chemical compound O=C1NC(=O)N(C)C=C1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 UVBYMVOUBXYSFV-XUTVFYLZSA-N 0.000 claims description 2
- ZXIATBNUWJBBGT-JXOAFFINSA-N 5-methoxyuridine Chemical compound O=C1NC(=O)C(OC)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 ZXIATBNUWJBBGT-JXOAFFINSA-N 0.000 claims description 2
- 208000037259 Amyloid Plaque Diseases 0.000 claims description 2
- 108091033409 CRISPR Proteins 0.000 claims description 2
- 229930185560 Pseudouridine Natural products 0.000 claims description 2
- PTJWIQPHWPFNBW-UHFFFAOYSA-N Pseudouridine C Natural products OC1C(O)C(CO)OC1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-UHFFFAOYSA-N 0.000 claims description 2
- WGDUUQDYDIIBKT-UHFFFAOYSA-N beta-Pseudouridine Natural products OC1OC(CN2C=CC(=O)NC2=O)C(O)C1O WGDUUQDYDIIBKT-UHFFFAOYSA-N 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 238000009472 formulation Methods 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 150000003904 phospholipids Chemical class 0.000 claims description 2
- PTJWIQPHWPFNBW-GBNDHIKLSA-N pseudouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-GBNDHIKLSA-N 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 108020004414 DNA Proteins 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 56
- 208000024891 symptom Diseases 0.000 description 4
- 210000004027 cell Anatomy 0.000 description 3
- 230000035772 mutation Effects 0.000 description 3
- 206010060860 Neurological symptom Diseases 0.000 description 2
- 108020004485 Nonsense Codon Proteins 0.000 description 2
- 230000003941 amyloidogenesis Effects 0.000 description 2
- 230000000747 cardiac effect Effects 0.000 description 2
- 230000037430 deletion Effects 0.000 description 2
- 238000012217 deletion Methods 0.000 description 2
- 230000037433 frameshift Effects 0.000 description 2
- 238000003780 insertion Methods 0.000 description 2
- 230000037431 insertion Effects 0.000 description 2
- 230000006780 non-homologous end joining Effects 0.000 description 2
- 230000037434 nonsense mutation Effects 0.000 description 2
- 102220470698 BUD13 homolog_V30P_mutation Human genes 0.000 description 1
- 238000010453 CRISPR/Cas method Methods 0.000 description 1
- 206010007509 Cardiac amyloidosis Diseases 0.000 description 1
- 206010007559 Cardiac failure congestive Diseases 0.000 description 1
- 208000034846 Familial Amyloid Neuropathies Diseases 0.000 description 1
- 206010019280 Heart failures Diseases 0.000 description 1
- 206010019889 Hereditary neuropathic amyloidosis Diseases 0.000 description 1
- 102220558130 Olfactory receptor 2A1/2A42_V30L_mutation Human genes 0.000 description 1
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical class O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 description 1
- 210000001072 colon Anatomy 0.000 description 1
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 210000005229 liver cell Anatomy 0.000 description 1
- 201000001119 neuropathy Diseases 0.000 description 1
- 230000007823 neuropathy Effects 0.000 description 1
- 208000033808 peripheral neuropathy Diseases 0.000 description 1
- 102200150628 rs151220873 Human genes 0.000 description 1
- 102200017272 rs28931576 Human genes 0.000 description 1
- 102220128569 rs777178486 Human genes 0.000 description 1
- 210000003497 sciatic nerve Anatomy 0.000 description 1
- 210000003594 spinal ganglia Anatomy 0.000 description 1
- 210000002784 stomach Anatomy 0.000 description 1
- 201000007905 transthyretin amyloidosis Diseases 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025016673A JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962825637P | 2019-03-28 | 2019-03-28 | |
| US62/825,637 | 2019-03-28 | ||
| PCT/US2020/025513 WO2020198697A1 (en) | 2019-03-28 | 2020-03-27 | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025016673A Division JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022525428A JP2022525428A (ja) | 2022-05-13 |
| JPWO2020198697A5 true JPWO2020198697A5 (https=) | 2023-12-15 |
| JP2022525428A5 JP2022525428A5 (https=) | 2023-12-15 |
| JP7631215B2 JP7631215B2 (ja) | 2025-02-18 |
Family
ID=70296147
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021557397A Active JP7631215B2 (ja) | 2019-03-28 | 2020-03-27 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
| JP2025016673A Pending JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025016673A Pending JP2025072483A (ja) | 2019-03-28 | 2025-02-04 | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20230044994A1 (https=) |
| EP (1) | EP3946598A1 (https=) |
| JP (2) | JP7631215B2 (https=) |
| KR (1) | KR20220004648A (https=) |
| CN (1) | CN113874076A (https=) |
| AU (1) | AU2020244887A1 (https=) |
| BR (1) | BR112021019196A2 (https=) |
| CA (1) | CA3134271A1 (https=) |
| CO (1) | CO2021014559A2 (https=) |
| EA (1) | EA202192636A1 (https=) |
| IL (1) | IL286524A (https=) |
| MX (1) | MX2021011565A (https=) |
| PH (1) | PH12021552301A1 (https=) |
| SG (1) | SG11202110434PA (https=) |
| WO (1) | WO2020198697A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN104114572A (zh) * | 2011-12-16 | 2014-10-22 | 现代治疗公司 | 经修饰的核苷、核苷酸和核酸组合物 |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| JP7543259B2 (ja) | 2018-10-18 | 2024-09-02 | アクイタス セラピューティクス インコーポレイテッド | 活性剤の脂質ナノ粒子送達のための脂質 |
| IL303195A (en) | 2020-11-25 | 2023-07-01 | Akagera Medicines Inc | Lipid nanoparticles for delivery of nucleic acids and related methods of use |
| WO2022246266A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| US20240261435A1 (en) * | 2021-06-04 | 2024-08-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof |
| KR20240038705A (ko) * | 2021-06-22 | 2024-03-25 | 인텔리아 테라퓨틱스, 인크. | 간 유전자의 생체 내 편집 방법 |
| WO2023185697A2 (en) | 2022-03-29 | 2023-10-05 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of transthyretin amyloidosis |
| EP4531819A2 (en) | 2022-05-25 | 2025-04-09 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids and methods of use thereof |
| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| EP4590820A2 (en) | 2022-09-22 | 2025-07-30 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease |
| EP4627083A1 (en) * | 2022-11-30 | 2025-10-08 | Arbor Biotechnologies, Inc. | Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2026003582A2 (en) * | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
| WO2026061971A1 (en) | 2024-09-17 | 2026-03-26 | Lonza Ltd | Nucleic acid untranslated regions |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| KR940703846A (ko) | 1991-12-24 | 1994-12-12 | 비. 린네 파샬 | 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides) |
| US6169169B1 (en) | 1994-05-19 | 2001-01-02 | Dako A/S | PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis |
| US20050244869A1 (en) * | 2004-04-05 | 2005-11-03 | Brown-Driver Vickie L | Modulation of transthyretin expression |
| WO2006007712A1 (en) | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| DK2931898T3 (en) * | 2012-12-12 | 2016-06-20 | Massachusetts Inst Technology | CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS |
| CA2895155C (en) | 2012-12-17 | 2021-07-06 | President And Fellows Of Harvard College | Rna-guided human genome engineering |
| EP3608308B1 (en) | 2013-03-08 | 2021-07-21 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| ES2895651T3 (es) | 2013-12-19 | 2022-02-22 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
| EP3169309B1 (en) | 2014-07-16 | 2023-05-10 | Novartis AG | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| EP3265559B1 (en) | 2015-03-03 | 2021-01-06 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| JP6937740B2 (ja) * | 2015-07-28 | 2021-09-22 | ダニスコ・ユーエス・インク | ゲノム編集システムおよび使用方法 |
| ES2964690T3 (es) | 2015-09-21 | 2024-04-09 | Trilink Biotechnologies Llc | Método para sintetizar ARN con caperuza 5' |
| TWI773666B (zh) | 2016-03-30 | 2022-08-11 | 美商英特利亞醫療公司 | Crispr/cas 組分之脂質奈米粒子調配物 |
| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| WO2018067447A1 (en) | 2016-10-03 | 2018-04-12 | Itellia Therapeutics, Inc. | Improved methods for identifying double strand break sites |
| AR113154A1 (es) * | 2017-09-29 | 2020-01-29 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para edición del genoma |
| EP3688161A1 (en) * | 2017-09-29 | 2020-08-05 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis |
| PT3688162T (pt) * | 2017-09-29 | 2024-04-23 | Intellia Therapeutics Inc | Formulações |
| WO2019237069A1 (en) * | 2018-06-08 | 2019-12-12 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
| BR112021005718A2 (pt) * | 2018-09-28 | 2021-06-22 | Intellia Therapeutics, Inc. | composições e métodos para edição de genes de lactato desidrogenase (ldha) |
-
2020
- 2020-03-27 EP EP20720292.0A patent/EP3946598A1/en active Pending
- 2020-03-27 AU AU2020244887A patent/AU2020244887A1/en active Pending
- 2020-03-27 BR BR112021019196A patent/BR112021019196A2/pt unknown
- 2020-03-27 EA EA202192636A patent/EA202192636A1/ru unknown
- 2020-03-27 MX MX2021011565A patent/MX2021011565A/es unknown
- 2020-03-27 KR KR1020217035004A patent/KR20220004648A/ko active Pending
- 2020-03-27 JP JP2021557397A patent/JP7631215B2/ja active Active
- 2020-03-27 CN CN202080038208.6A patent/CN113874076A/zh active Pending
- 2020-03-27 WO PCT/US2020/025513 patent/WO2020198697A1/en not_active Ceased
- 2020-03-27 CA CA3134271A patent/CA3134271A1/en active Pending
- 2020-03-27 PH PH1/2021/552301A patent/PH12021552301A1/en unknown
- 2020-03-27 SG SG11202110434PA patent/SG11202110434PA/en unknown
-
2021
- 2021-09-19 IL IL286524A patent/IL286524A/en unknown
- 2021-09-27 US US17/486,735 patent/US20230044994A1/en not_active Abandoned
- 2021-10-28 CO CONC2021/0014559A patent/CO2021014559A2/es unknown
-
2023
- 2023-07-31 US US18/362,867 patent/US20240124897A1/en active Pending
-
2025
- 2025-02-04 JP JP2025016673A patent/JP2025072483A/ja active Pending
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