JP2024099770A5 - - Google Patents
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- JP2024099770A5 JP2024099770A5 JP2024073380A JP2024073380A JP2024099770A5 JP 2024099770 A5 JP2024099770 A5 JP 2024099770A5 JP 2024073380 A JP2024073380 A JP 2024073380A JP 2024073380 A JP2024073380 A JP 2024073380A JP 2024099770 A5 JP2024099770 A5 JP 2024099770A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotides
- nucleic acid
- acid trans
- splicing molecule
- length
- 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
- 239000002773 nucleotide Substances 0.000 claims 21
- 125000003729 nucleotide group Chemical group 0.000 claims 21
- 150000007523 nucleic acids Chemical class 0.000 claims 18
- 108020004707 nucleic acids Proteins 0.000 claims 17
- 102000039446 nucleic acids Human genes 0.000 claims 17
- 101000801643 Homo sapiens Retinal-specific phospholipid-transporting ATPase ABCA4 Proteins 0.000 claims 7
- 102100033617 Retinal-specific phospholipid-transporting ATPase ABCA4 Human genes 0.000 claims 7
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 230000035772 mutation Effects 0.000 claims 5
- 108090000565 Capsid Proteins Proteins 0.000 claims 4
- 102100023321 Ceruloplasmin Human genes 0.000 claims 4
- 108700024394 Exon Proteins 0.000 claims 4
- 230000000295 complement effect Effects 0.000 claims 3
- 101150039555 ABCA4 gene Proteins 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 208000027073 Stargardt disease Diseases 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 210000000608 photoreceptor cell Anatomy 0.000 claims 2
- 239000013612 plasmid Substances 0.000 claims 2
- 230000001566 pro-viral effect Effects 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 239000002299 complementary DNA Substances 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 125000006850 spacer group Chemical group 0.000 claims 1
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862658658P | 2018-04-17 | 2018-04-17 | |
| US201862658667P | 2018-04-17 | 2018-04-17 | |
| US62/658,658 | 2018-04-17 | ||
| US62/658,667 | 2018-04-17 | ||
| JP2020557945A JP7561624B2 (ja) | 2018-04-17 | 2019-04-17 | トランススプライシング分子 |
| PCT/US2019/027981 WO2019204514A1 (en) | 2018-04-17 | 2019-04-17 | Trans-splicing molecules |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020557945A Division JP7561624B2 (ja) | 2018-04-17 | 2019-04-17 | トランススプライシング分子 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024099770A JP2024099770A (ja) | 2024-07-25 |
| JP2024099770A5 true JP2024099770A5 (enExample) | 2025-01-21 |
Family
ID=68239871
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020557945A Active JP7561624B2 (ja) | 2018-04-17 | 2019-04-17 | トランススプライシング分子 |
| JP2024073380A Pending JP2024099770A (ja) | 2018-04-17 | 2024-04-30 | トランススプライシング分子 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020557945A Active JP7561624B2 (ja) | 2018-04-17 | 2019-04-17 | トランススプライシング分子 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11993776B2 (enExample) |
| EP (1) | EP3781213A4 (enExample) |
| JP (2) | JP7561624B2 (enExample) |
| KR (2) | KR102866133B1 (enExample) |
| CN (1) | CN112449605A (enExample) |
| AU (1) | AU2019255708A1 (enExample) |
| BR (1) | BR112020021228A2 (enExample) |
| CA (1) | CA3097004A1 (enExample) |
| IL (1) | IL278019A (enExample) |
| MX (2) | MX2020010959A (enExample) |
| WO (1) | WO2019204514A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| KR20220002910A (ko) * | 2019-04-17 | 2022-01-07 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| JP2024533163A (ja) | 2021-09-03 | 2024-09-12 | タシト・セラピューティクス・インコーポレーテッド | スプライソソーム構成要素のリクルートを介したｒｎａ編集 |
| WO2023220742A2 (en) * | 2022-05-13 | 2023-11-16 | Ascidian Therapeutics, Inc. | Abca4 trans-splicing molecules |
| WO2024011203A2 (en) * | 2022-07-07 | 2024-01-11 | Intergalactic Therapeutics, Inc. | Ocular vectors and uses thereof |
| EP4594505A1 (en) * | 2022-09-30 | 2025-08-06 | Centre National de la Recherche Scientifique | Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations |
| AU2024223513A1 (en) * | 2023-02-15 | 2025-09-18 | Ascidian Therapeutics, Inc. | Htt trans-splicing molecules |
| WO2025166360A1 (en) * | 2024-02-03 | 2025-08-07 | Duke University | Compositions for and methods of engineering the transcriptome |
Family Cites Families (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0883344B2 (en) | 1995-12-15 | 2010-06-09 | VIRxSYS Corporation | Therapeutic molecules generated by trans-splicing |
| US6280978B1 (en) | 1995-12-15 | 2001-08-28 | Intronn Holdings, Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| AU735272B2 (en) | 1996-10-04 | 2001-07-05 | Intronn Llc | Sample collection devices and methods using markers and the use of such markers as controls in sample validation, laboratory evaluation and/or accreditation |
| US6929907B2 (en) | 1999-12-31 | 2005-08-16 | North Carolina State University | Methods and compositions for determining the purity of chemically synthesized nucleic acids |
| CA2452690C (en) | 2001-06-29 | 2016-08-16 | Veri-Q, Inc. | Methods and compositions for determining the purity of and purifying chemically synthesized nucleic acids |
| JP2006505242A (ja) | 2002-02-12 | 2006-02-16 | イントロン，インコーポレーテッド | スプライセオソームにより媒介されるｒｎａトランス−スプライシングにおいて使用するための方法および組成物 |
| US7399753B2 (en) | 2002-02-25 | 2008-07-15 | Virxsys Corporation | Trans-splicing mediated photodynamic therapy |
| EP1485397A4 (en) | 2002-02-25 | 2005-05-25 | Intronn Inc | IMAGING OF GENE EXPRESSION MEDIATED BY TRANS-SPISSAGE |
| CA2488249A1 (en) | 2002-06-05 | 2003-12-18 | Intronn, Inc. | Spliceosome mediated rna trans-splicing in stem cells |
| EP1521766B1 (en) | 2002-06-05 | 2012-11-07 | VIRxSYS Corporation | SPLICEOSOME MEDIATED RNA i TRANS /i -SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING |
| US20040018622A1 (en) | 2002-07-17 | 2004-01-29 | Mitchell Lloyd G. | Spliceosome mediated RNA trans-splicing for correction of skin disorders |
| DE60333032D1 (de) | 2002-10-23 | 2010-07-29 | Virxsys Corp | Screening-verfahren zur identifizierung wirksamer prä-trans-spleissmoleküle |
| WO2005070948A1 (en) | 2004-01-23 | 2005-08-04 | Intronn, Inc. | Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing |
| US20060177933A1 (en) | 2004-01-23 | 2006-08-10 | Madaiah Puttaraju | Expression of apoA-1 and variants thereof using spliceosome mediated RNA trans-splicing |
| WO2006026611A2 (en) | 2004-08-31 | 2006-03-09 | Mitchell Lloyd G | Reverse transcriptase mediated rna gene expression |
| US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US20090214478A1 (en) | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| AU2013234376B2 (en) * | 2008-10-22 | 2016-07-28 | Acucela, Inc. | Compounds for treating ophthalmic diseases and disorders |
| US8648053B2 (en) | 2010-10-20 | 2014-02-11 | Rosalind Franklin University Of Medicine And Science | Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome |
| WO2012158757A1 (en) | 2011-05-16 | 2012-11-22 | The Trustees Of The University Of Pennsylvania | Proviral plasmids for production of recombinant adeno-associated virus |
| PL2753694T3 (pl) * | 2011-09-05 | 2018-01-31 | Stichting Katholieke Univ | Oligonukleotydy antysensowne do leczenia wrodzonej ślepoty Lebera |
| CA2878171C (en) | 2012-07-11 | 2021-04-27 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| WO2014062801A1 (en) | 2012-10-16 | 2014-04-24 | Massachusetts Institute Of Technology | Production of stable non-polyadenylated rnas |
| GB201219762D0 (en) | 2012-11-02 | 2012-12-19 | Bauer Johann | A RNA trans-splicing molecule (RTM) for use in the treatment of cancer |
| US10266845B2 (en) | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US10494645B2 (en) | 2013-04-18 | 2019-12-03 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| WO2015009575A1 (en) | 2013-07-16 | 2015-01-22 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to cep290 |
| EP3553176A1 (en) | 2014-03-10 | 2019-10-16 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| JP6930910B2 (ja) | 2014-07-09 | 2021-09-01 | レクソジェン・ゲゼルシャフト・ミット・ベシュレンクテル・ハフツングＬＥＸＯＧＥＮ ＧｍｂＨ | Ｒｎａ転写産物バリアントを定量するための方法及び製品 |
| DK3265571T3 (da) * | 2015-03-03 | 2022-06-27 | Fond Telethon | Fler-vektorsystem og anvendelse heraf |
| WO2017087900A1 (en) * | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| EP3390634A4 (en) * | 2015-12-14 | 2019-08-14 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES |
| TWI820034B (zh) | 2017-07-31 | 2023-11-01 | 香港商映像生物有限公司 | 眼部疾病之細胞模式及用於眼部疾病的療法 |
| US11795455B2 (en) | 2017-07-31 | 2023-10-24 | Massachusetts Institute Of Technology | RNA cleavage-induced transcript stabilizer and uses thereof |
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| KR20220002910A (ko) | 2019-04-17 | 2022-01-07 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자 |
| WO2020214990A1 (en) | 2019-04-17 | 2020-10-22 | Ascidian Therapeutics, Inc. | Abca4 cell line and methods of use |
| EP3962295A1 (en) | 2019-05-01 | 2022-03-09 | The Procter & Gamble Company | Probiotic bacterial strains that produce short chain fatty acids and compositions comprising same |
| WO2023220742A2 (en) | 2022-05-13 | 2023-11-16 | Ascidian Therapeutics, Inc. | Abca4 trans-splicing molecules |
-
2019
- 2019-04-17 KR KR1020207032936A patent/KR102866133B1/ko active Active
- 2019-04-17 CA CA3097004A patent/CA3097004A1/en active Pending
- 2019-04-17 EP EP19789043.7A patent/EP3781213A4/en active Pending
- 2019-04-17 CN CN201980040706.1A patent/CN112449605A/zh active Pending
- 2019-04-17 WO PCT/US2019/027981 patent/WO2019204514A1/en not_active Ceased
- 2019-04-17 JP JP2020557945A patent/JP7561624B2/ja active Active
- 2019-04-17 US US17/047,496 patent/US11993776B2/en active Active
- 2019-04-17 MX MX2020010959A patent/MX2020010959A/es unknown
- 2019-04-17 KR KR1020257032132A patent/KR20250150157A/ko active Pending
- 2019-04-17 BR BR112020021228-8A patent/BR112020021228A2/pt unknown
- 2019-04-17 AU AU2019255708A patent/AU2019255708A1/en active Pending
-
2020
- 2020-10-13 IL IL278019A patent/IL278019A/en unknown
- 2020-10-15 MX MX2025007209A patent/MX2025007209A/es unknown
-
2024
- 2024-04-30 JP JP2024073380A patent/JP2024099770A/ja active Pending
- 2024-05-24 US US18/673,713 patent/US12442003B2/en active Active
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