JPWO2019204514A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2019204514A5 JPWO2019204514A5 JP2020557945A JP2020557945A JPWO2019204514A5 JP WO2019204514 A5 JPWO2019204514 A5 JP WO2019204514A5 JP 2020557945 A JP2020557945 A JP 2020557945A JP 2020557945 A JP2020557945 A JP 2020557945A JP WO2019204514 A5 JPWO2019204514 A5 JP WO2019204514A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- abca4
- exons
- splicing
- nucleotides
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims description 171
- 108020004707 nucleic acids Proteins 0.000 claims description 153
- 102000039446 nucleic acids Human genes 0.000 claims description 153
- 101000801643 Homo sapiens Retinal-specific phospholipid-transporting ATPase ABCA4 Proteins 0.000 claims description 142
- 102100033617 Retinal-specific phospholipid-transporting ATPase ABCA4 Human genes 0.000 claims description 142
- 108700024394 Exon Proteins 0.000 claims description 101
- 239000002773 nucleotide Substances 0.000 claims description 97
- 125000003729 nucleotide group Chemical group 0.000 claims description 97
- 230000035772 mutation Effects 0.000 claims description 52
- 108091092195 Intron Proteins 0.000 claims description 30
- 239000008194 pharmaceutical composition Substances 0.000 claims description 26
- 102100035673 Centrosomal protein of 290 kDa Human genes 0.000 claims description 23
- 101710198317 Centrosomal protein of 290 kDa Proteins 0.000 claims description 23
- 230000000295 complement effect Effects 0.000 claims description 18
- 239000013612 plasmid Substances 0.000 claims description 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 9
- 108090000565 Capsid Proteins Proteins 0.000 claims description 8
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 8
- 230000001717 pathogenic effect Effects 0.000 claims description 7
- 101150039555 ABCA4 gene Proteins 0.000 claims description 5
- 241000702421 Dependoparvovirus Species 0.000 claims description 5
- 208000027073 Stargardt disease Diseases 0.000 claims description 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 210000004027 cell Anatomy 0.000 claims description 4
- 238000002347 injection Methods 0.000 claims description 4
- 239000007924 injection Substances 0.000 claims description 4
- 210000000608 photoreceptor cell Anatomy 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims description 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 210000000234 capsid Anatomy 0.000 claims description 2
- 239000002299 complementary DNA Substances 0.000 claims description 2
- 238000010253 intravenous injection Methods 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 125000006850 spacer group Chemical group 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims 4
- 229940079593 drug Drugs 0.000 claims 4
- 239000008177 pharmaceutical agent Substances 0.000 claims 3
- 108020004414 DNA Proteins 0.000 claims 2
- 238000000034 method Methods 0.000 description 7
- 208000035475 disorder Diseases 0.000 description 5
- 230000001566 pro-viral effect Effects 0.000 description 2
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 description 1
- 101150078156 Cep290 gene Proteins 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024073380A JP2024099770A (ja) | 2018-04-17 | 2024-04-30 | トランススプライシング分子 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862658667P | 2018-04-17 | 2018-04-17 | |
| US201862658658P | 2018-04-17 | 2018-04-17 | |
| US62/658,658 | 2018-04-17 | ||
| US62/658,667 | 2018-04-17 | ||
| PCT/US2019/027981 WO2019204514A1 (en) | 2018-04-17 | 2019-04-17 | Trans-splicing molecules |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024073380A Division JP2024099770A (ja) | 2018-04-17 | 2024-04-30 | トランススプライシング分子 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021520837A JP2021520837A (ja) | 2021-08-26 |
| JP2021520837A5 JP2021520837A5 (https=) | 2022-04-25 |
| JPWO2019204514A5 true JPWO2019204514A5 (https=) | 2022-04-25 |
| JP7561624B2 JP7561624B2 (ja) | 2024-10-04 |
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 After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024073380A Pending JP2024099770A (ja) | 2018-04-17 | 2024-04-30 | トランススプライシング分子 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11993776B2 (https=) |
| EP (1) | EP3781213A4 (https=) |
| JP (2) | JP7561624B2 (https=) |
| KR (2) | KR102866133B1 (https=) |
| CN (1) | CN112449605A (https=) |
| AU (1) | AU2019255708B2 (https=) |
| BR (1) | BR112020021228A2 (https=) |
| CA (1) | CA3097004A1 (https=) |
| IL (1) | IL278019A (https=) |
| MX (2) | MX2020010959A (https=) |
| WO (1) | WO2019204514A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3097004A1 (en) | 2018-04-17 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Trans-splicing molecules |
| WO2020214973A1 (en) * | 2019-04-17 | 2020-10-22 | The Trustees Of The University Of Pennsylvania | Triple helix terminator for efficient rna trans-splicing |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| AU2023269865A1 (en) * | 2022-05-13 | 2024-11-21 | 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 |
| CN121099993A (zh) * | 2023-02-15 | 2025-12-09 | 海鞘治疗公司 | Htt反式剪接分子 |
| WO2025166360A1 (en) * | 2024-02-03 | 2025-08-07 | Duke University | Compositions for and methods of engineering the transcriptome |
| WO2026012439A1 (en) * | 2024-07-11 | 2026-01-15 | Sichuan Real & Best Biotech Co., Ltd. | Rna trans-splicing molecules and systems |
Family Cites Families (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6280978B1 (en) | 1995-12-15 | 2001-08-28 | Intronn Holdings, Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| DE69636937T3 (de) | 1995-12-15 | 2011-01-05 | Virxsys Corp. | Durch trans-spaltung erhaltene therapeutische molekule |
| JP2001510557A (ja) | 1996-10-04 | 2001-07-31 | イントロン エルエルシー | サンプル収集デバイス及びマーカーを用いたサンプル収集方法、及びサンプル確認、ラボの評価及び／又は認定の際の比較試料としてのマーカーの使用 |
| US6929907B2 (en) | 1999-12-31 | 2005-08-16 | North Carolina State University | Methods and compositions for determining the purity of chemically synthesized nucleic acids |
| JP4500540B2 (ja) | 2001-06-29 | 2010-07-14 | ヴェリ‐キュー，インコーポレイテッド | 化学的に合成された核酸の純度を決定し、かつ化学的に合成された核酸を精製するための方法および組成物 |
| 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 |
| AU2003213270A1 (en) | 2002-02-25 | 2003-09-09 | Intronn, Inc. | Trans-splicing mediated imaging of gene expression |
| US7399753B2 (en) | 2002-02-25 | 2008-07-15 | Virxsys Corporation | Trans-splicing mediated photodynamic therapy |
| AU2003247505A1 (en) | 2002-06-05 | 2003-12-22 | University Of Iowa Research Foundation | Spliceosome mediated rna trans-splicing in stem cells |
| CA2488246A1 (en) | 2002-06-05 | 2003-12-18 | Intronn, Inc. | Spliceosome mediated rna trans-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 |
| EP1579004B1 (en) | 2002-10-23 | 2010-06-16 | VIRxSYS Corporation | Screening methods for identification of efficient pre-trans-splicing molecules |
| WO2005070948A1 (en) | 2004-01-23 | 2005-08-04 | Intronn, Inc. | Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing |
| JP2007518423A (ja) | 2004-01-23 | 2007-07-12 | イントロン、インコーポレイテッド | スプライセオソーム仲介型ｒｎａトランススプライシングを使用するアポａ−１及びその変異体の発現 |
| 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 |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| BR112014004895B1 (pt) * | 2011-09-05 | 2022-07-05 | Stichting Radboud Universitair Medisch Centrum | Oligonucleotídeos antissentido para o tratamento de amaurose congênita de leber |
| EP2872183B1 (en) | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| CN104955951A (zh) | 2012-10-16 | 2015-09-30 | 麻省理工学院 | 稳定的非多腺苷酸化rna的产生 |
| 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 |
| CA2909733C (en) | 2013-04-18 | 2023-10-17 | 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 |
| WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| WO2016005524A1 (en) | 2014-07-09 | 2016-01-14 | Lexogen Gmbh | Methods and products for quantifying rna transcript variants |
| RS63416B1 (sr) * | 2015-03-03 | 2022-08-31 | Fond Telethon | Sistem višestrukih vektora i njegove primene |
| EP3377116A4 (en) | 2015-11-19 | 2019-07-10 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE |
| EP3390634A4 (en) * | 2015-12-14 | 2019-08-14 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES |
| KR20200036912A (ko) * | 2017-07-31 | 2020-04-07 | 리플렉션 바이오테크놀러지스 리미티드 | 안과 질환을 위한 세포 모델 및 치료요법 |
| US11795455B2 (en) | 2017-07-31 | 2023-10-24 | Massachusetts Institute Of Technology | RNA cleavage-induced transcript stabilizer and uses thereof |
| CA3097004A1 (en) | 2018-04-17 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Trans-splicing molecules |
| WO2020214990A1 (en) | 2019-04-17 | 2020-10-22 | Ascidian Therapeutics, Inc. | Abca4 cell line and methods of use |
| WO2020214973A1 (en) | 2019-04-17 | 2020-10-22 | The Trustees Of The University Of Pennsylvania | Triple helix terminator for efficient rna trans-splicing |
| CA3138520C (en) | 2019-05-01 | 2023-10-10 | The Procter & Gamble Company | Probiotic bacterial strains that produce short chain fatty acids and compositions comprising same |
| AU2023269865A1 (en) | 2022-05-13 | 2024-11-21 | Ascidian Therapeutics, Inc. | Abca4 trans-splicing molecules |
-
2019
- 2019-04-17 CA CA3097004A patent/CA3097004A1/en active Pending
- 2019-04-17 KR KR1020207032936A patent/KR102866133B1/ko active Active
- 2019-04-17 KR KR1020257032132A patent/KR20250150157A/ko active Pending
- 2019-04-17 AU AU2019255708A patent/AU2019255708B2/en active Active
- 2019-04-17 MX MX2020010959A patent/MX2020010959A/es unknown
- 2019-04-17 US US17/047,496 patent/US11993776B2/en active Active
- 2019-04-17 BR BR112020021228-8A patent/BR112020021228A2/pt unknown
- 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
-
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
-
2025
- 2025-09-17 US US19/331,448 patent/US20260078380A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021520837A5 (https=) | ||
| JP7707261B2 (ja) | 筋疾患の治療のための改変ａａｖカプシドポリペプチド | |
| JP7482549B2 (ja) | 新規のマイクロジストロフィンおよび使用の関連する方法 | |
| JP2024099770A5 (https=) | ||
| US20250161485A1 (en) | Aav capsid variants and uses thereof | |
| TW202417466A (zh) | Ａａｖ蛋白殼變異體及其用途 | |
| JP2021534814A5 (https=) | ||
| JPWO2019204514A5 (https=) | ||
| WO2020142714A1 (en) | Aav expression cassette and aav vectors comprising the same | |
| TW202417467A (zh) | Ａａｖ蛋白殼變異體及其用途 | |
| WO2025038430A1 (en) | Aav capsid variants and uses thereof | |
| WO2025038802A1 (en) | Compositions and methods for the treatment of disorders related to ataxin-2 | |
| JPWO2020082047A5 (https=) | ||
| WO2016037931A1 (en) | Nucleic acid constructs and expression vectors for gene therapy of acute porphyrias and other diseases | |
| JP7811409B2 (ja) | 筋ジストロフィー治療のための遺伝子導入発現カセット | |
| EP4705318A2 (en) | Kidney-targeting aav capsids and methods of use thereof | |
| WO2025090628A1 (en) | Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies | |
| HK40092059A (zh) | 用於治疗突触核蛋白病的组合物和方法 | |
| HK40114438A (zh) | Aav衣壳变体及其用途 | |
| HK1238297A1 (en) | Novel micro-dystrophins and related methods of use | |
| HK1238297B (en) | Novel micro-dystrophins and related methods of use |