JPWO2022015715A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2022015715A5 JPWO2022015715A5 JP2023502675A JP2023502675A JPWO2022015715A5 JP WO2022015715 A5 JPWO2022015715 A5 JP WO2022015715A5 JP 2023502675 A JP2023502675 A JP 2023502675A JP 2023502675 A JP2023502675 A JP 2023502675A JP WO2022015715 A5 JPWO2022015715 A5 JP WO2022015715A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- mirna
- coding sequence
- sequence
- vector
- 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
- 108091026890 Coding region Proteins 0.000 claims 50
- 108091070501 miRNA Proteins 0.000 claims 47
- 239000002679 microRNA Substances 0.000 claims 47
- 239000013598 vector Substances 0.000 claims 36
- 150000007523 nucleic acids Chemical group 0.000 claims 22
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 19
- 230000001105 regulatory effect Effects 0.000 claims 14
- 101001018717 Homo sapiens Mitofusin-2 Proteins 0.000 claims 13
- 102000057083 human MFN2 Human genes 0.000 claims 13
- 239000013603 viral vector Substances 0.000 claims 12
- 241000702421 Dependoparvovirus Species 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 210000000234 capsid Anatomy 0.000 claims 7
- 210000004027 cell Anatomy 0.000 claims 7
- 239000000203 mixture Substances 0.000 claims 7
- 108020004999 messenger RNA Proteins 0.000 claims 5
- 108091037620 miR-1693 stem-loop Proteins 0.000 claims 3
- 201000001119 neuropathy Diseases 0.000 claims 3
- 230000007823 neuropathy Effects 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 208000033808 peripheral neuropathy Diseases 0.000 claims 3
- 125000006850 spacer group Chemical group 0.000 claims 3
- 230000008685 targeting Effects 0.000 claims 3
- 230000003612 virological effect Effects 0.000 claims 3
- RZVAJINKPMORJF-UHFFFAOYSA-N Acetaminophen Chemical compound CC(=O)NC1=CC=C(O)C=C1 RZVAJINKPMORJF-UHFFFAOYSA-N 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
- UGJMXCAKCUNAIE-UHFFFAOYSA-N Gabapentin Chemical compound OC(=O)CC1(CN)CCCCC1 UGJMXCAKCUNAIE-UHFFFAOYSA-N 0.000 claims 2
- 241000713666 Lentivirus Species 0.000 claims 2
- 241000700584 Simplexvirus Species 0.000 claims 2
- 102000001435 Synapsin Human genes 0.000 claims 2
- 108050009621 Synapsin Proteins 0.000 claims 2
- 230000000692 anti-sense effect Effects 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 239000000041 non-steroidal anti-inflammatory agent Substances 0.000 claims 2
- 229940021182 non-steroidal anti-inflammatory drug Drugs 0.000 claims 2
- 239000002245 particle Substances 0.000 claims 2
- 208000031996 Autosomal dominant Charcot-Marie-Tooth disease type 2 Diseases 0.000 claims 1
- 208000035758 Charcot-Marie-Tooth disease type 2S Diseases 0.000 claims 1
- 101000575685 Homo sapiens Synembryn-B Proteins 0.000 claims 1
- 101150050341 Mfn2 gene Proteins 0.000 claims 1
- 208000012902 Nervous system disease Diseases 0.000 claims 1
- 241000125945 Protoparvovirus Species 0.000 claims 1
- 102100026014 Synembryn-B Human genes 0.000 claims 1
- 229940123445 Tricyclic antidepressant Drugs 0.000 claims 1
- 239000001961 anticonvulsive agent Substances 0.000 claims 1
- 229960003965 antiepileptics Drugs 0.000 claims 1
- 239000007900 aqueous suspension Substances 0.000 claims 1
- 229960000623 carbamazepine Drugs 0.000 claims 1
- FFGPTBGBLSHEPO-UHFFFAOYSA-N carbamazepine Chemical compound C1=CC2=CC=CC=C2N(C(=O)N)C2=CC=CC=C21 FFGPTBGBLSHEPO-UHFFFAOYSA-N 0.000 claims 1
- HISOCSRUFLPKDE-KLXQUTNESA-N cmt-2 Chemical compound C1=CC=C2[C@](O)(C)C3CC4C(N(C)C)C(O)=C(C#N)C(=O)[C@@]4(O)C(O)=C3C(=O)C2=C1O HISOCSRUFLPKDE-KLXQUTNESA-N 0.000 claims 1
- 238000011970 concomitant therapy Methods 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 229960002870 gabapentin Drugs 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 108091058006 miR1518 stem-loop Proteins 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 229960005489 paracetamol Drugs 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 230000000717 retained effect Effects 0.000 claims 1
- 239000003029 tricyclic antidepressant agent Substances 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063051336P | 2020-07-13 | 2020-07-13 | |
| US63/051,336 | 2020-07-13 | ||
| US202163173045P | 2021-04-09 | 2021-04-09 | |
| US63/173,045 | 2021-04-09 | ||
| PCT/US2021/041406 WO2022015715A1 (en) | 2020-07-13 | 2021-07-13 | Compositions useful for treatment of charcot-marie-tooth disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023534037A JP2023534037A (ja) | 2023-08-07 |
| JPWO2022015715A5 true JPWO2022015715A5 (enExample) | 2024-07-23 |
Family
ID=77317406
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023502675A Pending JP2023534037A (ja) | 2020-07-13 | 2021-07-13 | シャルコー・マリー・トゥース病の治療に有用な組成物 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20230270884A1 (enExample) |
| EP (1) | EP4179097A1 (enExample) |
| JP (1) | JP2023534037A (enExample) |
| KR (1) | KR20230038503A (enExample) |
| CN (1) | CN116438311A (enExample) |
| AU (1) | AU2021309645A1 (enExample) |
| BR (1) | BR112023000578A2 (enExample) |
| CA (1) | CA3185281A1 (enExample) |
| CO (1) | CO2023001500A2 (enExample) |
| IL (1) | IL299762A (enExample) |
| MX (1) | MX2023000658A (enExample) |
| TW (1) | TW202216244A (enExample) |
| WO (1) | WO2022015715A1 (enExample) |
| ZA (1) | ZA202300505B (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023049846A1 (en) * | 2021-09-24 | 2023-03-30 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| CN121127599A (zh) | 2022-12-17 | 2025-12-12 | 宾夕法尼亚州大学信托人 | 具有心肌和骨骼肌特异性靶向基序的重组aav突变载体及含有其的组合物 |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| WO2024155966A1 (en) * | 2023-01-20 | 2024-07-25 | Loma Linda University Health | Methods and compositions for treatment of niemann-pick disease type c and charcot-marie-tooth disease |
| WO2024261139A1 (en) | 2023-06-21 | 2024-12-26 | Fundació Institut De Recerca Biomèdica (Irb Barcelona) | Variants of mfn2 and their use in the treatment/prevention of diseases associated with alterations in endoplasmatic reticulum function |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| MX353900B (es) | 2008-11-07 | 2018-02-01 | Massachusetts Inst Technology | Lipidoides de aminoalcohol y usos de los mismos. |
| SG10201502270TA (en) | 2010-03-29 | 2015-05-28 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| EP3391877A1 (en) | 2010-04-08 | 2018-10-24 | The Trustees of Princeton University | Preparation of lipid nanoparticles |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| US20140206753A1 (en) | 2011-06-08 | 2014-07-24 | Shire Human Genetic Therapies, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| WO2013182683A1 (en) | 2012-06-08 | 2013-12-12 | Ethris Gmbh | Pulmonary delivery of messenger rna |
| US20150366997A1 (en) | 2012-12-07 | 2015-12-24 | Shire Human Genetics Therapies, Inc. | COMPOSITIONS AND METHODS FOR mRNA DELIVERY |
| CN105873902B (zh) | 2013-11-18 | 2019-03-08 | 阿克丘勒斯治疗公司 | 用于rna递送的可电离的阳离子脂质 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| WO2017100704A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| WO2018168956A1 (ja) | 2017-03-16 | 2018-09-20 | 株式会社デンソー | 自己位置推定装置 |
| EP3758724A4 (en) | 2018-02-27 | 2022-07-06 | The Trustees of The University of Pennsylvania | NOVEL VECTORS OF ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
| MX2020008932A (es) | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| CA3110665A1 (en) * | 2018-08-29 | 2020-03-05 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of mutant gars protein |
| JOP20210160A1 (ar) | 2018-12-21 | 2023-01-30 | Univ Pennsylvania | تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg |
-
2021
- 2021-07-13 TW TW110125641A patent/TW202216244A/zh unknown
- 2021-07-13 MX MX2023000658A patent/MX2023000658A/es unknown
- 2021-07-13 WO PCT/US2021/041406 patent/WO2022015715A1/en not_active Ceased
- 2021-07-13 AU AU2021309645A patent/AU2021309645A1/en active Pending
- 2021-07-13 JP JP2023502675A patent/JP2023534037A/ja active Pending
- 2021-07-13 CA CA3185281A patent/CA3185281A1/en active Pending
- 2021-07-13 IL IL299762A patent/IL299762A/en unknown
- 2021-07-13 US US18/005,504 patent/US20230270884A1/en active Pending
- 2021-07-13 BR BR112023000578A patent/BR112023000578A2/pt unknown
- 2021-07-13 KR KR1020237003861A patent/KR20230038503A/ko active Pending
- 2021-07-13 CN CN202180062652.6A patent/CN116438311A/zh active Pending
- 2021-07-13 EP EP21754870.0A patent/EP4179097A1/en active Pending
-
2023
- 2023-01-11 ZA ZA2023/00505A patent/ZA202300505B/en unknown
- 2023-02-10 CO CONC2023/0001500A patent/CO2023001500A2/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20220331409A1 (en) | Factor ix gene therapy | |
| US12359219B2 (en) | Transgene cassettes designed to express a human MECP2 gene | |
| RU2018129159A (ru) | Рекомбинантные aav векторы, экспрессирующие остеопротективные гены, включая has2 и лубрицин, пригодные при лечении остеоартрита и сходных заболеваний суставов у млекопитающих | |
| US20220281923A1 (en) | Aav capsid variants for gene therapy | |
| KR20210096168A (ko) | 신경퇴행성 질환을 위한 유전자 요법 | |
| US12281314B2 (en) | Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder | |
| CN114072514A (zh) | 用于治疗atp酶介导的疾病的组合物和方法 | |
| JPWO2020069461A5 (enExample) | ||
| CN114276419B (zh) | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 | |
| US20200268904A1 (en) | Wilson's Disease Gene Therapy | |
| JPWO2022015715A5 (enExample) | ||
| US20210324411A1 (en) | Compositions and methods for treatment of neurological disorders | |
| JP2020510433A5 (enExample) | ||
| JPWO2020232297A5 (enExample) | ||
| US12415850B2 (en) | Compositions and methods for regulating production of an antibody like protein and ribonucleic acid | |
| JP2022551554A (ja) | 遺伝子構築物 | |
| JP7785352B2 (ja) | 遺伝子治療のためのａａｖカプシドバリアント | |
| CN111093716A (zh) | 用于治疗神经性疼痛的方法和组合物 | |
| RU2021102051A (ru) | Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии | |
| JPWO2022016055A5 (enExample) | ||
| OA21365A (en) | Synergistic effect of SMN1 and MIR-23A in treating spinal muscular atrophy. | |
| JP2024528997A (ja) | 放射線誘発性唾液機能低下を予防するためのａｑｐ１遺伝子療法 | |
| JPWO2020176562A5 (enExample) | ||
| RU2021105703A (ru) | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови IX, и ее применение | |
| JPWO2021081338A5 (enExample) |