JPWO2021202532A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2021202532A5 JPWO2021202532A5 JP2022559589A JP2022559589A JPWO2021202532A5 JP WO2021202532 A5 JPWO2021202532 A5 JP WO2021202532A5 JP 2022559589 A JP2022559589 A JP 2022559589A JP 2022559589 A JP2022559589 A JP 2022559589A JP WO2021202532 A5 JPWO2021202532 A5 JP WO2021202532A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- raav
- seq
- enhancer
- promoter
- 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 description 26
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 15
- 230000008488 polyadenylation Effects 0.000 claims description 15
- 241000702421 Dependoparvovirus Species 0.000 claims description 12
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims description 12
- 210000000234 capsid Anatomy 0.000 claims description 12
- 239000003623 enhancer Substances 0.000 claims description 12
- 241000701022 Cytomegalovirus Species 0.000 claims description 11
- 101001098989 Homo sapiens Propionyl-CoA carboxylase alpha chain, mitochondrial Proteins 0.000 claims description 11
- 101001098982 Homo sapiens Propionyl-CoA carboxylase beta chain, mitochondrial Proteins 0.000 claims description 11
- 102100039022 Propionyl-CoA carboxylase alpha chain, mitochondrial Human genes 0.000 claims description 11
- 102100039025 Propionyl-CoA carboxylase beta chain, mitochondrial Human genes 0.000 claims description 11
- 108091033319 polynucleotide Proteins 0.000 claims description 11
- 102000040430 polynucleotide Human genes 0.000 claims description 11
- 239000002157 polynucleotide Substances 0.000 claims description 11
- MXCVHSXCXPHOLP-UHFFFAOYSA-N 4-oxo-6-propylchromene-2-carboxylic acid Chemical compound O1C(C(O)=O)=CC(=O)C2=CC(CCC)=CC=C21 MXCVHSXCXPHOLP-UHFFFAOYSA-N 0.000 claims description 10
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 10
- 150000007523 nucleic acids Chemical group 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 9
- 238000000034 method Methods 0.000 claims description 7
- 108700002232 Immediate-Early Genes Proteins 0.000 claims description 6
- 108010071690 Prealbumin Proteins 0.000 claims description 6
- 102000009190 Transthyretin Human genes 0.000 claims description 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
- 102000002248 Thyroxine-Binding Globulin Human genes 0.000 claims description 4
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims description 4
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 claims description 4
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims description 4
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims description 4
- 108010006025 bovine growth hormone Proteins 0.000 claims description 4
- 201000004012 propionic acidemia Diseases 0.000 claims description 4
- XUIIKFGFIJCVMT-UHFFFAOYSA-N thyroxine-binding globulin Natural products IC1=CC(CC([NH3+])C([O-])=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-UHFFFAOYSA-N 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 3
- 101710095339 Apolipoprotein E Proteins 0.000 claims description 3
- 102100029470 Apolipoprotein E Human genes 0.000 claims description 3
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 2
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 2
- 241000283973 Oryctolagus cuniculus Species 0.000 claims description 2
- 101150089617 PCCA gene Proteins 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 101150036991 pccB gene Proteins 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 5
- 102000013918 Apolipoproteins E Human genes 0.000 claims 1
- 108010025628 Apolipoproteins E Proteins 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063002541P | 2020-03-31 | 2020-03-31 | |
| US63/002,541 | 2020-03-31 | ||
| PCT/US2021/024892 WO2021202532A1 (fr) | 2020-03-31 | 2021-03-30 | Thérapie génique pour le traitement de l'acidémie propionique |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023520402A JP2023520402A (ja) | 2023-05-17 |
| JPWO2021202532A5 true JPWO2021202532A5 (fr) | 2024-04-10 |
Family
ID=75660311
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022559589A Pending JP2023520402A (ja) | 2020-03-31 | 2021-03-30 | プロピオン酸血症を処置するための遺伝子治療 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230129893A1 (fr) |
| EP (1) | EP4127189A1 (fr) |
| JP (1) | JP2023520402A (fr) |
| AR (1) | AR121709A1 (fr) |
| TW (1) | TW202204624A (fr) |
| WO (1) | WO2021202532A1 (fr) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023130003A2 (fr) * | 2021-12-29 | 2023-07-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Constructions de thérapie génique améliorées pour le traitement de l'acidémie propionique provoquée par des mutations dans la propionyl-coa carboxylase alpha |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG157224A1 (en) | 2001-11-13 | 2009-12-29 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP1453547B1 (fr) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Sequences du serotype 8 du virus associe a l'adenovirus (aav), vecteurs les contenant et utilisations correspondantes |
| SI2357189T1 (sl) | 2003-06-19 | 2017-07-31 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in njihova uporaba |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| DK2359867T3 (en) | 2005-04-07 | 2015-01-05 | Univ Pennsylvania | A method for increasing an AAV vector function |
| DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US10392632B2 (en) | 2011-02-14 | 2019-08-27 | The Children's Hospital Of Philadelphia | AAV8 vector with enhanced functional activity and methods of use thereof |
| EP3693025B1 (fr) | 2011-04-22 | 2021-10-13 | The Regents of The University of California | Virions de virus adéno-associé avec capside variant et procédés d'utilisation de ceux-ci |
| MY172457A (en) | 2012-04-18 | 2019-11-26 | Childrens Hospital Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US10266845B2 (en) | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| EP3044318B1 (fr) | 2013-09-13 | 2019-05-01 | California Institute of Technology | Récupération sélective |
| WO2016049230A1 (fr) | 2014-09-24 | 2016-03-31 | City Of Hope | Variants de vecteur de virus adénoassocié pour une édition de haute efficacité du génome et procédés correspondants |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| EP3384034B1 (fr) | 2015-12-02 | 2020-07-08 | The Board of Trustees of the Leland Stanford Junior University | Nouveaux capsides de virus adéno-associés recombinants avec du tropisme pour les muscles sqeletiques humains amélioré |
| CN109415704B (zh) | 2016-02-16 | 2022-02-25 | 利兰斯坦福初级大学董事会 | 对先已存在的人中和抗体有抗性的新型重组腺相关病毒衣壳 |
| WO2017165859A1 (fr) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Protéines de capside virale modifiées |
| IL298604A (en) | 2016-04-15 | 2023-01-01 | Univ Pennsylvania | Novel aav8 mutant capsids and preparations containing them |
| KR20230039779A (ko) | 2016-07-29 | 2023-03-21 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법 |
| CA3054131C (fr) | 2017-02-21 | 2021-05-25 | University Of Florida Research Foundation, Incorporated | Proteines des capsides aav modifiees et leurs utilisations |
| WO2018222503A1 (fr) | 2017-05-31 | 2018-12-06 | The Regents Of The University Of California | Virus adéno-associé à capside variante et ses méthodes d'utilisation |
| JP7196104B2 (ja) | 2017-06-05 | 2022-12-26 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 増強された改変ウイルスカプシドタンパク質 |
| CA3091806A1 (fr) | 2018-02-27 | 2019-09-06 | The Trustees Of The University Of Pennsylvania | Nouveaux vecteurs de virus adeno-associes (vaa), vecteurs de vaa presentant une desamidation de capside reduite et utilisations associees |
| BR112021006052A2 (pt) | 2018-10-01 | 2021-09-08 | Ultragenyx Pharmaceutical Inc. | Terapia gênica para tratar acidemia propiônica |
-
2021
- 2021-03-30 US US17/906,902 patent/US20230129893A1/en active Pending
- 2021-03-30 WO PCT/US2021/024892 patent/WO2021202532A1/fr unknown
- 2021-03-30 EP EP21721256.2A patent/EP4127189A1/fr active Pending
- 2021-03-30 JP JP2022559589A patent/JP2023520402A/ja active Pending
- 2021-03-31 TW TW110111870A patent/TW202204624A/zh unknown
- 2021-03-31 AR ARP210100811A patent/AR121709A1/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EP3313991B1 (fr) | Facteur ix modifié, et compositions, méthodes et utilisations pour un transfert de gènes dans des cellules, des organes et des tissus | |
| FI3906066T3 (fi) | Geeniterapiakonstrukteja wilsonin taudin hoitamiseksi | |
| JP2021087431A5 (fr) | ||
| JP2020537544A5 (fr) | ||
| US20040142416A1 (en) | Treatment for phenylketonuria | |
| RU2015144234A (ru) | Композиции и способы лечения mps1 | |
| JP2023088902A5 (fr) | ||
| RU2018145010A (ru) | Оптимизированные гены и экспрессионные кассеты cln1, и их применение | |
| WO2020142714A1 (fr) | Cassette d'expression d'aav et vecteurs aav la comprenant | |
| CA3158518A1 (fr) | Vecteurs de virus adeno-associes a triple fonction (aav) pour le traitement de maladies associees a c9orf72 | |
| JP2021533799A (ja) | 第ｉｘ因子をコードするヌクレオチド | |
| JPWO2020142653A5 (fr) | ||
| CA3178591A1 (fr) | Agents immunosuppresseurs et methodes de re-dosage d'administration virale pour therapie genique | |
| JPWO2021202532A5 (fr) | ||
| KR20210153069A (ko) | 섬유아세포 성장 인자 23 관련 저인산혈증성 질환의 유전자 요법 | |
| JPWO2020072451A5 (fr) | ||
| CA3209673A1 (fr) | Composition et methodes de traitement de la maladie de fabry | |
| JPWO2021163322A5 (fr) | ||
| JP2023543125A (ja) | Ｇｌｐ－１受容体アゴニスト融合物をコードするウイルスベクター及び代謝性疾患の治療におけるその使用 | |
| JPWO2019165050A5 (fr) | ||
| JPWO2020041773A5 (fr) | ||
| JPWO2020010042A5 (fr) | ||
| RU2021123108A (ru) | Генотерапевтические конструкции для лечения болезни вильсона | |
| JPWO2020172490A5 (fr) | ||
| JPWO2020237130A5 (fr) |