JPWO2022076803A5 - - Google Patents
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- Publication number
- JPWO2022076803A5 JPWO2022076803A5 JP2023521706A JP2023521706A JPWO2022076803A5 JP WO2022076803 A5 JPWO2022076803 A5 JP WO2022076803A5 JP 2023521706 A JP2023521706 A JP 2023521706A JP 2023521706 A JP2023521706 A JP 2023521706A JP WO2022076803 A5 JPWO2022076803 A5 JP WO2022076803A5
- Authority
- JP
- Japan
- Prior art keywords
- expression cassette
- seq
- hgla
- raav
- sequence
- 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
- 101000718525 Homo sapiens Alpha-galactosidase A Proteins 0.000 claims 13
- 102000043404 human GLA Human genes 0.000 claims 12
- 235000001014 amino acid Nutrition 0.000 claims 4
- 150000001413 amino acids Chemical class 0.000 claims 4
- 230000001105 regulatory effect Effects 0.000 claims 4
- 239000013598 vector Substances 0.000 claims 4
- 239000013603 viral vector Substances 0.000 claims 4
- 239000013612 plasmid Substances 0.000 claims 3
- 108091026890 Coding region Proteins 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 2
- 125000000539 amino acid group Chemical group 0.000 claims 2
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 102100026277 Alpha-galactosidase A Human genes 0.000 claims 1
- 229920001661 Chitosan Polymers 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 108091061960 Naked DNA Proteins 0.000 claims 1
- 241000125945 Protoparvovirus Species 0.000 claims 1
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 208000013829 diffuse lymphatic malformation Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000009472 formulation Methods 0.000 claims 1
- 239000010954 inorganic particle Substances 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 108091070501 miRNA Proteins 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063089850P | 2020-10-09 | 2020-10-09 | |
| US63/089,850 | 2020-10-09 | ||
| US202163146286P | 2021-02-05 | 2021-02-05 | |
| US63/146,286 | 2021-02-05 | ||
| US202163186092P | 2021-05-08 | 2021-05-08 | |
| US63/186,092 | 2021-05-08 | ||
| PCT/US2021/054145 WO2022076803A1 (en) | 2020-10-09 | 2021-10-08 | Compositions and methods for treatment of fabry disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023545433A JP2023545433A (ja) | 2023-10-30 |
| JPWO2022076803A5 true JPWO2022076803A5 (enExample) | 2024-10-10 |
Family
ID=81125461
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023521706A Pending JP2023545433A (ja) | 2020-10-09 | 2021-10-08 | ファブリー病の治療のための組成物及び方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230365955A1 (enExample) |
| EP (1) | EP4225906A1 (enExample) |
| JP (1) | JP2023545433A (enExample) |
| KR (1) | KR20230118075A (enExample) |
| AU (1) | AU2021356684A1 (enExample) |
| CA (1) | CA3193833A1 (enExample) |
| TW (1) | TW202229560A (enExample) |
| WO (1) | WO2022076803A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR124981A1 (es) * | 2021-02-26 | 2023-05-24 | Takeda Pharmaceuticals Co | Composición y métodos para el tratamiento de la enfermedad de fabry |
| WO2023102517A1 (en) * | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| WO2024011237A1 (en) * | 2022-07-08 | 2024-01-11 | Cornell University | Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease |
| TW202413648A (zh) * | 2022-08-25 | 2024-04-01 | 日商武田藥品工業股份有限公司 | 用於治療法布立氏病（ｆａｂｒｙ ｄｉｓｅａｓｅ）之組合物及方法 |
| KR20250135916A (ko) | 2022-12-17 | 2025-09-15 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물 |
| 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 (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
| US6136597A (en) | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| US7419829B2 (en) | 2000-10-06 | 2008-09-02 | Oxford Biomedica (Uk) Limited | Vector system |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2010053572A2 (en) | 2008-11-07 | 2010-05-14 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| SI2473095T1 (sl) | 2009-09-03 | 2015-02-27 | Evonik Roehm Gmbh | Oralna dozirna oblika, ki vsebuje vsaj eno biološko aktivno sredstvo, pomožne substance formulacije in delce, ki jih je mogoče namagnetiti |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| PT2717893T (pt) | 2011-06-08 | 2019-08-20 | Translate Bio Inc | Composições de nanopartículas lipídicas e métodos para transferência de arnm |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| SG10201707319UA (en) | 2013-03-15 | 2017-10-30 | Univ Pennsylvania | Compositions and methods for treating mpsi |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| US20170051267A1 (en) * | 2015-08-20 | 2017-02-23 | Research Foundation Of The City University Of New York | Novel alpha-galactosidase a derivatives |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| WO2017136500A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| IL299325B2 (en) | 2016-09-12 | 2025-08-01 | Inst Nat Sante Rech Med | Acid-alpha glucosidase variants and uses thereof |
| SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
| KR102930150B1 (ko) | 2018-06-14 | 2026-02-25 | 리젠엑스바이오 인크. | 재조합 aav 생성을 위한 음이온 교환 크로마토그래피 |
| WO2020077114A2 (en) * | 2018-10-10 | 2020-04-16 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
| PE20211581A1 (es) * | 2018-12-21 | 2021-08-17 | Univ Pennsylvania | Composiciones para la reduccion de la expresion transgenica especifica de drg |
| CN113423419A (zh) * | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
| WO2021158915A1 (en) | 2020-02-06 | 2021-08-12 | Precision Biosciences, Inc. | Recombinant adeno-associated virus compositions and methods for producing and using the same |
| EP3868886A1 (en) | 2020-02-21 | 2021-08-25 | Bia Separations D.O.O. | A method for separation or depletion of empty aav capsids from full aav capsids |
| KR20230026986A (ko) * | 2020-04-27 | 2023-02-27 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 코돈 최적화된 gla 유전자 및 이의 용도 |
-
2021
- 2021-10-08 AU AU2021356684A patent/AU2021356684A1/en not_active Abandoned
- 2021-10-08 JP JP2023521706A patent/JP2023545433A/ja active Pending
- 2021-10-08 EP EP21805710.7A patent/EP4225906A1/en active Pending
- 2021-10-08 WO PCT/US2021/054145 patent/WO2022076803A1/en not_active Ceased
- 2021-10-08 US US18/246,872 patent/US20230365955A1/en active Pending
- 2021-10-08 TW TW110137528A patent/TW202229560A/zh unknown
- 2021-10-08 KR KR1020237015206A patent/KR20230118075A/ko not_active Withdrawn
- 2021-10-08 CA CA3193833A patent/CA3193833A1/en active Pending
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