JP2023531451A5 - - Google Patents

Info

Publication number
JP2023531451A5
JP2023531451A5 JP2022578663A JP2022578663A JP2023531451A5 JP 2023531451 A5 JP2023531451 A5 JP 2023531451A5 JP 2022578663 A JP2022578663 A JP 2022578663A JP 2022578663 A JP2022578663 A JP 2022578663A JP 2023531451 A5 JP2023531451 A5 JP 2023531451A5
Authority
JP
Japan
Prior art keywords
raav
ligand
cdr
seq
delivered
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
Application number
JP2022578663A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023531451A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/037575 external-priority patent/WO2021257668A1/en
Publication of JP2023531451A publication Critical patent/JP2023531451A/ja
Publication of JP2023531451A5 publication Critical patent/JP2023531451A5/ja
Pending legal-status Critical Current

Links

JP2022578663A 2020-06-17 2021-06-16 遺伝子療法患者の治療のための組成物及び方法 Pending JP2023531451A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063040381P 2020-06-17 2020-06-17
US63/040,381 2020-06-17
US202163135998P 2021-01-11 2021-01-11
US63/135,998 2021-01-11
US202163152085P 2021-02-22 2021-02-22
US63/152,085 2021-02-22
PCT/US2021/037575 WO2021257668A1 (en) 2020-06-17 2021-06-16 Compositions and methods for treatment of gene therapy patients

Publications (2)

Publication Number Publication Date
JP2023531451A JP2023531451A (ja) 2023-07-24
JP2023531451A5 true JP2023531451A5 (cg-RX-API-DMAC7.html) 2024-06-24

Family

ID=77127052

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022578663A Pending JP2023531451A (ja) 2020-06-17 2021-06-16 遺伝子療法患者の治療のための組成物及び方法

Country Status (11)

Country Link
US (1) US20230220069A1 (cg-RX-API-DMAC7.html)
EP (1) EP4171738A1 (cg-RX-API-DMAC7.html)
JP (1) JP2023531451A (cg-RX-API-DMAC7.html)
KR (1) KR20230025000A (cg-RX-API-DMAC7.html)
CN (1) CN115968302A (cg-RX-API-DMAC7.html)
AU (1) AU2021292200A1 (cg-RX-API-DMAC7.html)
CA (1) CA3183153A1 (cg-RX-API-DMAC7.html)
IL (1) IL299167A (cg-RX-API-DMAC7.html)
MX (1) MX2022016528A (cg-RX-API-DMAC7.html)
TW (1) TW202214695A (cg-RX-API-DMAC7.html)
WO (1) WO2021257668A1 (cg-RX-API-DMAC7.html)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112019001806A2 (pt) 2016-07-29 2019-05-07 Momenta Pharmaceuticals, Inc. anticorpos para fcrn e métodos de uso dos mesmos
EA202190264A1 (ru) 2018-07-20 2021-05-20 Момента Фармасьютикалз, Инк. КОМПОЗИЦИИ АНТИТЕЛ ПРОТИВ FcRn
CA3148826A1 (en) * 2019-08-01 2021-02-04 Janssen Biotech, Inc. Fcrn antibodies and methods of use thereof
IL325936A (en) * 2020-01-22 2026-03-01 Spark Therapeutics Inc Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
AU2022262771A1 (en) 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
CA3247109A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers
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
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
JP2026522354A (ja) 2023-06-12 2026-07-07 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症iiibのためのaav遺伝子療法
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
CN117126270B (zh) * 2023-10-25 2024-02-13 首都儿科研究所 一种2型人博卡病毒型别特异性抗体及其应用
KR20260114566A (ko) 2023-11-14 2026-07-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
EP4699615A1 (en) * 2024-08-22 2026-02-25 Universität Zürich Fcrn inhibitor and therapeutic polypeptide
CN120272478B (zh) * 2025-03-28 2025-12-09 山西医科大学 一种抗寨卡病毒的靶点抑制剂、重组基因、表达载体及应用

Family Cites Families (35)

* Cited by examiner, † Cited by third party
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
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US6406861B1 (en) * 1998-10-07 2002-06-18 Cell Genesys, Inc. Methods of enhancing effectiveness of therapeutic viral immunogenic agent administration
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
CN103589692B (zh) 2001-11-13 2018-07-24 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
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
JP2008538919A (ja) * 2005-04-29 2008-11-13 ザ ジャクソン ラボラトリー FcRn抗体およびその使用
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US20070027438A1 (en) 2005-07-26 2007-02-01 Frieder Loesel System and method for compensating a corneal dissection
TW200745163A (en) 2006-02-17 2007-12-16 Syntonix Pharmaceuticals Inc Peptides that block the binding of IgG to FcRn
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20070292922A1 (en) 2006-03-31 2007-12-20 Cell Genesys, Inc. Regulated expression of recombinant proteins from adeno-associated viral vectors
EP2315779A2 (en) 2008-07-09 2011-05-04 Biogen Idec MA Inc. Compositions comprising antibodies to lingo or fragments thereof
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
BR112013030352B1 (pt) 2011-06-02 2020-05-19 Dyax Corp. anticorpo anti-fcrn isolado, composição farmacêutica que compreende o dito anticorpo, ácido nucleico isolado, vetor, célula e uso terapêutico do dito anticorpo
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
EA037448B1 (ru) 2012-06-08 2021-03-30 Этрис Гмбх Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
US9527890B2 (en) 2013-06-18 2016-12-27 The Brigham And Womens's Hospital, Inc. FC receptor (FcRn) binding peptides and uses thereof
EP3074047A2 (en) 2013-11-26 2016-10-05 The Brigham and Women's Hospital, Inc. Receptor-targeted nanoparticles for enhanced transcytosis mediated drug delivery
NZ733918A (en) 2015-02-04 2022-05-27 Marianne Raichle Method for the production of milk with a high content of native vitamin d
MX386491B (es) * 2015-03-09 2025-03-18 argenx BV Metodos para reducir los niveles de suero de agentes que contienen fc que usan antagonistas de fcrn.
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
EP3387138B2 (en) 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017106244A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
BR112020011310A2 (pt) * 2017-12-13 2020-11-17 Momenta Pharmaceuticals, Inc. anticorpos contra fcrn e seus métodos de uso
IL276859B2 (en) 2018-02-27 2025-12-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
EP3768695A4 (en) 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
MX2021007600A (es) 2018-12-21 2021-08-11 Univ Pennsylvania Composiciones para la reduccion de la expresion transgenica especifica de drg.
WO2020223232A1 (en) 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
US20220193261A1 (en) 2019-04-30 2022-06-23 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
CN113769058B (zh) * 2020-06-10 2024-09-06 上海宝济药业股份有限公司 一种药物组合及其应用

Similar Documents

Publication Publication Date Title
JP6091435B2 (ja) アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達
JP7410522B2 (ja) 抗fam19a5抗体のアデノ関連ウイルス(aav)伝達
US20190240328A1 (en) Novel humanized anti-ebola antibodies useful in preventing ebola infections
JP2019501115A (ja) 遺伝子治療のためのアデノ随伴ウイルスベクターのくも膜下腔内投与
EA037980B1 (ru) Рекомбинантный аденоассоциированный вирус
KR20220011664A (ko) 변형된 바이러스 입자 및 이의 용도
JP2009532025A (ja) アデノ関連ウイルスベクターからの組換えタンパク質の制御された発現
US20190002915A1 (en) Compositions and methods for regulatable antibody expression
CA2886161A1 (en) Veterinary composition and methods for non-surgical neutering and castration
US20240148903A1 (en) Delivery of antibody by using dual viral vector system
CN117242183A (zh) 用于治疗与血管生成相关的眼部疾病的组合物和方法
US20230175015A1 (en) Immunosuppressive agents and viral delivery re-dosing methods for gene therapy
US20240092885A1 (en) Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
US10946094B2 (en) Adenoassociated viral mediated persistant anti-VEGF therapy for ovarian cancer
EP4410988A1 (en) An aav2-vector variant for targeted transfer of genes
TW202400796A (zh) 雙向雙重啟動子表現載體及其用途
US20260043046A1 (en) Cassettes of anti-complement component 3 antibody, vectorization and theraputic application
US20240216535A1 (en) Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022072829A1 (en) Compositions for intranasally delivered passive immunization for airborne pathogens
JP2025512121A5 (cg-RX-API-DMAC7.html)
WO2025076091A1 (en) Cd20 antibody aav vectors and methods of use thereof
HK40092158A (en) A gene therapy vector nucleic acid construct for allergic diseases and method of using same
HK40092158B (en) A gene therapy vector nucleic acid construct for allergic diseases and method of using same
WO2024054993A1 (en) Vector constructs for delivery of nucleic acids encoding therapeutic anti-ctla4 antibodies and methods of using the same