CN115175991A - 工程化肌肉靶向组合物 - Google Patents

工程化肌肉靶向组合物 Download PDF

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Publication number
CN115175991A
CN115175991A CN202080084923.3A CN202080084923A CN115175991A CN 115175991 A CN115175991 A CN 115175991A CN 202080084923 A CN202080084923 A CN 202080084923A CN 115175991 A CN115175991 A CN 115175991A
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China
Prior art keywords
aav
vector
disease
viral
polypeptide
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Pending
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CN202080084923.3A
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English (en)
Chinese (zh)
Inventor
P·萨贝蒂
M·塔贝博德巴尔
S·耶
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Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
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Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/6435—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent the peptide or protein in the drug conjugate being a connective tissue peptide, e.g. collagen, fibronectin or gelatin
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C07K2319/00—Fusion polypeptide
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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CN202080084923.3A 2019-10-16 2020-10-16 工程化肌肉靶向组合物 Pending CN115175991A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962916221P 2019-10-16 2019-10-16
US201962916207P 2019-10-16 2019-10-16
US62/916,207 2019-10-16
US62/916,221 2019-10-16
US202063018454P 2020-04-30 2020-04-30
US63/018,454 2020-04-30
US202063055252P 2020-07-22 2020-07-22
US63/055,252 2020-07-22
PCT/US2020/056133 WO2021077000A1 (en) 2019-10-16 2020-10-16 Engineered muscle targeting compositions

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CN115175991A true CN115175991A (zh) 2022-10-11

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CN202080084923.3A Pending CN115175991A (zh) 2019-10-16 2020-10-16 工程化肌肉靶向组合物

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US (4) US20220340929A1 (enExample)
EP (1) EP4045637A4 (enExample)
JP (2) JP2022551986A (enExample)
CN (1) CN115175991A (enExample)
AU (1) AU2020368539A1 (enExample)
CA (1) CA3153902A1 (enExample)
WO (1) WO2021077000A1 (enExample)

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WO2024234803A1 (zh) * 2023-05-15 2024-11-21 杭州新昶基因技术有限公司 腺相关病毒衣壳蛋白、包含其的腺相关病毒及应用
WO2025185625A1 (zh) * 2024-03-04 2025-09-12 苏州新芽基因生物技术有限公司 特异性靶向人组织的多肽序列及其在构建递送载体中的应用

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EP4028064A1 (en) * 2019-09-12 2022-07-20 The Broad Institute, Inc. Engineered adeno-associated virus capsids
US12552838B2 (en) 2020-04-20 2026-02-17 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
AU2021378075B2 (en) 2020-11-11 2025-03-27 Borea Therapeutics S.R.L. Modified viral particles for gene therapy
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
WO2022226375A1 (en) * 2021-04-23 2022-10-27 Locanabio, Inc. Tissue-targeted modified aav capsids and methods of use thereof
CA3216004A1 (en) * 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
JP2024530023A (ja) * 2021-08-05 2024-08-14 ザ・ブロード・インスティテュート・インコーポレイテッド 操作された筋肉標的化組成物
US20250041450A1 (en) * 2021-08-05 2025-02-06 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
EP4392039A4 (en) * 2021-08-24 2025-10-15 Prime Medicine Inc GENOME EDITING COMPOSITIONS AND METHODS FOR TREATING RETINOPATHY
WO2023039476A1 (en) * 2021-09-08 2023-03-16 The Broad Institute, Inc. Engineered muscle and central nervous system compositions
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
AU2022360985A1 (en) * 2021-10-05 2024-05-16 Massachusetts Institute Of Technology Engineered cardiac muscle compositions
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2023111351A1 (en) * 2021-12-17 2023-06-22 Christian Kupatt Aav vectors for gene therapy in endothelial cells
US20250297280A2 (en) * 2022-02-01 2025-09-25 The Broad Institute, Inc. Adeno-associated viral vectors and uses thereof
WO2023183860A1 (en) * 2022-03-23 2023-09-28 University Of Delaware Engineered extracellular vesicles for targeted drug delivery to muscle
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
WO2024184781A1 (en) 2023-03-03 2024-09-12 Universitat Heidelberg Engineered aav capsids
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
EP4680741A2 (en) 2023-03-15 2026-01-21 RegenxBio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025048709A1 (en) * 2023-08-25 2025-03-06 Agency For Science, Technology And Research Composition and methods for identification of novel aav variants for differentiated gene delivery to cardiomyoctes and/or skeletal myofibers
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN116870198B (zh) * 2023-09-06 2023-11-28 中国农业大学 一种调节骨骼肌损伤修复的方法
WO2025054256A1 (en) * 2023-09-06 2025-03-13 Tenaya Therapeutics, Inc. Targeting peptides, engineered aav capsid proteins and virions
WO2025079110A1 (en) * 2023-10-11 2025-04-17 Peptris Technologies Private Limited Atovaquone for the treatment and management of muscular dystrophies
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
CN120000812A (zh) * 2023-12-21 2025-05-16 中山大学中山眼科中心 Crb1基因在治疗眼部和肠道等全身疾病中的用途
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
US20250276095A1 (en) 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025217586A1 (en) * 2024-04-11 2025-10-16 The Children's Medical Center Corporation Compositions and methods for treating selenoprotein-associated diseases
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN121344093A (zh) * 2025-11-26 2026-01-16 中国农业科学院北京畜牧兽医研究所 一种肌酸激酶启动子定点替换华西牛基因启动子的方法

Citations (8)

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