AU2018253959B2 - Gene therapy - Google Patents

Gene therapy Download PDF

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Publication number
AU2018253959B2
AU2018253959B2 AU2018253959A AU2018253959A AU2018253959B2 AU 2018253959 B2 AU2018253959 B2 AU 2018253959B2 AU 2018253959 A AU2018253959 A AU 2018253959A AU 2018253959 A AU2018253959 A AU 2018253959A AU 2018253959 B2 AU2018253959 B2 AU 2018253959B2
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Prior art keywords
ifn
ctrl
cells
cell
injection
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AU2018253959A
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AU2018253959A1 (en
Inventor
Giulia ESCOBAR
Bernhard Rudolf Gentner
Adele MUCCI
Luigi Naldini
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Telethon Foundation
IRCCS Ospedale San Raffaele
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Telethon Foundation
IRCCS Ospedale San Raffaele
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Priority claimed from GBGB1706410.6A external-priority patent/GB201706410D0/en
Priority claimed from GBGB1801511.5A external-priority patent/GB201801511D0/en
Application filed by Telethon Foundation, IRCCS Ospedale San Raffaele filed Critical Telethon Foundation
Publication of AU2018253959A1 publication Critical patent/AU2018253959A1/en
Assigned to OSPEDALE SAN RAFFAELE S.R.L., FONDAZIONE TELETHON ETS reassignment OSPEDALE SAN RAFFAELE S.R.L. Amend patent request/document other than specification (104) Assignors: FONDAZIONE TELETHON, OSPEDALE SAN RAFFAELE S.R.L.
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/403—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with carbocyclic rings, e.g. carbazole
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    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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    • C07K—PEPTIDES
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AU2018253959A 2017-04-21 2018-04-20 Gene therapy Active AU2018253959B2 (en)

Applications Claiming Priority (5)

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Families Citing this family (16)

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Publication number Priority date Publication date Assignee Title
CA3128871A1 (en) * 2019-02-09 2020-08-13 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Genetically modified hematopoietic stem and progenitor cells (hspcs) and mesenchymal cells as a platform to reduce or prevent metastasis, treat autoimmune and inflammatory disorders, and rebalance the immune milieu_and dysregulated niches
US20220105143A1 (en) * 2019-02-22 2022-04-07 Oncolytics Biotech Inc. T cell repertoire dynamics and oncolytic viral therapy
EP3941490A4 (en) * 2019-03-20 2023-01-04 2seventy bio, Inc. ADOPTIVE CELLULAR THERAPY
CN112280829B (zh) * 2020-04-07 2023-12-26 南方科技大学 一种试剂盒、样本标记方法、单细胞测序方法
CA3176697A1 (en) * 2020-05-11 2021-11-18 Doughlas J. JOLLY Vectors and methods for in vivo transduction
GB202007169D0 (en) * 2020-05-14 2020-07-01 Ospedale San Raffaele Srl Epidermal growth factor receptor
EP4161536A4 (en) * 2020-06-04 2024-08-14 Carisma Therapeutics Inc. Novel constructs for chimeric antigen receptors
CN114426953A (zh) * 2020-10-29 2022-05-03 未来智人再生医学研究院(广州)有限公司 一种表达il-12的多能干细胞衍生物及应用
AU2021374598A1 (en) * 2020-11-03 2023-06-22 Edigene (Guangzhou) Inc. Cell preparation, use of protein in characterizing hematopoietic stem cells, and method for determining hematopoietic stem cells
CN114807155A (zh) * 2021-01-18 2022-07-29 华东师范大学 用于基因编辑的组合物及其应用
CN115819612A (zh) * 2022-03-11 2023-03-21 郑州大学第一附属医院 表达载体、宿主细胞、抗肿瘤药物组合物及其应用
EP4593868A1 (en) * 2022-09-26 2025-08-06 Institut Curie Myeloid cells expressing il-2 and uses thereof for quick anticancer therapy
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WO2024194223A2 (en) * 2023-03-17 2024-09-26 Ospedale San Raffaele S.R.L. Gene therapy
WO2025217427A1 (en) * 2024-04-11 2025-10-16 Genenta Science S.P.A. Combination immunotherapy of solid cancers
WO2026050378A1 (en) * 2024-08-28 2026-03-05 Cedars-Sinai Medical Center Humanized genetically-engineered autochthonous brain tumors compatible with use in immunocompetent rodents

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
US8389282B2 (en) * 2007-03-30 2013-03-05 Memorial Sloan-Kettering Cancer Center Constitutive expression of costimulatory ligands on adoptively transferred T lymphocytes
MX2011011508A (es) * 2009-04-30 2012-02-13 San Raffaele Centro Fond Vector genico.
EP3434774A1 (en) * 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP3347375B1 (en) * 2015-09-10 2020-12-23 The United States of America, as represented by The Secretary, Department of Health and Human Services Anti-cd276 chimeric antigen receptors
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ESCOBAR G ET AL: "Genetic Engineering of Hematopoiesis for Targeted IFN-alpha Delivery Inhibits Breast Cancer Progression", SCIENCE TRANSLATIONAL MEDICINE, vol. 6, no. 217, 217RA3, 1 January 2014 (2014-01-01) *

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