EP3820487A4 - Geneditierung für autoimmunerkrankungen - Google Patents

Geneditierung für autoimmunerkrankungen Download PDF

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Publication number
EP3820487A4
EP3820487A4 EP19803321.9A EP19803321A EP3820487A4 EP 3820487 A4 EP3820487 A4 EP 3820487A4 EP 19803321 A EP19803321 A EP 19803321A EP 3820487 A4 EP3820487 A4 EP 3820487A4
Authority
EP
European Patent Office
Prior art keywords
autoimmune disorders
gene editing
editing
gene
autoimmune
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
EP19803321.9A
Other languages
English (en)
French (fr)
Other versions
EP3820487A1 (de
Inventor
Matthew Rabinowitz
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Themba Inc
Original Assignee
Themba Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Themba Inc filed Critical Themba Inc
Publication of EP3820487A1 publication Critical patent/EP3820487A1/de
Publication of EP3820487A4 publication Critical patent/EP3820487A4/de
Pending legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Zoology (AREA)
  • Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
EP19803321.9A 2018-05-14 2019-05-14 Geneditierung für autoimmunerkrankungen Pending EP3820487A4 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862762708P 2018-05-14 2018-05-14
PCT/US2019/032214 WO2019222212A1 (en) 2018-05-14 2019-05-14 Gene editing for autoimmune disorders

Publications (2)

Publication Number Publication Date
EP3820487A1 EP3820487A1 (de) 2021-05-19
EP3820487A4 true EP3820487A4 (de) 2022-03-09

Family

ID=68540991

Family Applications (1)

Application Number Title Priority Date Filing Date
EP19803321.9A Pending EP3820487A4 (de) 2018-05-14 2019-05-14 Geneditierung für autoimmunerkrankungen

Country Status (4)

Country Link
EP (1) EP3820487A4 (de)
JP (1) JP2021523943A (de)
CN (1) CN112384230A (de)
WO (1) WO2019222212A1 (de)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220339192A1 (en) * 2019-09-07 2022-10-27 Themba Inc. Gene editing for viral infections
US20230223102A1 (en) * 2020-03-31 2023-07-13 Themba Inc. Genetic model validation methods
WO2022175674A1 (en) * 2021-02-19 2022-08-25 Oxford University Innovation Limited Modified human cells and uses thereof in the treatment of immune-mediated diseases

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018035388A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018064208A1 (en) * 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016049251A1 (en) * 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2017066561A2 (en) * 2015-10-16 2017-04-20 President And Fellows Of Harvard College Regulatory t cell pd-1 modulation for regulating t cell effector immune responses
MX2018010924A (es) * 2016-03-11 2019-02-13 Bluebird Bio Inc Células efectoras inmunitarias con edición genómica.
US20190264267A1 (en) * 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
MA45996A (fr) * 2016-08-16 2021-06-02 Bluebird Bio Inc Variants d'endonucléase de homing du récepteur alpha de l'il-10, compositions et méthodes d'utilisation associées
EP3500497B1 (de) * 2016-08-16 2020-01-01 Mars, Incorporated Offenwandige packung
BR112019004629A2 (pt) * 2016-09-08 2019-10-01 Bluebird Bio Inc variantes de endonuclease de homing pd-1, composições e métodos de uso

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018035388A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018064208A1 (en) * 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
GLOCKER ERIK-OLIVER ET AL: "IL-10 and IL-10 receptor defects in humans : IL-10/IL-10R deficiency", ANNALS OF THE NEW YORK ACADEMY OF SCIENCES, vol. 1246, no. 1, 1 December 2011 (2011-12-01), US, pages 102 - 107, XP055883124, ISSN: 0077-8923, DOI: 10.1111/j.1749-6632.2011.06339.x *
HUANG HAILIANG ET AL: "Fine-mapping inflammatory bowel disease loci to single-variant resolution", NATURE, vol. 547, no. 7662, 1 July 2017 (2017-07-01), London, pages 173 - 178, XP055882978, ISSN: 0028-0836, Retrieved from the Internet <URL:https://www.nature.com/articles/nature22969.pdf> DOI: 10.1038/nature22969 *
See also references of WO2019222212A1 *
SIVANESAN DURGA ET AL: "IL23R (Interleukin 23 Receptor) Variants Protective against Inflammatory Bowel Diseases (IBD) Display Loss of Function due to Impaired Protein Stability and Intracellular Trafficking", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 291, no. 16, 1 April 2016 (2016-04-01), US, pages 8673 - 8685, XP055883125, ISSN: 0021-9258, DOI: 10.1074/jbc.M116.715870 *

Also Published As

Publication number Publication date
WO2019222212A1 (en) 2019-11-21
CN112384230A (zh) 2021-02-19
JP2021523943A (ja) 2021-09-09
EP3820487A1 (de) 2021-05-19

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Ipc: C12N 5/0781 20100101ALI20220131BHEP

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