Title:
MUSCLE-TARGETED MINICIRCLE DNA GENE THERAPY
Document Type and Number:
WIPO Patent Application WO/2021/042944
Kind Code:
A1
Abstract:
Provided is muscle-targeted minicircle DNA gene therapy. In particular, a minicircle DNA vector for long-term efficient expression of a therapeutic gene product in muscle is provided. The minicircle DNA vector is combined with a muscle-targeted delivery technology, and can efficiently express the therapeutic gene product in muscle for a long time, thereby overcoming the defects of a limited half-life period of protein drugs and an unsustainable therapeutic effect. The minicircle DNA vector can be used for treating common hereditary genetic defects or chronic diseases, and has the advantages of safety, effectiveness and low cost.
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Inventors:
CHEN PING (CN)
XIE YIWU (CN)
CHEN ZHIYING (CN)
XIE YIWU (CN)
CHEN ZHIYING (CN)
Application Number:
PCT/CN2020/108361
Publication Date:
March 11, 2021
Filing Date:
August 11, 2020
Export Citation:
Assignee:
SYNO MINICIRCLE BIOTECHNOLOGY CO LTD (CN)
International Classes:
C12N15/85; A61K38/37; A61K38/48; A61K48/00; A61P7/04; A61P43/00; C12N15/12; C12N15/57; C12N15/62
Domestic Patent References:
| WO2019032898A1 | 2019-02-14 |
Foreign References:
| CN110684798A | 2020-01-14 | |||
| EP2881463A1 | 2015-06-10 | |||
| EP2881463A1 | 2015-06-10 | |||
| CN104342453A | 2015-02-11 |
Other References:
P. QUADE-LYSSY, P. MILANOV, D. ABRISS, C. UNGERER, C. KÖNIGS, E. SEIFRIED, J. SCHÜTTRUMPF: "Oral gene therapy for hemophilia B using chitosan-formulated FIX mutants", JOURNAL OF THROMBOSIS AND HAEMOSTASIS, BLACKWELL PUB., C2003-, vol. 12, no. 6, 10 June 2014 (2014-06-10), pages 932 - 942, XP055132778, ISSN: 15387933, DOI: 10.1111/jth.12572
SAMELSON-JONES BENJAMIN J., ARRUDA VALDER R.: "Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy", MOLECULAR THERAPY- METHODS & CLINICAL DEVELOPMENT, NATURE PUBLISHING GROUP, GB, vol. 12, 1 March 2019 (2019-03-01), GB, pages 184 - 201, XP055788971, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2018.12.007
HOUGH C., LILLICRAP D.: "Gene therapy for hemophilia: an imperative to succeed : Gene therapy for hemophilia", JOURNAL OF THROMBOSIS AND HAEMOSTASIS, BLACKWELL PUBLISHING, OXFORD, GB, vol. 3, no. 6, 1 June 2005 (2005-06-01), GB, pages 1195 - 1205, XP055788976, ISSN: 1538-7933, DOI: 10.1111/j.1538-7836.2005.01401.x
G. N. NGUYEN, L. A. GEORGE, J. I. SINER, R. J. DAVIDSON, C. B. ZANDER, X. L. ZHENG, V. R. ARRUDA, R. M. CAMIRE, D. E. SABATINO: "Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A", JOURNAL OF THROMBOSIS AND HAEMOSTASIS, BLACKWELL PUBLISHING, OXFORD, GB, vol. 15, no. 1, 1 January 2017 (2017-01-01), GB, pages 110 - 121, XP055399640, ISSN: 1538-7933, DOI: 10.1111/jth.13543
SAMELSON-JONES BENJAMIN J., ARRUDA VALDER R.: "Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy", MOLECULAR THERAPY- METHODS & CLINICAL DEVELOPMENT, NATURE PUBLISHING GROUP, GB, vol. 12, 1 March 2019 (2019-03-01), GB, pages 184 - 201, XP055788971, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2018.12.007
HOUGH C., LILLICRAP D.: "Gene therapy for hemophilia: an imperative to succeed : Gene therapy for hemophilia", JOURNAL OF THROMBOSIS AND HAEMOSTASIS, BLACKWELL PUBLISHING, OXFORD, GB, vol. 3, no. 6, 1 June 2005 (2005-06-01), GB, pages 1195 - 1205, XP055788976, ISSN: 1538-7933, DOI: 10.1111/j.1538-7836.2005.01401.x
G. N. NGUYEN, L. A. GEORGE, J. I. SINER, R. J. DAVIDSON, C. B. ZANDER, X. L. ZHENG, V. R. ARRUDA, R. M. CAMIRE, D. E. SABATINO: "Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A", JOURNAL OF THROMBOSIS AND HAEMOSTASIS, BLACKWELL PUBLISHING, OXFORD, GB, vol. 15, no. 1, 1 January 2017 (2017-01-01), GB, pages 110 - 121, XP055399640, ISSN: 1538-7933, DOI: 10.1111/jth.13543
Attorney, Agent or Firm:
BEIJING CHENGHUI LAW FIRM (CN)
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