Title:
RECOMBINANT FOLLISTATIN-FC FUSION PROTEINS AND USE IN TREATING DUCHENNE MUSCULAR DYSTROPHY
Document Type and Number:
WIPO Patent Application WO/2017/152090
Kind Code:
A3
Abstract:
The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant follistatin fusion protein such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.
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Inventors:
ISKENDERIAN ANDREA (US)
NORTON ANGELA W (US)
SHEN CHUAN (US)
PAN CLARK (US)
NORTON ANGELA W (US)
SHEN CHUAN (US)
PAN CLARK (US)
Application Number:
PCT/US2017/020723
Publication Date:
October 19, 2017
Filing Date:
March 03, 2017
Export Citation:
Assignee:
SHIRE HUMAN GENETIC THERAPIES (US)
International Classes:
A61K38/17; C07K14/47; A61K38/22; A61P21/00
Domestic Patent References:
| WO2014187807A1 | 2014-11-27 | |||
| WO2014116981A1 | 2014-07-31 | |||
| WO2015187977A1 | 2015-12-10 | |||
| WO2008030367A2 | 2008-03-13 |
Foreign References:
| US20030162714A1 | 2003-08-28 |
Other References:
Y. SIDIS ET AL: "Heparin and Activin-Binding Determinants in Follistatin and FSTL3", ENDOCRINOLOGY, vol. 146, no. 1, 7 October 2004 (2004-10-07) - 1 January 2005 (2005-01-01), pages 130 - 136, XP055073187, ISSN: 0013-7227, DOI: 10.1210/en.2004-1041
S. SUMITOMO ET AL: "The Heparin Binding Site of Follistatin Is Involved in Its Interaction with Activin", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 208, no. 1, 8 March 1995 (1995-03-08), AMSTERDAM, NL, pages 1 - 9, XP055378106, ISSN: 0006-291X, DOI: 10.1006/bbrc.1995.1297
DATTA-MANNAN AMITA ET AL: "An engineered human follistatin variant: insights into the pharmacokinetic and pharmocodynamic relationships of a novel molecule with broad therapeutic potential", JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEU, AMERICAN SOCIETY FOR PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS, US, vol. 344, no. 3, 19 February 2013 (2013-02-19) - 1 March 2013 (2013-03-01), pages 616 - 623, XP009171518, ISSN: 1521-0103, [retrieved on 20121217], DOI: 10.1124/JPET.112.201491
A. DATTA-MANNAN ET AL: "Insights into the Impact of Heterogeneous Glycosylation on the Pharmacokinetic Behavior of Follistatin-Fc-Based Biotherapeutics", DRUG METABOLISM AND DISPOSITION, vol. 43, no. 12, 9 September 2015 (2015-09-09), pages 1882 - 1890, XP055378136, DOI: 10.1124/dmd.115.064519
KEUTMANN H T ET AL: "The Role of Follistatin Domains in Follistatin Biological Action", MOLECULAR ENDOCRINO, THE ENDOCRINE SOCIETY, US, vol. 18, no. 1, 1 January 2004 (2004-01-01), pages 228 - 240, XP009177952, ISSN: 0888-8809, DOI: 10.1210/ME.2003-0112
T. F. LERCH ET AL: "Structural and Biophysical Coupling of Heparin and Activin Binding to Follistatin Isoform Functions", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 282, no. 21, 3 April 2007 (2007-04-03), pages 15930 - 15939, XP055116755, ISSN: 0021-9258, DOI: 10.1074/jbc.M700737200
S. SUMITOMO ET AL: "The Heparin Binding Site of Follistatin Is Involved in Its Interaction with Activin", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 208, no. 1, 8 March 1995 (1995-03-08), AMSTERDAM, NL, pages 1 - 9, XP055378106, ISSN: 0006-291X, DOI: 10.1006/bbrc.1995.1297
DATTA-MANNAN AMITA ET AL: "An engineered human follistatin variant: insights into the pharmacokinetic and pharmocodynamic relationships of a novel molecule with broad therapeutic potential", JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEU, AMERICAN SOCIETY FOR PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS, US, vol. 344, no. 3, 19 February 2013 (2013-02-19) - 1 March 2013 (2013-03-01), pages 616 - 623, XP009171518, ISSN: 1521-0103, [retrieved on 20121217], DOI: 10.1124/JPET.112.201491
A. DATTA-MANNAN ET AL: "Insights into the Impact of Heterogeneous Glycosylation on the Pharmacokinetic Behavior of Follistatin-Fc-Based Biotherapeutics", DRUG METABOLISM AND DISPOSITION, vol. 43, no. 12, 9 September 2015 (2015-09-09), pages 1882 - 1890, XP055378136, DOI: 10.1124/dmd.115.064519
KEUTMANN H T ET AL: "The Role of Follistatin Domains in Follistatin Biological Action", MOLECULAR ENDOCRINO, THE ENDOCRINE SOCIETY, US, vol. 18, no. 1, 1 January 2004 (2004-01-01), pages 228 - 240, XP009177952, ISSN: 0888-8809, DOI: 10.1210/ME.2003-0112
T. F. LERCH ET AL: "Structural and Biophysical Coupling of Heparin and Activin Binding to Follistatin Isoform Functions", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 282, no. 21, 3 April 2007 (2007-04-03), pages 15930 - 15939, XP055116755, ISSN: 0021-9258, DOI: 10.1074/jbc.M700737200
Attorney, Agent or Firm:
CHEN, Fangli et al. (US)
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