Title:
TREATMENT OF ENPP1 DEFICIENCY AND ABCC6 DEFICIENCY
Document Type and Number:
WIPO Patent Application WO/2023/196820
Kind Code:
A3
Abstract:
The present disclosure provides, among other things, specific doses of an ENPP1 agent for in vivo treatment of an ENPP1 deficiency, such as for treatment of Generalized Arterial Calcification of Infancy (GACI), Autosomal Recessive Hypophosphatemic Rickets 2 (ARHR2), and other diseases resulting from pathological calcification, ENPP1 deficiency, ABCC6 deficiency such as diseases or disorders involving ectopic calcification of soft tissue in a subject.
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Inventors:
HUERTAS PEDRO (US)
WENKERT DEBORAH (US)
WENKERT DEBORAH (US)
Application Number:
PCT/US2023/065340
Publication Date:
November 16, 2023
Filing Date:
April 04, 2023
Export Citation:
Assignee:
INOZYME PHARMA INC (US)
International Classes:
C12N9/16; A61K38/17; A61K38/46; A61P9/00; A61P13/12; A61P19/04
Domestic Patent References:
WO2021243054A1 | 2021-12-02 |
Foreign References:
US20220072127A1 | 2022-03-10 | |||
US20210187067A1 | 2021-06-24 |
Other References:
NITSCHKE YVONNE, RUTSCH FRANK: "Generalized arterial calcification of infancy and pseudoxanthoma elasticum: two sides of the same coin", FRONTIERS IN GENETICS, FRONTIERS RESEARCH FOUNDATION, SWITZERLAND, vol. 3, Switzerland , XP093112984, ISSN: 1664-8021, DOI: 10.3389/fgene.2012.00302
ANONYMOUS: "Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE", CLINICALTRIALS.GOV; NCT05030831, 1 September 2021 (2021-09-01), XP093112986, Retrieved from the Internet [retrieved on 20231218]
ANONYMOUS: "Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE", CLINICALTRIALS.GOV; NCT05030831, 1 September 2021 (2021-09-01), XP093112986, Retrieved from the Internet
Attorney, Agent or Firm:
WILLIAMS, Kathleen, M. et al. (US)
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