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Methods for Treating Osteogenesis Imperfecta

A technology for osteogenesis imperfecta and uses, which can be used in antibody medical components, chemical instruments and methods, pharmaceutical formulations, etc., and can solve problems such as reduced life expectancy

Pending Publication Date: 2020-04-14
GENZYME CORP +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Due to these symptoms and the propensity for fatal fractures and complications, the life expectancy of OI patients is reduced compared to the general population

Method used

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  • Methods for Treating Osteogenesis Imperfecta
  • Methods for Treating Osteogenesis Imperfecta
  • Methods for Treating Osteogenesis Imperfecta

Examples

Experimental program
Comparison scheme
Effect test

Embodiment approach

[0114] Several methods can be used to measure and characterize bone structure, density and quality, including histology and histomorphometry, atomic force microscopy, confocal Raman microscopy, nanoindentation, three-point bending test, X-ray Imaging and micro-computed tomography (μ-CT). In an exemplary embodiment, bone is measured and characterized by at least one of these methods.

[0115] The term "bone volume density" refers to the fraction of a given volume of bone (total volume or TV) that contains calcified material (bone volume or BV). Therefore, bone volume density was calculated as BV / TV and reported as a percentage. The term "specific bone surface area" refers to the total bone surface area (BS) per given bone volume. Therefore, the specific bone surface area was calculated as BS / TV. Other common bone measurements include: bone area (B.Ar), trabecular number (Tb.N); trabecular space (Tb.Sp); N.Oc (number of osteoclasts); Oc.S (osteoclast surface area ); Oc.S / BS;...

Embodiment 1

[0233] Example 1: Crtap - / - Altered TGFβ signaling in the calvaria

[0234] Analyze Crtap - / - Activation of pSmad2, a member of the TGFβ signaling pathway, and expression of other downstream targets of TGFβ in mice and age-matched littermate wild-type (WT) controls. The calvaria was excised and RNA and protein were extracted and analyzed by real-time-PCR and Western blot, respectively. as seen in figure 1 A and 1B, Crtap compared with wild-type mice - / - Mice had a 100% high ratio of pSmad2 to total Smad2, as determined by Western blot and quantified by densitometry, indicating that TGFβ signaling in Crtap - / - was elevated in mice. Transcriptional targets of TGFβ, such as Col1a1 and p21, are elevated compared to WT controls, as measured by RT-PCR and described respectively in figure 1 C and figure 1 d. measured the fibrillar ECM protein connective tissue growth factor (CTGF) and found that compared to WT controls, in Crtap - / - Approximately 50% higher in mice as determ...

Embodiment 2

[0235] Example 2: In Crtap - / - mice in vivo and in crtap - / - Increased TGFb activity in osteoblasts

[0236] Crtap - / - Mice were crossed with TGF[beta] reporter mice (Jackson Laboratory; B6.Cg-Tg(SBE / TK-luc)7Twc / J) expressing luciferase in response to activation of TGF[beta] signaling. P9 mice were injected 10 minutes before imaging with the substrate D-luciferin (150 mg / kg). Such as figure 2 As shown in A, compared with WT control, Crtap - / - Mice have much higher fluorescence in their tail, long bones and calvaria, indicating that crtap - / - Increased TGFb activity in mice. figure 2 B, Quantification of luciferase activity at the calvaria.

[0237] Bone marrow stromal cells (BMSC) were isolated from Crtap - / - Mice and WT mice, cultured ex vivo under osteogenic conditions, and conditioned medium were analyzed for TGFβ activity using a cell line expressing luciferase in response to activation of TGFβ signaling. Such as figure 2 Shown in C, compared with BMSCs from W...

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Abstract

The present invention provides methods for treating and improving the symptoms of osteogenesis imperfecta (OI) in a subject by administering to the subject a therapeutically effective amount of a binding agent that binds to transforming growth factor beta (TGFbeta).

Description

[0001] The application of the present invention is based on the application date of March 20, 2014, the application number is 201480028990.8 (the international application number is PCT / US2014 / 031279), and the title is the divisional case of the invention patent application named "method for treating osteogenesis imperfecta" Apply. [0002] related application [0003] This application is related to U.S. Provisional Patent Application No. 61 / 803,647, filed March 20, 2013, U.S. Provisional Patent Application No. 61 / 875,399, filed September 9, 2013, and U.S. Provisional Patent Application No. 61 / 875,399, filed October 26, 2013 Patent Application No. 61 / 883,151, the respective contents of which are hereby incorporated by reference in their entirety. technical field [0004] The present invention relates to methods for treating osteogenesis imperfecta (OI). More specifically, the present invention relates to methods of treating OI using a binding protein (such as an antibody...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K39/395A61K31/663A61K38/29A61K38/23A61K45/06A61P19/08C07K16/22
CPCA61K33/42A61K39/39533A61K39/3955A61K45/06C07K16/22A61K2039/505C07K2317/51C07K2317/52C07K2317/515C07K2317/565C07K2317/56C07K2317/76C07K2317/21A61P19/00A61P11/00A61P13/12A61P19/08A61P27/16A61P43/00
Inventor B·李K·T·桑帕思
Owner GENZYME CORP
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