- 1、本文档共20页,可阅读全部内容。
- 2、有哪些信誉好的足球投注网站(book118)网站文档一经付费(服务费),不意味着购买了该文档的版权,仅供个人/单位学习、研究之用,不得用于商业用途,未经授权,严禁复制、发行、汇编、翻译或者网络传播等,侵权必究。
- 3、本站所有内容均由合作方或网友上传,本站不对文档的完整性、权威性及其观点立场正确性做任何保证或承诺!文档内容仅供研究参考,付费前请自行鉴别。如您付费,意味着您自己接受本站规则且自行承担风险,本站不退款、不进行额外附加服务;查看《如何避免下载的几个坑》。如果您已付费下载过本站文档,您可以点击 这里二次下载。
- 4、如文档侵犯商业秘密、侵犯著作权、侵犯人身权等,请点击“版权申诉”(推荐),也可以打举报电话:400-050-0827(电话支持时间:9:00-18:30)。
查看更多
Systemic Protein Replacement Therapy for
Dystrophic Epidermolysis Bullosa (DEB)
Mark P. de Souza, PhD
EB-CLINET Conference
Salzburg
October 6, 2012
Company Overview
• Founded in 2010 to develop recombinant Collagen Type VII (rC7) as protein
therapy for Dystrophic Epidermolysis Bullosa (DEB)
• Exclusive license to USC and Stanford intellectual property on rC7
• Compelling data in mouse and dog models demonstrates intravenous rC7
reverses the DEB phenotype
• Clear path to regulatory approval - longitudinal severity study in 2012, Phase 1
human clinical trial in 2013 and regulatory approval in DEB in 4-5 years
• Scientific leaders in C7 protein therapy and experienced management team
• Secured significant financing in June 2011
2
Intradermal rC7 Reverses DEB Phenotype
• Drs. Woodley and Chen – pioneers of protein therapy for DEB
• Recombinant collagen type VII (rC7) purified from a human fibroblast cell line using methods
described for a HEK 293 cell line1
• Intradermal rC7 injected into two mouse models of DEB:
• DEB skin equivalents transplanted onto athymic nude mice2
• C7 knock out mice3
• Intradermal injections restored C7 and anchoring fibrils at the basement membrane zone and
reversed RDEB disease phenotype in both mouse models
Athymic Nude Mouse Model C7 Knock Out Model
Untreated rC7 Treated Untreated rC7 Treated
Reverses separation of dermal-epidermal junction (DEJ) Corrects skin blister formation in C7 knock-out mice
of RDEB skin equivalents grafted on nude mice
1. Chen, M., et al., 2002. J. Bio
您可能关注的文档
最近下载
- 工具保养指南.pdf VIP
- 2019-2020中国入境游游客行为与态度研究报告.pptx
- 家禽生产学题库.docx VIP
- DS160美国签证申请表(中文版).docx VIP
- 第六单元 基层治理与社会保障 单元练习 高二历史统编版2019选择性必修1+国家制度与社会治理.docx VIP
- 富士达电梯UCMP测试说明.pdf
- 【2023部编高教版】中职历史 基础模块 中国历史 第五课秦汉时期的经济、科技与文化 (教案).docx VIP
- 癌痛理论考试测试卷附答案.doc
- 第六单元基层治理与社会保障知识清单高二历史(选择性必修1国家制度与社会治理).docx VIP
- 【2023部编高教版】中职历史 基础模块 中国历史 第十二课 明朝的兴亡 (教案).docx VIP
文档评论(0)