A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS

开始日期2018-09-06

申办/合作机构-

NCT03605069 / Terminated临床1/2期

A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With DDEB or RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene

A double-blind, randomized, intra-subject placebo-controlled, multicenter, multiple dose study, evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with confirmed DDEB or RDEB diagnosis with one or more pathogenic mutations in exon 73 in the COL7A1 gene.

开始日期2018-07-02

申办/合作机构

Phoenicis Therapeutics, Inc.

100 项与 QR-313 相关的临床结果

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100 项与 QR-313 相关的转化医学

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100 项与 QR-313 相关的专利（医药）

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项与 QR-313 相关的文献（医药）

2021-04-01·Journal of Investigative Dermatology1区 · 医学

QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study

1区 · 医学

Article

作者: Wally, Verena ; Hogervorst, Marieke ; Keene, Douglas R ; Haisma, Elisabeth M ; Hausser, Ingrid ; Nauroy, Pauline ; Nyström, Alexander ; Koller, Ulrich ; Marinkovich, M Peter ; Tufa, Sara F ; Hainzl, Stefan ; Bischof, Johannes ; Swildens, Jim ; Ritsema, Tita ; Platenburg, Gerard ; Murauer, Eva M ; Athanasiou, Ioannis ; Bornert, Olivier ; Kiritsi, Dimitra

Dystrophic epidermolysis bullosa (DEB) is a blistering skin disease caused by mutations in the gene COL7A1 encoding collagen VII. DEB can be inherited as recessive DEB (RDEB) or dominant DEB (DDEB) and is associated with a high wound burden. Perpetual cycles of wounding and healing drive fibrosis in DDEB and RDEB, as well as the formation of a tumor-permissive microenvironment. Prolonging wound-free episodes by improving the quality of wound healing would therefore confer substantial benefit for individuals with DEB. The collagenous domain of collagen VII is encoded by 82 in-frame exons, which makes splice-modulation therapies attractive for DEB. Indeed, antisense oligonucleotide-based exon skipping has shown promise for RDEB. However, the suitability of antisense oligonucleotides for treatment of DDEB remains unexplored. Here, we developed QR-313, a clinically applicable, potent antisense oligonucleotide specifically targeting exon 73. We show the feasibility of topical delivery of QR-313 in a carbomer-composed gel for treatment of wounds to restore collagen VII abundance in human RDEB skin. Our data reveal that QR-313 also shows direct benefit for DDEB caused by exon 73 mutations. Thus, the same topically applied therapeutic could be used to improve the wound healing quality in RDEB and DDEB.

100 项与 QR-313 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
Pasini 型大疱性营养不良表皮松解症	临床2期	西班牙	Phoenicis Therapeutics, Inc.	2018-07-02
Pasini 型大疱性营养不良表皮松解症	临床2期	法国	Phoenicis Therapeutics, Inc.	2018-07-02
Pasini 型大疱性营养不良表皮松解症	临床2期	美国	Phoenicis Therapeutics, Inc.	2018-07-02
营养不良性大疱性表皮松解	临床2期	美国	Wings Therapeutics, Inc.初创企业	2018-03-21
营养不良性大疱性表皮松解	临床2期	法国	Wings Therapeutics, Inc.初创企业	2018-03-21
营养不良性大疱性表皮松解	临床2期	捷克	Wings Therapeutics, Inc.初创企业	2018-03-21
营养不良性大疱性表皮松解	临床2期	英国	Wings Therapeutics, Inc.初创企业	2018-03-21
营养不良性大疱性表皮松解	临床2期	西班牙	Wings Therapeutics, Inc.初创企业	2018-03-21
营养不良性大疱性表皮松解	临床2期	德国	Wings Therapeutics, Inc.初创企业	2018-03-21