Hologene 5 DS ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
36表皮水疱症1

36. 表皮水疱症


臨床試験数 : 163 薬物数 : 185 - (DrugBank : 46) / 標的遺伝子数 : 50 - 標的パスウェイ数 : 125
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agemin
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agemax
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PhaseCountries
1EUCTR2018-000261-36-IT
(EUCTR)
07/09/202107/10/2020Gene Therapy for patient with Junctional Epidermolysis BullosaMULTICENTRE, OPEN-LABEL, UNCONTROLLED, PIVOTAL CLINICAL TRIAL TO CONFIRM THE EFFICACY AND SAFETY OF AUTOLOGOUS FIBRIN-CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED FOR RESTORATION OF EPIDERMIS IN PATIENTS WITH JUNCTIONAL EPIDERMOLYSIS BULLOSA (HOLOGENE 5) - Hologene 5 Inherited Epidermolysis Bullosa (EB) is a group of rare, devastating genetic disorders characterized by structural and mechanical fragility ofskin and mucosal membranes, impairing the patient's quality of life. Generalized JEB is a chronic, life-threatening condition caused bymutations in genes– encoding different chains of laminin 332. All of these mutations hamper hemidesmosome formation, causing blisters. The most frequent, and perhaps most severe, JEB is due to mutations in LAMB3.
MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Product Name: Hologene 5
Product Code: [Hologene 5 DS]
Other descriptive name: Ex-vivo expanded autologous human keratinocytes suspension containing epidermal stem cells genetically modified with a gamma-retroviral (rv) vector expressing the full-length LAMB3 cDNA.
HOLOSTEM TERAPIE AVANZATE S.R.L.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
6Phase 2;Phase 3France;Germany;Italy