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Hologene 17

Development stage: Unknown
Lead developer: Holostem
Modality: Cell Therapies, Gene Therapies
Administration: Surgical Implantation

Overview

Hologene 17 is an autologous cultured epidermal autograft that incorporates epidermal stem cells from the patient, which are genetically modified ex vivo using a gamma-retroviral vector carrying the COL17A1 complementary DNA (cDNA)[4]. These modified cells are expanded and prepared as sheets suitable for surgical transplantation, with the specific aim of restoring epidermal function in patients with junctional epidermolysis bullosa (JEB) caused by COL17A1 mutations[4]. The product represents a form of combined cell and gene therapy, classified as an Advanced Therapy Medicinal Product (ATMP). Its ultimate goal is to enable permanent regeneration of a healthy, functional, and renewing epidermis in JEB patients with COL17A1 gene defects[4].

Brand names: Hologene 17Hologene17Hologene-17
Other names: Hologene 17Hologene17Hologene-17

Targets

COL17A1

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