Drug intelligence / Profile preview

Hologene 5

Development stage
Discontinued
Lead developer
Holostem
Modality
Cell Therapies, Gene Therapies
Administration
Topical
01

Overview

**Hologene 5** is an autologous, ex vivo combined cell and gene therapy developed by Holostem for generalized intermediate junctional epidermolysis bullosa caused by pathogenic **LAMB3** variants. The product is a sterile 144 cm² fibrin-supported epidermal graft generated from a patient's clonogenic keratinocytes and epidermal stem cells. Cells are transduced ex vivo with a gamma-retroviral vector encoding full-length human **LAMB3** cDNA, restoring production of laminin subunit beta-3 and functional laminin-332 at the dermal-epidermal junction. The resulting transgenic epidermal sheet is transplanted onto surgically prepared skin lesions to provide durable re-epithelialization and improve mechanical integrity of the skin.

Other names
genetically corrected cultured epidermal autograft
02

Targets

LAMB3 (Laminin subunit beta-3)

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