Product profile

Cx501 is a cell therapy product for treating epidermolysis bullosa (EB), which is a hereditary disease that affects the skin. Patients with EB suffer from blistering and fragile skin that breaks at the slightest contact, often while carrying out daily living activities.

At present, there are no satisfactory treatments for EB, and sufferers have a high risk of dying at a young age. The leading cause of death is squamous cell carcinoma, although the incidence of death due to kidney failure is also high.

Cx501 represents a new therapeutic technology with potential for treating lesions caused by EB without problems of rejection. The product is currently under evaluation in a multicenter controlled phase II clinical trial.

Cx501 is an extremely novel product that consists of a chimeric skin equivalent containing components obtained from the patients themselves (autologous) and from a donor (allogeneic) comprising two layers (a dermal and epidermal layer). It has the following components:

• Artificial epidermis: Autologous human keratinocytes
• Artificial dermis: 3-dimensional matrix made up of a fiber-rich substrate obtained from pure donor blood plasma (allogeneic) which incorporates healthy human fibroblasts of allogeneic origin.
• These layers of skin are applied over the EB lesions.

Cx501 has been designated an Orphan Drug by the European Medicines Agency.

Advantages of Cx501

Cx501 is a unique product that differs in a number of aspects from other equivalent skin products:

Advantages of the cell component

• The combination of allogeneic cells (healthy fibroblasts) and cells from the patients themselves (keratinocytes) is a completely novel approach that offers a real and lasting therapeutic option, as the graft can perform the functions of healthy skin without rejection.
• The healthy fibroblasts persist after grafting and contribute to dermal healing.

Advantages of the 3-dimensional matrix

• The human plasma reproduces the physiological conditions of the wound healing process in the body.
• A stronger fibroblast and keratinocyte growth is favored, thereby allowing large surfaces of skin to be constructed from a minor biopsy.
• Material of animal origin, which would be potentially allergenic, is not required. This will also make it easier to obtain approval for clinical use and the risk of xenotropic contamination is lower.