Beremagene geperpavec

Beremagene geperpavec (also known as B-VEC) is a gene therapy product developed by Krystal Biotech for the treatment of Epidermolysis bullosa, a group of rare genetic skin disorders. It is currently in the late stages of clinical trials.

Overview

Beremagene geperpavec is a non-replicating, non-integrating viral vector that delivers a functional copy of the COL7A1 gene to the cells of the patient. This gene is responsible for the production of type VII collagen, a protein that plays a crucial role in the anchoring of the skin layers. Mutations in the COL7A1 gene lead to a deficiency of type VII collagen, causing the skin layers to separate and form blisters, a characteristic symptom of Epidermolysis bullosa.

Clinical Trials

The safety and efficacy of Beremagene geperpavec are currently being evaluated in a Phase 3 clinical trial (NCT04491604). Preliminary results from the Phase 1/2 clinical trial (NCT03536143) have shown promising results, with patients experiencing significant improvement in wound healing and a reduction in blister formation.

Mechanism of Action

Beremagene geperpavec works by delivering a functional copy of the COL7A1 gene to the patient's cells. This is achieved through the use of a viral vector, which is a virus that has been modified to carry the therapeutic gene. Once inside the cell, the vector releases the gene, which is then incorporated into the cell's own DNA. This allows the cell to produce a functional type VII collagen protein, thereby correcting the underlying genetic defect.

Potential Side Effects

As with any gene therapy, there are potential side effects associated with Beremagene geperpavec. These may include immune reactions to the viral vector, potential off-target effects, and the possibility of insertional mutagenesis. However, these risks are considered to be low and are outweighed by the potential benefits of the therapy.

See Also

• Gene therapy
• Epidermolysis bullosa
• COL7A1 gene
• Krystal Biotech