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Topical Beremagene Geperpavec
Beremagene geperpavec is a gene therapy that uses a viral vector to deliver a gene to the skin, which then produces a protein to treat the condition.
Beremagene geperpavec is a gene therapy that uses a viral vector to deliver a gene to the skin, which then produces a protein to treat the condition. Used for Dystrophic epidermolysis bullosa.
At a glance
| Generic name | Topical Beremagene Geperpavec |
|---|---|
| Also known as | HSV1-COL7A1, KB103 |
| Sponsor | Krystal Biotech, Inc. |
| Drug class | Gene therapy |
| Modality | Biologic |
| Therapeutic area | Dermatology |
| Phase | Phase 3 |
Mechanism of action
The gene therapy uses a viral vector to deliver a copy of the human COL7A1 gene to the skin, which then produces the protein collagen VII. This protein is essential for the formation of anchoring fibrils, which hold the epidermis to the dermis. In patients with dystrophic epidermolysis bullosa, the COL7A1 gene is mutated, leading to a deficiency of collagen VII and the characteristic skin blistering.
Approved indications
- Dystrophic epidermolysis bullosa
Common side effects
- Skin blistering
- Skin rash
- Skin redness
Key clinical trials
- A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa (PHASE3)
- Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB (PHASE3)
- A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients (PHASE1, PHASE2)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |