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Updated: Jul 23, 2025

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Beremagene Geperpavec: First Approval.

Sohita Dhillon¹

¹Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

|July 11, 2023

Summary

This summary is machine-generated.

Beremagene geperpavec-svdt (VYJUVEK™) is a gene therapy for dystrophic epidermolysis bullosa. It delivers functional COL7A1 genes to restore collagen, receiving US approval for wound treatment.

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Area of Science:

Biotechnology
Gene Therapy
Dermatology

Background:

Dystrophic epidermolysis bullosa (DEB) is a rare genetic disorder characterized by severe skin blistering.
Mutations in the COL7A1 gene impair collagen type VII production, crucial for skin integrity.
Current treatments for DEB are largely supportive, lacking disease-modifying options.

Purpose of the Study:

To summarize the development milestones of beremagene geperpavec-svdt (VYJUVEK™).
To highlight the gene therapy's journey leading to its first regulatory approval.
To provide an overview of its mechanism in treating dystrophic epidermolysis bullosa.

Main Methods:

Development of a replication-defective herpes simplex virus-1 (HSV-1) vector.
Topical application of the gene therapy to deliver functional human COL7A1 genes.
Transduction of keratinocytes and fibroblasts to restore COL7 protein expression.

Main Results:

Beremagene geperpavec-svdt successfully transduces both keratinocytes and fibroblasts.
Restoration of functional COL7 protein has been demonstrated.
Received US FDA approval in May 2023 for treating wounds in DEB patients aged 6 months and older.

Conclusions:

Beremagene geperpavec-svdt represents a significant advancement in DEB treatment.
The gene therapy offers a potential disease-modifying approach for dominant and recessive DEB.
Regulatory submissions in Europe are anticipated, expanding treatment accessibility.