Patent landscape of molecular and cellular targeted therapies for recessive dystrophic epidermolysis bullosa

ABSTRACT

Introduction: Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a monogenetic inherited genodermatosis associated with deleterious mutations in the gene encoding type VII collagen (COL7A1). COL7A1 is essential for promoting attachment of the epidermis to the dermis, and its dysfunction may lead to generalized mucosal and cutaneous blistering associated to severe deformities. Currently, management of RDEB patients is limited to supportive care, being aimed at treating and preventing common complications associated with this condition. There is a great demand to develop targeted therapies for this devastating disease and RDEB research advances are currently being translated into clinical trials.

Areas covered: Based on the literature and patent search, the authors have grouped the RDEB targeted therapies into five categories: a) cell-based therapies; b) gene therapy; c) protein replacement therapy; d) molecular therapy based on exon skipping; and e) drug-mediated premature termination codon read-through. The patent searching strategy involved inquiring Google and USPTO patent databases to reveal companies and institutions that are active in the area of RDEB targeted therapies.

Expert opinion: The patent landscape related to targeted therapies for RDEB is quite heterogeneous, with each targeted therapeutic approach being associated with its own challenges in achieving robust patent protection and identifying opportunities for future development.

KEYWORDS:

• Collagen VII (COL7A1)
• Recessive Dystrophic Epidermolysis Bullosa (RDEB)
• cellular and molecular therapeutic approaches
• target-specific therapies
• patent landscape

Article highlights

• Intense efforts are being made at developing targeted therapies for the recessive form of DEB, a genodermatosis to which management remains largely supportive.

• Many aspects of specific targeted therapies for RDEB have been patented over the last 20 years.

• Considering the RDEB patenting landscape, the main trends include molecular approaches, gene therapy, COL7A1 replacement therapy, and cell-based therapies.

• Each therapeutic approach is associated with its own challenges in obtaining robust patent protection and in identifying opportunities for future development.

• Some targeted therapeutic approaches for RDEB have been evaluated by clinical trials with promising results.

• However, there are several bottlenecks that need to be overcome, constituting interesting opportunities for innovation.

This box summarizes key points contained in the article.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.