Rare Diseases, Startups

Dutch rare disease biotech ProQR carves out new company dedicated to epidermolysis bullosa

Nine months after kickstarting human testing for its exon skipping drug for a rare skin disorder called dystrophic epidermolysis bullosa, Dutch biotech ProQR Therapeutics is spinning off the program into a new company — Wings Therapeutics — formed and financed by a nonprofit founded by a group of parents, including Pearl Jam’s Eddie Vedder.

Mark de Souza

The ongoing Phase I/II study is testing the drug — QR-313 — in patients with recessive dystrophic epidermolysis bullosa (DEB), a severe form of an inherited skin blistering disease that is characterized by fragile skin, chronic pain and a high risk of infection and malnutrition.

There are no treatments approved to target the underlying cause of DEB.

Infants with EB are born with skin so fragile they are often called ‘butterfly children’ – with skin is as vulnerable as the wing of a butterfly. Some type of EB occurs in an estimated 1 out of every 50,000 live births, according to the National Organization for Rare Disorders.

Mutations in exon 73 of the COL7A1 gene can cause the deprivation of a functional collagen protein, which culminates in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin. QR-313, which has won orphan drug status in the US and Europe, is designed to target this underlying cause of DEB by excluding exon 73 to restore the functionality of the anchoring fibrils.

The 8-patient Phase I/II trial is expected to be completed by mid-April.

Wings Therapeutics will shepherd QR-313 through clinical development, as well as additional RNA molecules designed to target other mutations that cause DEB. ProQR will hold a minority stake in the new company and will be eligible for milestone and royalty payments on any commercial products. Financial details of the deal were not disclosed.

Wings Therapeutics will be led by interim CEO Mark de Souza, the former CEO of Lotus Tissue Repair, and Hal Landy, former medical advisor to Lotus Tissue Repair and CMO of Enobia.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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