A new company called Wings Therapeutics has been tasked with continuing the development of QR-313, an investigational RNA therapy for dystrophic epidermolysis bullosa (DEB), following a spin-out from ProQR Therapeutics.
ProQR, which was previously leading QR-313 development, will keep a minority stake in Wings Therapeutics and qualifies for milestone and royalty rights to commercial products, a press release reports.
Wings Therapeutics has assumed control of ProQR’s pipeline dedicated to DEB and will take over the ongoing Phase 1/2 WINGS trial (NCT03605069) testing QR-313 for patients with recessive DEB due to mutations in exon 73 of the COL7A1 gene.
The company also has plans to progress other RNA-based candidates designed to target other mutations that cause DEB.
DEB is caused by mutations in the COL7A1 gene, specifically in a part of it called exon 73. Exons are gene segments that contain the information necessary for cells to make proteins, mediated by a genetic “blueprint” called messenger RNA (mRNA).
Mutations in COL7A1 exon 73 lead to defects in a protein called type VII collagen (C7), which is crucial for the proper bonding of two skin layers, the epidermis and the dermis.
QR-313 is an investigational RNA-based therapy designed to make cells skip the mutated exon 73 in C7 mRNA, and generate a shortened but functional C7 protein.
In doing so, the treatment is expected to restore the anchoring fibrils and strengthen the skin, resulting in better wound healing and blistering prevention.
The WING trial’s primary objectives are to evaluate the safety and tolerability, as well as the proof-of-mechanism, of a gel formulation of QR-313 applied directly over DEB wounds (topical administration).
To prove that QR-313 is working, molecular tests will be done to confirm its ability to exclude the DEB mutation from RNA, that is, to make cells skip exon 73 from COL7A1 mRNA.
Secondary objectives of the trial are to determine the effects of QR-313 in wound healing, skin strength, and the presence of collagen type 7 protein and anchoring fibrils — which connect the dermis and epidermis — in the skin.
The study will also measure the systemic distribution of QR-313 — that is, in the blood — after its topical administration.
Enrollment in the trial started last year and remains open. Up to eight participants with recessive DEB, who are 6 years of age or older, are expected to be recruited at sites in the U.S. and Europe. More information on contacts and locations is available here.
Wings Therapeutics was formed and is funded by EB Research Partnership (EBRP), a nonprofit dedicated to financing research to accelerate the discovery of treatments and cures for EB.
The new company will be led by interim CEO Mark de Souza, PhD, and Hal Landy, MD, both of whom formerly worked at Lotus Tissue Repair.
“We are very pleased with this strategic transaction. In partnership with Mark and Hal, we are able to focus Wings Therapeutics solely on exon skipping therapy for treating EB,” said Alex Silver, chairman of EBRP. “We are grateful for ProQR’s work to develop exon skipping drugs for DEB, their continued partnership to advance QR-313 through clinical trials, and we look forward to continuing on this path.”