Théa acquires ProQR’s RNA ophthalmic assets

RNA

ProQR Therapeutics and Laboratoires Théa have announced an agreement in which ProQR will divest its late-stage ophthalmic assets, sepofarsen and ultevursen, to Théa.

Under the terms of the agreement, ProQR will receive an initial payment of €12.5M and will also be eligible for up to €135M in further development, regulatory, and commercial payments.

“We are very excited to continue the development of sepofarsen and ultevursen for patients,” said Jean-Frédéric Chibret, President of the Théa group. “These two programmes can deliver hope for patients suffering from retinal diseases that lead to blindness.”

Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. It has been granted orphan drug designation in the United States (US) and the European Union (EU) and received fast-track designation and rare paediatric disease designation from the FDA, as well as access to the PRIME scheme by the EMA.

Ultevursen (formerly QR-421a) is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. It has been granted orphan drug designation in the US and the EU and received fast-track and rare paediatric disease designations from the FDA.

Daniel de Boer, Founder and Chief Executive Officer of ProQR, said: “We look forward to continuing to advance our Axiomer RNA editing platform, with an initial focus on targets for cholestatic and cardiovascular diseases, as we seek to develop a new class of therapies for patients with high unmet need.”

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