ProQR to Start Clinical Trial of QR-110 in Leber’s Congenital Amaurosis Type 10 Patients

May 12, 2017: By Jon Swedien

ProQRThe US FDA has given clearance to ProQR Therapeutics’ investigational new drug application for QR-110, a proposed treatment for Leber’s congenital amaurosis Type 10 (LCA 10) patients, the Netherlands company announced April 27.

ProQR said it intends to move forward with a Phase I/II open-label trial to assess the safety, tolerability, pharmacokinetics, and efficacy of multiple administrations of QR-110 in one eye of each patient. ProQR said the trial will include six adults and six children with LCA 10.

Topline results are expected in 2018.

QR-110 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of LCA 10, a mutation in the CEP290 gene. It is intended to be administered through intravitreal injections in the eye. QR-110 has been granted orphan drug designation in the US and European Union, the company said.

LCA 10 leads to progressive loss of vision, causing most patients to lose their sight in the first few years of life. There are no approved treatments or products in clinical development that treat the underlying cause of the disease, ProQR said.

ProQR is focused on RNA medicines for the treatment of severe genetic rare diseases such as LCA 10, cystic fibrosis, and dystrophic epidermolysis bullosa.

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