ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, announced that it was awarded an innovation credit by the Dutch government for the clinical development and efforts to obtain marketing approval (NDA/MAA) for the QR-110 program that is being developed for patients with Leber’s congenital amaurosis 10 (LCA10), a genetic eye disease that is the leading cause of childhood blindness. Repayment of the credit is triggered only by market approval of QR-110.
Innovation credit (“Innovatiekrediet”)
The Innovation Credit is awarded by the Dutch government through its agency RVO of the Ministry of Economic Affairs, and is aimed at the development of promising innovations. This can include the technical development of a new product or process or the clinical development of a medicine or device. ProQR was awarded an Innovation credit for the QR-110 program. Amounts will be drawn under this facility from 2018 thru 2021. The credit of € 4.7 million through December 31, 2021 will be used to conduct the Phase 2/3 clinical study and efforts to obtain regulatory and ethical market approval (NDA/MAA) of QR-110 for LCA10. The credit, including accrued interest, is repayable depending on obtaining market approval.
QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation by the FDA.