Jul 20, 2020 / 09:00PM GMT
Aniz Girach - ProQR Therapeutics N.V. - Chief Medical Officer
I want to welcome Dr. Ian MacDonald, who is one of the leading experts in the inherited retinal disease space to be present here with us today. Just as a background, sepofarsen, which is formerly known as QR-110, is being developed for Leber's congenital amaurosis type 10. This is an antisense oligonucleotide and really targets a disease which has no other treatments available for it, and which is a blinding disease for patients. We think that there's probably about 2,000 patients in the Western world currently suffering from this disease, but probably even more because of lack of formal genetic testing.
This drug, sepofarsen, is locally administered into the back of the eye, into the vitreous cavity through an intravitreal procedure, and it's anticipated that it will be twice-a-year dosing, ultimately. We have strong preclinical proof of concept through the retinal organoid models that has helped us in really shaping up some of the preclinical modeling around this.
And indeed, you may be aware that, and I'll summarize this in
ProQR Therapeutics NV Expert Perspectives Series -- LCA10 and Sepofarsen - Call Transcript
Jul 21, 2020
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