ProQR shares spike on positive PoC study for lead CF drug
“Patients with CF feel and do better when the CFTR protein channel works more normally,” said Noreen R. Henig, chief development officer of ProQR, in a statement. “Our important first step in helping patients with CF was to demonstrate that QR-010 could restore CFTR function in patients with CF due to ∆F508, the most common mutation. Our proof-of-concept NPD study did exactly that in CF patients homozygous for ∆F508; it demonstrated that CFTR protein channels are active in this cohort following administration of QR-010 as measured by the total chloride response. Having achieved this major step, we have increased confidence in QR-010’s potential to make a meaningful clinical impact for patients and will move forward with an aggressive development plan.”
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