ProQR IPO is this week - leading product is QR-010 targets del F508 mutation

enniob

Administrator
Staff member
ProQR IPO is this week. Their leading product is QR-010 which aims to restore the defect in CFTR protein caused by the del F508 mutation. Specifically, administration into the lungs of a chemically modified oligoribonucleotide that is capable of restoring the RNA sequence coding for CFTR, thereby restoring CFTR protein function, or at least improving it. Administration would be via a nebulized drug+hypertonic saline solution. Cost is not yet known to me. IPO is for the rollout of phase 1 clinical trials.

For those interesting in reading up on the specifics below are the resources. Please feel free to share your findings and thoughts! I will be studying these over time and the first clinical trial role out.

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- Chris
 
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