ProQR Receives Orphan Drug Status for F508del Therapy

ProQR Therapeutics recently received orphan drug designation for their Cystic Fibrosis therapy that targets the F508del mutation. This approach is different to the CFTR modulation approach (potentiators and correctors), which targets the defective protein. ProQR are targeting the Cystic Fibrosis defect at the RNA level.

I have explained this further here:

http://sixtyfiverosesblog.wordpress....08del-therapy/

Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 11, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums