ProQR Therapeutics recently received EMA and FDA orphan drug designation for their therapy targeting Cystic Fibrosis. ProQR Therapeutics are developing an antisense oligonucleotide that targets the F508del Cystic Fibrosis mutation. This is currently in the pre-clinical stage.

This approach is different to the CFTR modulation approach (potentiators and correctors), which targets the defective protein. CF gene mutations are present in the DNA, this defect is copied into the RNA, which is copied into the protein. ProQR are targeting the Cystic Fibrosis defect at the RNA level. This means the normal CFTR protein is made from the RNA, restoring chloride transport.

Sources: EMA COMP September 2013 Report and FDA Orphan Drug Designation


Join the conversation! 4 Comments

  1. Reblogged this on mummymedic and commented:
    More hope on the horizon for those with cystic fibrosis. New drug in development.

  2. Hi Jen,

    Does this work for all mutations or just DF508?


    • Hi Eric, Between this approach and the group working on RNA correction with premature stop codons, its possible multiple types of CF mutations may be helped (small deletions, nonsense mutations, possibly missense mutations).


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1. Class 2 Mutations & F508del, 2. ProQR - RNA Correction