ProQR Therapeutics recently received orphan drug designation for their Cystic Fibrosis therapy that targets the F508del mutation. ProQR are targeting the Cystic Fibrosis defect at the RNA level

This is currently in the pre-clinical stage.

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ProQR are developing an approach that corrects the CF defect at the RNA level.

This means a normal CFTR protein is made, helping the CF symptoms.

Currently in the pre-clinical trial stage, future trials planned.

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