“Leiden, the Netherlands, November 11th 2012, ProQR Therapeutics, the Dutch Cystic Fibrosis company, announced today that the Dutch Ministry of Economic Affairs, Agriculture and Innovation (Agentschap NL) supports ProQR by granting an “Innovation Credit” of €2.5m for pre-clinical and clinical development of its lead CF program.
ProQR Therapeutics is a pre-clinical stage bio-tech company, its proprietary RNA correction platform technology QRNATM allows the development of treatments for genetic diseases. ProQR has its primary focus on Cystic Fibrosis, a currently untreatable lethal disease. The “Innovation Credit” of Agentschap NL consists of an initial incentive of €2.5 million to support the development program including the planned Phase I/II study.”
- CF gene mutations are present in the DNA. This defect is copied into the RNA, which is copied into the protein. The protein cannot function normally, which results in CF symptoms.
- ProQR are developing a molecule that corrects the genetic defect at the RNA level
- This means a normal CFTR protein is made, helping the CF symptoms
- Currently in pre-clinical stage, future trials planned
Sources: Press Release and How ProQR Mechanism corrects the CF defect (video) *These links no longer work
Note: I cannot find details about exactly how the RNA correction works, so I am not sure if they are looking at one specific mutation or CF mutations in general.
and Press Release