“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.”

Background info:

  • 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

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Join the conversation! 1 Comment

  1. I found that in a registration statement about ProQR (http://www.sec.gov/Archives/edgar/data/1612940/000119312514334355/d749597df1a.htm) :

    The DF508 mutation is a deletion of three coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA sequences on both sides of the DF508 region of the mRNA and guide the insertion of the three missing nucleotides, thus repairing the mRNA and subsequently producing wild-type CFTR protein. This protein is expressed on the cell surface and restores normal CFTR functionality, including chloride transport, chloride-bicarbonate exchange and regulation of the epithelial sodium channel.

    QR-010 is designed to be delivered through a small, handheld aerosol delivery device, or nebulizer, in the form of a mist inhaled into the lungs. We believe this method will allow maximum exposure of QR-010 to the primary target organ, the lung, as well as significant exposure to other affected organs through systemic absorption into the blood.

    (QR-10 is the name of the active molecule here)

    I hope this information will complete yours 🙂
    Your website is absolutely amazing by the way ! Thank you very much for dissecting all the CF study !

    Reply

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2. ProQR - RNA Correction