RNA Therapy

Question

Hello,
There is gene therapy, protein therapy and now RNA Therapy.
What is it about ?
And above all, would positive results in RNA therapy address all patients (such as gene therapy) or would it depend on mutations (such as protein therapy)?
Thank you in advance for your answer.

Answer

Hello

It's an "innovative" therapy based on the technique of the transcript (the mRNA which is the messenger of the gene), which intervenes upstream in relation to protein therapy.

The ProQR laboratory develops a molecule, named QR-010, for CF patients with F508del mutation. QR-010 aims to repair the genetic defect (mutation) of the mRNA. This mRNA, once "repaired", will give a normal CFTR protein thus functional.

The QR-10 molecule (administered by inhalation) is the object of 2 CF clinical studies:
• a phase 1b, in patients with 2 mutations F508del (homozygotes)
• an exploratory concept validation phase involving patients with at least 1 copy of the F508del mutation.

In view of the current clinical trials, QR-010, if confirmed as an effective drug for CF patients, will be for patients concerned by the F508del mutation.

You will find on the website of the laboratory information concerning the development of QR-10:
www.proqr.com/qr-010-pour-mucoviscidose/?lang=en


Cordially
Anna Ronayette

20.01.2017