As Chief Science Officer, it’s my personal job to facilitate the ongoing research, to tackle upcoming problems and to make sure we get optimal results. Because it doesn’t stop after granting an application for funds. We keep pushing for the best preventions and cures, so that blind people benefit the most. If we don’t get the most bang for our buck, it’s just a waste of funds.”
What kind of collaborations do you pursue?
“If you talk about research focus, we don’t believe in any particular type of drug. The diseases causing blindness are as diverse as the mutations that cause them, and there is no silver bullet that will take care of it all. We therefore consider gene therapy, gene editing, RNA therapy – they all have potential. We also look at other diseases for inspiration. Take Parkinson’s disease or Alzheimer’s disease for example. These are brain-related, so they are based on neurobiology just like retinal diseases. The eye is the window to the brain and as such, understanding neurobiology has implications for both brain and retinal diseases.
In terms of partners, if it’s companies for the clinical development, we aim for parties that have a proven track record in safety, and that have been able to bring something to the clinic or even the market. When it’s researchers we scrutinize their science. We want the scientists we support to succeed. The group in Nijmegen, at Radboud University, is a good example. They belong to the best groups in the world, and have been an FFB center for years, receiving many investments from us. We actually funded their research that is now being taken to the clinic by ProQR as sepofarsen, which has gotten some very nice results. Still preliminary, but looking very promising. It’s important to get that proof of concept. Now we know that RNA therapies have the potential to do what we believe it will do in treating these diseases.”
How about the developments with QR-421a for Usher syndrome?
“Well, it’s not as far as sepofarsen, but the collaboration with ProQR so far has been fantastic. They’re transparent, forthcoming and I’m impressed with their level of professionalism. They really know how to bring something to the clinic with a high regard for safety. We’re actually looking at additional projects to work on together.”
Do you feel like they mirror your high regard for patients?
“Definitely. They really go the extra mile to ensure safety. My personal yardstick is whether I would put my own children in a trial. If I can’t say that, then I don’t want to be involved. And with ProQR, the answer is ‘absolutely.”