Gerard Platenburg, Chief Innovation Officer at ProQR Always excited about RNA therapies

Let’s start at the beginning. How did you meet Daniel de Boer, the other founder? 

“It was through a mutual acquaintance actually. Daniel has a child with cystic fibrosis (CF), a rare genetic disease, and at the time he was already looking for funds to find a treatment. I had been involved in RNA therapies for years, having co-founded a company for Duchenne muscular dystrophy, and I really enjoy turning new scientific ideas into clinical reality. Our mutual friend had a keen eye for that, and he introduced us. We hit it off and we decided that we should try to find that CF treatment ourselves instead of just funding research. We found interesting technologies we were able to license in, for both CF and the retina as well. The rest is history, as they say.”

What made you pursue RNA therapy as a potential treatment for these diseases? 

“Well, part of me wanted to do this because I really want to help people. But I didn’t want to create yet another medicine against headache, for example, and it appealed to me that nobody was looking at RNA therapy as a tool against genetic diseases. That is the personal part, my motivation. More importantly, RNA therapy is just a great approach. A lot of those genetic diseases are diseases because the genetic mutation causes a protein to be made in the wrong way, with the wrong shape, a missing part, or things like that. Our knowledge about what certain mutations do has grown a lot over the years, and we have developed a toolbox of various ways in which we can use RNA therapies to fix these mistakes. At this moment we really are at the forefront of this development, but there is still so much potential out there. We currently have ten different techniques in our toolbox, but I think there are up to a hundred ways to apply RNA therapies. And if you look at the diseases, we have only covered about one percent of the retinal diseases so far. So, I think we are only at the start of the total potential of RNA therapies.”

But what about gene therapy, isn’t that an option?

“There are several reasons to choose RNA therapy over gene therapy or even gene editing using CRISPR-Cas, the latest hype. And the main reason is that it is reversible. RNA therapies do not make changes to your DNA, so when you find out that a therapy has adverse effects, you can stop it, like any other medicine. With gene therapy or gene editing, if you change something for the worse, than that is permanent as well. Additionally, to apply gene therapy you have to use viruses, which may initiate an unwanted immune response.”

What does your current role, Chief Innovation Officer, entail?

“My responsibility is the discovery of new techniques and ideas at ProQR, and to develop those to a certain level. Personally, I draw inspiration from the conferences I visit, and I read a lot. I try to make new combinations and to be open to new ideas that I encounter. I see the potential often before there is proof, so thankfully I have a team of around 55 excellent scientists that help me bring the theory into reality with the evidence.” 

What can you tell us about the recent developments at ProQR? 

“So far, we have been very effective in bringing ideas to the treating patients with it in clinical trials, with an average of almost one per year. And we will keep doing this, as we have promised.”

“In terms of technology, I’m also very excited that we are still expanding our toolbox. We can now do single base RNA editing, turning a single A nucleotide into an I. It doesn’t get more nano-surgery than that. As it happens, there are twenty thousand mutations that could potentially be reversed by such an edit. Of course, we must look at each one individually, but the potential is enormous, and I’m proud to say that we are way ahead in this field.”

“To mention one more, we are looking into doing more with our data. We have so much data from animal studies, from human trials, from the different tools in our toolbox, and we want to see what we can learn from it. How can we make the screenings more efficient for example? Can we learn more about our molecules, and does it help to optimize our protocols? Those are just some of the questions we’re trying to answer.”

Your innovation department is very busy. Can you handle everything that they come up with? 

(smiles) “I have to be thankful to (our CEO) Daniel that he wants to invest so much in innovation. About one third of the company is dedicated to innovation, which is unique. And they generate many ideas, or they find applications for ideas that I come up with myself. At the same time, we want to maintain focus and right now that is eye diseases. If our team comes up with an idea, a target or a technology that doesn’t fit that focus, we spin those ideas out as separate companies. That way they can be explored and developed further, while generating the value that allows us to keep innovating. I may be biased, but I think innovation is the mothership for ProQR.”

Learn more about RNA therapy.