Hee Lam Chan, Principal Researcher Ophthalmology at ProQR Still going strong / One challenge after another

What attracted you to work for ProQR when it was still so small? 

“I studied biomedical sciences in Leiden, and when I graduated, I was able to stay for one year at the place I did my internship at. But after that year I had to look for something else. I didn’t really like the prospect of doing a PhD, because it requires you to really attach yourself to one topic for four years, and that’s not my personality. I started looking at the Science Park here in Leiden for options. At the time ProQR was looking for five technicians, so I figured that I had the best chance there, being fresh on the job market.”

Seven years later and you are still here. It must have captivated you.

“Yes, it’s a very dynamic organization. Everything moves quickly, and you must be flexible. This pace suits me well. I really enjoy diving into something and figuring it all out. But when I hear that there is a new project, I don’t mind switching gears and committing to the new challenge at hand. The last seven years just flew by. Sometimes friends ask me if it’s not time to look for something else, but I disagree. There are plenty of opportunities that can keep me interested here.”

What kind of opportunities did you have at ProQR? 

“All kinds. I started out with the cystic fibrosis project that started it all. Then I worked on the Leber congenital amaurosis program, and then retinitis pigmentosa. And now I work on the organoids we use for retinal disease research. I don’t always ask for these projects; sometimes when they start something new they’re like ‘Hee Lam will like this’, and then it just falls in my lap. (laughs) But I also get to do other things. For example, I am in the organizing committee for the annual ProQR conference. During that conference we go somewhere with the whole company for a change of scenery, workshops, presentations and to have fun. It’s nice, and a great way to meet others in the organization.”

How would you describe the company culture?

“It’s really open actually, like a big family. When I got here and there were just six of us, it’s only natural that you know what everybody’s working on but also what they’re up to in their free time. But even as the company grows, they are trying to maintain that openness. For me that’s great, because I’m a bit introverted and this makes it easier to get to know others. It was a chance for me to develop my soft skills. And there are so many interesting people here too! I think we have around 35 different nationalities, and everybody respects each other. It’s okay to be different.”

You already mentioned retinal organoids, the little organs that resemble a patient’s retina, which makes for great testing environment for new therapies. Is that your latest topic? 

“That’s right. We used to rely on partners to make the organoids, but it’s always better to have that capability in-house. So, me and a few colleagues tried to create them ourselves. We were inspired by our partners of course, but we also used a lot of literature and distilled from that what works. The past year we have been working on optimizing protocols to grow cells from a patient’s skin into a tiny retina in our laboratory. We have another group at ProQR that looks at new targets for eye diseases, and when they have screened them in normal cells, we screen them in organoids because it resembles the human eye so much more. When they pass that test, we can move further with the development. Now that we are doing these tests, we discover that every cell line is different. Just like every patient reacts differently to certain compounds, so do their organoids. That means we have to test a variety of protocols for every cell line.”

How did you test sepofarsen in the organoids?

“We have started testing sepofarsen, our investigational RNA therapy for Leber congenital amaurosis, in the organoids as well. It’s like a step between cell screenings and animal studies. The organoids are a better model than the mouse models traditionally used, because it shows what actually happens in the right tissue, in humans. We are still perfecting it and it has so much potential. We could use this in our clinical trials. We can get a skin sample from the participants, and see how the patient reacts to the treatment but also study the same treatment in the organoid to see if the results match. If it matches we could predict what treatment works in a patient before we even start a trial. That would be a big step towards truly personalized medicine.”

Interested to learn more about what ProQR is doing for inherited retinal diseases? Check out our pipeline of investigations RNA therapies.