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Erik Aznauryan on Curative Treatment of Genetic Diseases

The Humans of the Wyss (HOW) series features members of the Wyss community discussing their work, the influences that shape them as scientists, and their collaborations at the Wyss Institute and beyond. 

Growing up with an ophthalmologist as a father, Erik Aznauryan’s interest in medicine began at a young age. Eventually, he realized that the scientific aspects of medicine interested him far more than the clinical aspects. Now, instead of prescribing treatments, he’s creating them. At the Wyss, he’s developing a next-generation genome editing platform to more safely and effectively treat genetic diseases, like beta-thalassemia or cystic fibrosis. Learn more about Erik and his work in this summer Humans of the Wyss.

What are you working on?

Erik Aznauryan on Curative Treatment of Genetic Diseases
Erik Aznauryan, Technology Development Fellow. Credit: Wyss Institute at Harvard University

I’m working on a next-generation genome editing platform called HarborSite. This multi-component technology allows us to make large, efficient, durable, and safe changes in the human genome for therapeutic purposes. Specifically, we can use it to introduce functional copies of mutated genes into desired spots in the genome of the affected cells to alleviate the effects of those mutations.   

What real-world problem does this solve?

Genetic diseases, like cystic fibrosis, are conditions that result from detrimental mutations in human genes. Often, these are large genes that are altered in multiple spots. To treat them, you would need to fix each mutation in the gene. Current gene-editing methods are focused on fixing one mutation at a time, which is not scalable, or if they address all of them by introducing an entire functional copy of the gene, the process is either very inefficient or unsafe, or the effects are not durable.  

Our tool attempts to treat genetic diseases by inserting full copies of therapeutic genes into safe locations in the human genome in a more efficient way than the options available now.  

What inspired you to get into this field?

My dad is a pediatric ophthalmologist, so he instilled in me an interest in medicine at a very young age. At first, I thought I’d become a doctor, but I soon realized the scientific aspects of medicine, like developing new therapies, excited me more than the clinical aspects, so I switched my focus. Being exposed to a wide range of scientific projects during undergraduate and master’s studies – from mitochondrial diseases to metabolic engineering to non-coding RNAs – helped me decide what to focus on in graduate school.  

What continues to motivate you?

Erik Aznauryan on Curative Treatment of Genetic Diseases
Erik Aznauryan (right) with Tina Lebar (center) along with Wyss Business Development Manager Bill Bedell (left) are working on developing and de-risking HarborSite. Credit: Wyss Institute at Harvard University

It’s an incredible opportunity to build a new, exciting technology from the ground up and ultimately be able to impact a lot of people. Plus, I’m not doing this alone. My primary partner is Tina Lebar, and the energy that she puts into this work radiates onto me. We also partnered with Business Development Manager Bill Bedell who helps us think of our technology in commercially relevant terms.  

What excites you most about your work?

The environment you are in dictates your state of mind to a large extent, and the environment at the Wyss is so supportive of translational research.  Being surrounded by people who are not only scientifically rigorous, but also looking to apply their knowledge and skills to create therapeutic products, is incredibly motivatingyou realize that your mindset is shared by many of your peers.  

The environment you are in dictates your state of mind to a large extent, and the environment at the Wyss is so supportive of translational research.

Erik Aznauryan

What are some of the challenges that you face?

You can spend a year without any desired or meaningful data that you need to move on to the next step. Of course, when you finally get it there’s a big celebration. But maintaining that motivation and direction towards your goal can be challenging when there are a lot of setbacks. For me, the potential to create something that will one day be a solution for many people keeps me going during those times.  

How did you begin your journey with the Wyss? Why did you want to work here?

Erik Aznauryan on Curative Treatment of Genetic Diseases
Erik and other members of the Church Lab, including Denitsa Milanova (second from the right), during the 2019 Wyss Retreat, while he was a visiting graduate student. Credit: Wyss Institute at Harvard University

It’s very hard not to hear about the Wyss if you’re in the biotech world! It’s one of the best institutes for biology research and beyond. George Church is a pioneer of genome editing, so I also wanted to work with him. During my Ph.D. I reached out to him, shared my previous research and future project ideas. Luckily, he was interested in having me come over and connected me with former Wyss postdoc, Denitsa Milanova. We got along immediately. I appreciate both of them so much for their part in my scientific journey. I worked with them as a visiting graduate student from October 2019 to May 2020, which was a very fruitful time. Once I finished my Ph.D., I returned to the Wyss as a postdoc – and now as a Technology Development Fellow – to focus on my current work.  

What is unique about the Wyss and how has that impacted your work?

One thing I really like about the Wyss is the diversity of expertise, which I think is most important for science. You have people who are world leaders in different scientific areas under one roof. I can tap into their knowledge, and they can tap into mine. This dramatically facilitates the progress of my research. Additionally, the internal project funding structure developed in the Institute is incredibly valuable for early-stage technologies seeking further de-risking.  

How do you collaborate with other teams across the Wyss?

Erik Aznauryan on Curative Treatment of Genetic Diseases
Erik enjoys learning from researchers across disciplines at the Wyss. Here he and Tina Lebar discuss a demo presentation at the 2022 Wyss Retreat. Credit: Wyss Institute at Harvard University

Because there are people with all different expertise, it’s easy when there’s a central point of contact. Jenny Tam is a member of the Advanced Technology Team and oversees many projects. Whenever I’m looking for something, she can connect me with the right person. For example, when we initially applied to become a Validation Project, we were going to use a type of stem cells that we had never tried before. It turned out there was a group in Don Ingber’s lab that had been working on them for a long time, and another group in the Church lab that was starting to use them as well. All three teams connected over that, sharing protocols, knowledge, experiments, and relevant results.  

How have your previous work and personal experiences shaped your approach to your work today?

The key to working in a field where you have very delayed gratification or results, like science, is to maintain your focus and motivation. Doing a Ph.D. is such a lengthy and complicated journey, and you’re so entrenched in your topic 24/7, that it is very easy to be overwhelmed. Going through that helped me build the mental resilience to handle unexpected or unfortunate outcomes. That’s something I’ll apply throughout my career. Additionally, being an immigrant makes you accustomed to leaving your comfort zone, which is a very useful skill when navigating the scientific unknowns.

The key to working in a field where you have very delayed gratification or results, like science, is to maintain your focus and motivation.

Erik Aznauryan

When you’re not in the lab, how do you like to spend your time?

I have two kids, and they take up most of my non-work time. Mornings, evenings, and weekends are dedicated to them. Our broader family is very closely connected, so we try to share our free time together. I also volunteer for science education initiatives in Armenia where I come from. 

What’s something unique about you that someone wouldn’t know from your resume?

I really enjoy playing piano and saxophone. I don’t think I’m particularly good at either, but I enjoy it as something for myself. The saxophone is hard on the neighbors, but we have an electric piano, so I can adjust the volume, making it easier to practice.  

If you had to choose an entirely different career path, what would it be?

It would be something related to foreign relations in a government organization, like some kind of diplomatic work. It’s exciting building ties with people from different places and cultures and aligning diverging interests into mutually beneficial solutions. Plus, I speak four languages – English, Armenian, Russian, and Spanish – so that would help.  

What does it feel like to be working on cutting-edge technology that has the potential to have a real and significant impact on people’s lives and society?

I know I’m in a great place, given this amazing chance, surrounded by smart people, so it’s my duty to take advantage and develop something that will be valuable for others.

Erik Aznauryan

It’s very inspiring. Knowing what I do will ultimately be beneficial to others is what motivates me during those times of delayed gratification. It also brings a lot of responsibility – I know I’m in a great place, given this amazing chance, surrounded by smart people, so it’s my duty to take advantage and develop something that will be valuable for others.

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