As with many RARE entrepreneurs, Courtney Young was thrust into the RARE space through personal experience and is now dedicated to developing therapies for rare muscle diseases. She shares the importance of having a passion for what you do and of connecting with the RARE patient community

RARE entrepreneurs: meeting the beating hearts behind the RARE brands

MyoGene Bio is a biotechnology startup dedicated to developing therapies for rare muscle diseases. Our first approach is a gene editing therapy for Duchenne muscular dystrophy to permanently remove the mutations that cause Duchenne for around 50 per cent of patients. We are inspired by patients to develop truly meaningful therapies for rare muscle disorders. MyoGene Bio was spun out in 2019 by Drs Courtney Young, Melissa Spencer and April Pyle.

1.

What was the driving force is starting your own business in the rare disease space? Was there an unmet need you were responding to?

I was exposed to the muscle disease space when my cousin was diagnosed with Duchenne muscular dystrophy when I was in high school. Duchenne is a rare muscle wasting disease that leads to premature death and has only limited treatments. Before my cousin’s diagnosis I had not heard of this disease and when we found out there was no cure, my family was devastated. I became interested in muscle disorders and began working in various related research laboratories around the world starting in undergraduate and continuing through to my postdoctoral fellowship at the University of California, Los Angeles (UCLA). We started MyoGene Bio based on my PhD and postdoctoral work and I have been working there full-time since mid 2019. I have dedicated my career towards developing therapies for rare muscle diseases. There is still no cure for Duchenne, but ongoing research and clinical trials offer hope. 

2.

How does your business benefit the rare disease community?

MyoGene Bio is a biotechnology startup dedicated to developing meaningful therapies for muscle diseases. Our first approach is a gene editing therapy for Duchenne muscular dystrophy that removes the underlying cause of the disease to restore the protein otherwise lacking. We are committed to developing therapies that have truly meaningful impacts on disease progression for rare muscle wasting disorders.

3.

What advice, if any, did you get when setting up your business? Has there been anyone in particular who has been pivotal in supporting your business?

MyoGene Bio is co-founded by myself, Dr Melissa Spencer and Dr April Pyle. Drs Spencer and Pyle were my advisors during my PhD at UCLA and together we developed this first therapy for Duchenne. I wouldn’t have been able to do the research or start MyoGene without their support. Additionally, I have been strongly supported by my family and friends who offer advice, provided initial funding and inspire me. Some advice I received was to pursue something you are passionate about and to hire a good lawyer.

4.

How do you manage the demands of running a business with your own health needs, those of someone you care for, or those of your employees?

Running a business can be all-consuming, thus I believe it is important to have some work-life balance through hobbies or routines where you are forced to stop thinking about your business for some amount of time. Some of the things I do include playing tennis, baking, and reading before I go to bed every night to take me into a different story.

5.

What advice do you have for someone starting their own business?

Starting your own business forces you to learn a lot very quickly. You should be prepared for a steep learning curve and don’t hesitate to ask for advice from others around you. I would also say it’s important to do something you are passionate about that you couldn’t imagine someone else doing instead of you.

6.

What are the most rewarding aspects of establishing and running your own business?

The most rewarding aspect of running MyoGene Bio is being able to plan how we will bring a therapy to patients. Our ultimate goal is to treat patients that have rare devastating disorders with treatments that would make a meaningful impact on their disease progression. Thus, being able to direct the development of therapies to have the biggest impact on patient’s lives is extremely rewarding.

7.

What would you consider to be the greatest achievements of your business thus far?

I think the greatest achievement at MyoGene Bio so far is our recently awarded CIRM TRAN grant of $3.4m. Read more here. This funding provides validation and support for our ongoing preclinical studies as we work to bring our therapy to patients.

8.

What advice would you give someone considering working in the rare disease space?

Working in the rare disease space, my advice would be to connect to the patient community. Usually in rare diseases there is strong patient advocacy and it can be really meaningful to interact with them to get feedback and understand the needs of the patients.

9.

What are your hopes for the future of your business?

Looking towards the future, I am hopeful we can continue to develop safe and effective therapies for the thousands of patients with rare muscle wasting diseases.

10.

If you hadn’t founded MyoGene Bio, what was Plan B?  What did your 10-year-old self want to be?

If I hadn’t founded MyoGene Bio I would still have wanted to go into research in muscle diseases, at a university or company. However, my 10-year-old self wanted to be a vet.