Letter to the Community – Myosana Therapeutics


Dear Duchenne Community, 

I’m truly thrilled about our investment in a new biotech company called Myosana Therapeutics. Myosana aims to develop next-generation gene therapy for Duchenne muscular dystrophy and neuromuscular diseases. Because of this investment, the team is now positioned to conduct further experiments towards achieving their goal.  

I’m moved to express sincere thanks on behalf of CureDuchenne to all our donors and families who supported us with contributions of every size to fund today’s investment. There is a reason why we refer to today as the present – because it’s a gift. Being grateful for today and looking forward to tomorrow all starts with appreciating that gift. We’re very excited about this latest venture and I want to tell you why! 

Myosana’s approach targets skeletal and cardiac muscle over other tissues in the body to deliver full-length dystrophin. That’s a complete replacement of the massive dystrophin gene, something thought impossible using current viral treatments. Their approach will theoretically work for anyone with Duchenne, regardless of a specific mutation. This is a non-viral treatment, so any pre-existing viral immunity associated with viral delivery is irrelevant to this treatment. Redosing of full-length dystrophin could also be possible. This technology builds on what we already have in place, takes a new approach and does not interfere with ongoing therapies. It is potentially a comprehensive treatment for everyone. 

While we’re potentially closing in on something big here, it’s always important to temper our excitement with cautious optimism. We discovered Myosana’s research at a very early stage and they are many experiments away from clinical trials. They will undoubtedly need more support as they continue to prove the efficacy of their treatment, but the research we’ve seen to date is promising. You can find more information on this endeavor and Myosana’s approach on our blog, and the press release can be found here.  If you would like to see this research progress as much as we do, I encourage you to offer your support!  

When you can’t yet see the final answer to a problem, we deem it wise to leave no stone unturned. That’s why we’re constantly searching for innovative ways to offer transformative treatments for every individual with Duchenne muscular dystrophy. This is a fundamental aspect of our venture philanthropy model and this CureDuchenne Ventures investment in Myosana is representative of that approach. By building a light of hope that outshines any darkness, we’re continuing to fight strategically and on multiple scientific fronts.  

Together, we WILL cure Duchenne! 

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