Dear Duchenne Supporters,
I’m truly thrilled about our investment in a new biotech company
called Myosana Therapeutics. Myosana is developing a 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 and follow-on investments.
I’d like to express sincere thanks on behalf of CureDuchenne to all the donors
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!
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 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. While the research we’ve seen to date is promising, they
will undoubtedly need more support as they continue to prove the efficacy of
their treatment. You can find more information on this endeavor and Myosana’s
approach on our blog or in
the press release. 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!
