Hey there!
We have a lot of exciting updates from the last month: Rose's cells are in the lab and looking good. We had a great call with our science team a few weeks ago discussing a lot of the variables involved in getting us to the next steps. Rose, like most girls, has two-X chromosomes: one with a good gene, and one with the mutated one that's causing her disease.
It's tricky getting the cells we produce in the lab to present data from the mutated one - the one we want to fix. The science is complicated - but we are finding a way though. I'm so grateful to work with a brilliant team across the country who not only know how to do all of these amazing things and get around those issues... but truly care about Rose.
Instead of putting all our eggs in one basket, we're looking at multiple paths to a cure for Rose. We're looking at drugs we can repurpose, small molecules, and we're developing our Oligos - also known as ASOs - a newer genetic treatment that has worked wonders on other monogenic rare diseases.
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We're printing 200 ASOs and each one is about $300. Since we've asked folks to sponsor each design, we've covered the cost of 37 of the 200 we're making, from 30 different sponsors. Thank you all!
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(In case you missed it, Wes and I sung a song about Oligos a few days ago!)
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FYI - We are halfway through our matching donation campaign! For the next few weeks, every donation you make will be matched, dollar for dollar, up to $50,000.
Please help us reach that full 50K!
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I hope you join us at our next Fundraiser - the first ever Rose Garden Games!
It'll be like a big play-day for grown ups. We'll have food, drinks, golf, cornhole, karaoke, oversized lawn games, a concert... it's going to be epic. We have tickets available now, so you can grab a friend and register as a player in one of our tournaments, OR you can grab a ticket to just hang out and enjoy the festivities. Either way - hope to see you there!
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ROSE GARDEN GAMES
SATURDAY, NOVEMBER 5th
BUTLER PITCH AND PUTT
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Going into this, I knew it would be an expensive journey. Developing a drug from start to finish can take years, and cost tens of millions of dollars. We're moving a lot quicker, and spending a lot less money.
We've raised around half a million dollars so far to get to this point, and we still need to raise another $600,000 over the next six months to reach the next stage of this process. I'm shocked when I think about how big these numbers are to me, but how small they are relative to what it would usually take to do this. It's honestly amazing.
I'm so honored to be here, and to see a better life for these kids in the not-so distant future. And that's all because of you. Thanks for being here and making this happen.
Fighting together for these children,
Casey
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