Q4 Newsletter: Accelerating Momentum and Impact at AlphaRose Therapeutics.
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We are filled with immense gratitude for the growth and continued support as AlphaRose closes out a quarter marked by significant achievements and accelerating momentum. Our partnerships with investors, scientists, and patients have been instrumental in our mission to revolutionize access to medicines for ultra-rare disease patients! |
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The Mission: Scaling Genetic Treatments
This past quarter has been transformative. I've had the distinct privilege of presenting AlphaRose's groundbreaking model at prestigious events including the Mayo Clinic, Cell and Gene Therapy on the Mesa, IC3, Capital Factory, Impact Accelerator Summit, Rare Trials Summit, and as the keynote speaker at the Luminary Awards at THBI. These engagements underscore the critical role AlphaRose plays in creating sustainable access to vital medicines and establishing a robust market for ultra-rare disease therapeutics.
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Pipeline: Rapid Progress In The Lab | |
The lab has been exceptionally productive, with five ASOs currently in our pipeline at various stages of development, targeting a range of diseases from bleeding disorders to neurogenetic conditions. We're particularly excited about two ASOs showing early licensing potential, which we believe could significantly contribute to patients, as well as early funding initiatives. Rosiphersen's safety tests continue to yield clean results, paving the way for a clinical trial in 2026. The regulatory landscape, particularly with the FDA and MHRA, is evolving rapidly and favorably for diseases affecting fewer than 1K patients. This global recognition of ultra-rare diseases, which is said to constitute 80-90% of genetic diseases, validates AlphaRose's essential role in advancing human health.
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| | AI: Revolutionizing Drug Design and Efficiency | Our MetaMorph ASO design tool is fully operational in the lab and is now integral to the design of all our new drugs. The team's dedicated efforts in integrating machine learning, AI, RNA folding, and other critical parameters have yielded significant success in predictive toxicity and efficacy. Our CSO, VP of Engineering, and I are actively exploring ways to seamlessly integrate AI into our daily tasks, fostering an organic process of problem-solving that promises substantial gains in efficiency. | | | Platforms: Innovating for Safer and More Effective Therapies | |
Our commitment to innovation is evident in our platform advancements. We are collaborating with NATA in the UK on a crucial experiment to further de-risk our abcDNA ASO chemistry. Our aim is to develop a potentially safer and more durable alternative to the widely used standard MOE ASO. Simultaneously, initial experiments are underway for our epigenetic RNA platform, "SOT Caller." We are optimistic that this technology will prove its ability to regulate gene expression, offering an incredibly impactful solution for loss-of-function genetic diseases.
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Rose: The "Why" We're Here
Rose continues to thrive, remaining happy and stable. Her seizures are effectively managed by her current medication, a blessing for which we are immensely grateful as it mitigates the risk of death in epilepsy. She enjoys watching animal videos and diligently works on life skills at school, including potty training, sharing, and throwing a ball.
Check out the most recent blog I wrote about how Rose has inspired me...
| | | | Our Regulation CrowdFunding round was a success, raising $1.7M from investors across the globe. We are actively pursuing significant non-dilutive capital opportunities, including the ARPA-H THRIVE grant, an SBIR, and the UO1 with the NIH over the next 3-6 months. These initiatives could collectively bring in $50-$100M, and we are incredibly excited about the potential impact these partnerships will enable. | |
Convertible Note #2: $3M Oct 1st - Nov 31st.
We have launched Convertible Note #2 for $3M, open from October 1st to November 31st, and have already received incredible interest. We have secured verbal commitments for $2M, leaving $1M available for accredited investors to participate. Highlights of this note include a minimum check size of $50,000, a $20M cap, a 15% discount, a 7% interest rate, and a percentage of warrants.
If you are an accredited investor and wish to learn more about this compelling investment opportunity, please email me to learn more or schedule an appointment.
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As you may can tell, AlphaRose is experiencing an unprecedented period of acceleration and momentum, with a multitude of opportunities, strategic partnerships, and value-creating events on the horizon. We deeply appreciate your partnership as we collectively strive to build a successful commercial company that delivers life-changing solutions for patients.
Sincerely,
Casey McPherson
CEO
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