AlphaRose Therapeutics

About the offering

Alpha Rose Therapeutics, born from a father's love for his daughter, is pioneering personalized medicine at scale for children with genetic diseases. Our innovative approach rapidly develops and commercializes treatments for patients ignored by large pharma. With over $1 million in funding, our lead product, Rosiphersen, is advancing towards a clinical trial, backed by the NCATS TRND program acceptance and led by a passionate, experienced team. AlphaRose Therapeutics is an early-stage biotechnology company, and its treatments, including Rosiphersen, are still in development and have not yet been approved for clinical use. Addressing a Critical Need: 200m+ children suffer from genetic diseases, and only 5% have any treatments. Alpha Rose’s technology and business model are designed to treat many of these patients that large pharma has ignored. Niche Market Advantage: With the focus on neurodevelopmental conditions using antisense oligonucleotide technology, Alpha Rose is positioned in the growing $21 billion genetic disease treatment market. Lower Risk: Historically, drug development costs $500M+ with low success rates due to safety concerns. Our ASO platform has the potential to mitigate risk and reduce costs for small population diseases. THE STORY Alpha Rose Therapeutics was founded by Casey McPherson, lead singer and song-writer, entrepreneur and father to Rose who was diagnosed as a baby with a rare genetic disease. At eight years old, Rose is now forced to face the world without words, scared by her seizures, unable to feed or take care of herself, and often alone. Casey has been fighting for his daughter with a leading voice in the rare disease field, initially through his non profit foundation and scientific lab. Casey and his science team developed a preclinical treatment for his daughter’s disease in less than a year. In his journey, he recognized the need for an innovative business model in biotech to bring his team’s process to millions of children like his daughter, which other companies are not focused on. McPherson founded Alpha Rose Therapeutics, a PBC, together with industry leaders, Belinda Termeer, Masako Nakamura, and Alan Walts, who worked alongside Henri Termeer at Genzyme in pioneering the field of rare disease biotech. Together they are building a “patient first” company again, with Alpha Rose. THE PROBLEM The technology is here. The business model is not. Hundreds of millions of sick and dying children suffer from rare genetic diseases. Most of these diseases fall underneath the threshold of what the current pharma considers worthy programs. While individually rare, they make up over 10,000 different diseases, and 400m patients worldwide. Most of these diseases have populations in the hundreds that traditional biotech and pharma companies will not prioritize for development. The current pharma model focuses on developing treatments that reach large populations, often only treating symptoms. This leaves the majority of genetic diseases with small populations, half of those being children, without any potential treatments. What about the millions of these other children? THE SOLUTION Ushering in a new era of personalized medicines Alpha Rose’s technology, platform, and sustainable business model are built to bring patients access to these medicines. They aim to rapidly develop, and profitably commercialize these small population medicines at scale with their antisense oligonucleotide (ASO) technology. They have witnessed this technology to be efficacious in more common rare diseases like SMA, ALS, and Angelman’s, where treatments are enabling many patients to walk and even talk. Alpha Rose is developing treatments for these ignored patient populations with the intention of exploring global commercialization. Alpha Rose plans to develop a portfolio of these medicines, while creating efficiencies and scale through iterative learning, AI and robotics. By putting patients first, Alpha Rose believes they can build the next pharma company, for good. “When you’re trying to save the life of a child, the system will reorganize itself to help you.” - Henri Termeer THE MARKET A Global Impact for Underserved Patients Personalized genetic medicine is a rapidly growing market, as science shows that precision medicines targeting the underlying cause of disease leads to a better outcome for patients. Rospihersen, Alpha Rose’s lead product, targets a market estimated to be between $100M - $700M in annual peak revenue, based on current market estimates. The pediatric genetic treatment market is valued at over $21 billion and climbing. They are starting with rare neurodevelopmental conditions affecting children, like severe autism, and are positioned well within this growing field of antisense oligonucleotide (ASO) therapy—a specialized sector that is well known to be an innovative tailored treatment solution for small population patients. This market includes a high demand for precision medicine due to increased diagnostic capabilities, anticipated growth of rare disease-focused therapeutics, and the lack of companies in this space. TRACTION Alpha Rose’s team, in collaboration with To Cure A Rose Foundation, developed the proprietary treatment, Rosiphersen, for genetic mutations in HNRNPH2, which can cause severe autism, seizures, severe developmental delays, and early morbidity. Rosiphersen has shown to be safe in vitro and in vivo models. They plan to use these funds to treat Rose and other patients with Rosiphersen, after preparing for the FDA clinical trial that will begin in Q4 2025/Q1 2026. A Developing Pipeline. Through their initial AI program “Argus”, they have discovered many new disease targets amenable to their technology, and plan to design the new treatments, file patents, and move them through their iterative rapid development platform. Acquiring a new ASO technology company. They are in active discussions to acquire a potential groundbreaking technology with the ability to further differentiate their platform. This would also give them the ability to license the technology to other companies in various areas of medicine, pursuing co-development and co-licensing agreements. From the founder Casey McPherson, CEO/Founder "I'm thrilled to offer you the chance to join us in revolutionizing medicine for children in need. By investing in Alpha Rose, you're not just supporting a company; you're becoming a champion for these children, ushering in a future where personalized medicine is accessible to all."

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