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Austin, TX

Advancing AI-driven, personalized genetic medicines for children with rare diseases.

• Addressing an Unmet Need: Developing therapies for genetic diseases that affect millions of children yet remain unaddressed by large pharmaceutical companies.
• Scalable Drug Development Model: Using antisense oligonucleotide (ASO) technology to efficiently create tailored treatments for small patient populations.
• Strong Market Opportunity: Operating in the growing $21 billion genetic disease treatment market with high demand for precision medicine.
• Proven Scientific and Business Leadership: Led by experts from Genzyme, Amylyx, and other pioneers in the rare disease space.
• Clinical Trial Readiness: Preparing for FDA clinical trials for Rosiphersen, with a long-term vision to commercialize multiple life-changing therapies.

AlphaRose Therapeutics is pioneering a new approach to personalized medicine for children suffering from rare genetic diseases. Founded by Casey McPherson, whose daughter was diagnosed with a severe genetic disorder, the company is built on a mission to bring life-saving treatments to underserved pediatric patients. Traditional pharmaceutical models overlook small population diseases, leaving millions of children without viable treatment options. AlphaRose is addressing this gap by leveraging antisense oligonucleotide (ASO) technology to develop targeted therapies that go beyond symptom management to directly address the genetic root causes of these conditions. The company’s lead treatment, Rosiphersen, is designed to treat neurodevelopmental conditions associated with the HNRNPH2 mutation, a disorder causing severe autism, developmental delays, and early mortality.

Backed by over $1 million in funding and acceptance into the NCATS TRND program, AlphaRose is preparing for its first FDA clinical trial in late 2025 or early 2026. The company’s innovative AI-driven drug discovery platform, Argus, has already identified additional disease targets that could be treated using its proprietary ASO technology. Beyond internal drug development, AlphaRose is exploring strategic acquisitions and licensing opportunities to further expand its capabilities. By building a scalable model for personalized medicine, the company aims to redefine rare disease treatment, creating sustainable solutions for patients long ignored by the pharmaceutical industry. The funds raised will accelerate clinical development, expand the therapeutic pipeline, and bring much-needed treatments to children and families worldwide.

Company Info

AlphaRose Therapeutics develops personalized medicines for children with rare genetic diseases, using antisense oligonucleotide technology to create scalable treatments.

AlphaRose Therapeutics is a biotechnology company focused on developing personalized medicines for children with rare genetic diseases. The company leverages antisense oligonucleotide (ASO) technology to create targeted therapies for conditions that have been largely ignored by traditional pharmaceutical companies due to their small patient populations. With a mission to bring effective treatments to the millions of children affected by rare neurodevelopmental conditions, AlphaRose is pioneering a new approach that combines scientific innovation with a sustainable business model. Their lead product, Rosiphersen, is advancing toward clinical trials with the goal of treating severe autism, seizures, and developmental delays linked to HNRNPH2 mutations.

Founded by Casey McPherson, a musician and advocate for rare diseases, AlphaRose Therapeutics was born from a personal mission to find a cure for his daughter’s condition. The company has built a leadership team with deep experience in biotech, including executives from Genzyme, Amylyx, and Alnylam, who bring expertise in rare disease treatment and commercialization. The company is also developing a pipeline of additional treatments using AI-driven drug discovery and robotics to accelerate development. By focusing on small-population diseases and leveraging innovative scientific approaches, AlphaRose aims to transform the landscape of pediatric genetic medicine while creating scalable, life-changing solutions for patients worldwide.