AlphaRose Therapeutics, a pioneer in precision genetic medicine, announced it has been awarded National Institute of Health’s (NIH) Small Business Innovation Research (SBIR) Fast Track grant. This highly competitive funding will accelerate the clinical development of Rosiphersen (ART-001), the company’s lead antisense oligonucleotide (ASO) therapy designed to treat Bain Syndrome, an ultra-rare and severe pediatric neurodevelopmental disease.

Bain Syndrome is caused by pathogenic genetic variants in the HNRNPH2 gene, resulting in devastating symptoms, including severe developmental delay, autism, seizures, loss of communication and impaired motor function. With approximately 185 cases confirmed to date, there is currently no cure for HNRNPH2-related neurodevelopmental disorders.

Driven by a mission to leave no patient behind, AlphaRose leveraged its proprietary AI drug design operating system, RINAE.AI, to generate Rosiphersen. This state-of-the-art platform significantly reduces discovery timelines from months to minutes. Using training data, predictive algorithms, AI and machine learning, RINAE.AI designs best-in-class ASO candidates that demonstrate compelling safety and efficacy profiles in preclinical models. 

“I founded AlphaRose after my daughter, Rose, was diagnosed with an ultra-rare genetic disease with no cure. This SBIR Fast Track grant is a pivotal milestone for AlphaRose and the patients we serve,” said Casey McPherson, CEO and Founder of AlphaRose Therapeutics.

Accelerating to the Clinic

The SBIR funding is strategically allocated to advance Rosiphersen toward an Investigational New Drug (IND) application. Key upcoming clinical development milestones include:

• IND-Enabling Studies: Finalising critical regulatory requirements and safety toxicology validations.
• Natural History Study: Expanding the Enhanced Monitoring for Rare Longitudinal Data (EMRLD) initiative.
• First-In-Human Data: Targeting first-in-human safety data by end of 2027 to demonstrate clinical viability.

“Leading our scientific and technology teams alongside Casey to develop treatments for rare genetic diseases is a metamorphic mission. I am immensely pleased that we have reached this milestone, which underscores the power of the RINAE.AI platform to create a repeatable, scalable model for genetic medicine,” said Robert M Cabrera, CSO of AlphaRose Therapeutics.

Beyond the capital, this SBIR award represents a significant validation of AlphaRose’s mission to fundamentally transform the landscape of rare disease drug development. As Rosiphersen advances toward the clinic, it establishes a definitive proof of principle for the RINAE.AI platform, paving the way for a repeatable drug discovery engine capable of delivering hope to millions of rare disease patients who have been left behind.