AlphaRose Secures NIH SBIR Fast Track Grant to Advance Rosiphersen for Bain Syndrome

AlphaRose Therapeutics said it has received a multi-million-dollar Small Business Innovation Research Fast Track grant from the National Institutes of Health to support development of Rosiphersen, an experimental antisense oligonucleotide therapy for Bain syndrome, an ultra-rare pediatric neurodevelopmental disorder.

The company said the unspecified funding will be used to advance Rosiphersen, also known as ART-001, through the studies required before filing an investigational new drug application with the U.S. Food and Drug Administration. AlphaRose is targeting initial human safety data by the end of 2027.

Bain syndrome is associated with disease-causing variants in the HNRNPH2 gene. The condition can cause severe developmental delays, autism, seizures, loss of communication abilities and impaired motor function. About 185 cases have been confirmed to date, according to AlphaRose, and there are no approved disease-modifying treatments for HNRNPH2-related neurodevelopmental disorders.

Rosiphersen is an antisense oligonucleotide, or ASO, therapy. ASOs are short, synthetic strands of genetic material designed to alter RNA processing or expression. The approach has been used in several rare genetic diseases, although AlphaRose’s candidate remains preclinical.

The NIH Fast Track award is intended to support both early research and later-stage development activities. AlphaRose said the grant will fund IND-enabling safety and toxicology studies, as well as expansion of its natural-history program, Enhanced Monitoring for Rare Longitudinal Data, or EMRLD.

“This SBIR Fast Track grant is a pivotal milestone for AlphaRose and the patients we serve,” said Casey McPherson, the company’s founder and CEO. McPherson founded the company after his daughter, Rose, was diagnosed with an ultra-rare genetic condition with no cure.

AlphaRose said it used its proprietary artificial intelligence drug-design platform, RINAE.AI, to generate Rosiphersen. The company said the platform combines training data, predictive algorithms and machine-learning methods to identify and design ASO candidates, with the goal of shortening the drug-discovery process.

Robert Cabrera, AlphaRose’s chief scientific officer, said the grant also supports the company’s ambition to develop a repeatable platform for creating therapies for rare genetic diseases.

“Reaching this milestone underscores the power of the RINAE.AI platform to create a repeatable, scalable model for genetic medicine,” Cabrera said in a statement.

Photo: Casey McPherson, founder and CEO of AlphaRose