The regulatory path for individualized therapies
The FDA has issued draft guidance on evaluating individualized therapies when very small patient populations make randomized controlled trials impractical. The agency’s framework concerns evidence of safety and effectiveness for therapies aimed at a specific genetic, cellular or molecular cause, according to the FDA. A study cited in PubMed Central says there is currently no FDA-approved treatment addressing the underlying mechanism of HNRNPH2-related neurodevelopmental disorder; care remains focused on symptoms.
Testing and development
A National Institutes of Health grant will support development of Rosiphersen, AlphaRose Therapeutics' lead antisense oligonucleotide candidate for HNRNPH2-related disease, toward an IND application.
AlphaRose Therapeutics identifies Rosiphersen, also called ART-001, as its lead antisense oligonucleotide candidate for HNRNPH2-related disease. In an announcement carried by CheckOrphan, the company said its RINAE.AI platform helped design the drug and set a goal of obtaining first-in-human safety data by the end of 2027. These are company-reported development claims.
Funding remains a major hurdle
The Statesman reported that McPherson estimates another $5 million would be needed to reach a first human trial.
Rose has HNRNPH2-related neurodevelopmental disorder, and the Statesman reported that she lost the ability to communicate as the condition progressed. McPherson founded AlphaRose Therapeutics after her diagnosis. The grant is intended to advance Rosiphersen toward an IND application.
