Treatment categories
The FOXP1 Medical Research Foundation takes a rigorous, evidence-driven approach to treatment development. We prioritize strategies that reflect the most advanced and clinically validated therapeutic modalities for rare, single-gene neurological disorders, drawing on lessons learned from related conditions. Our objective is to move as rapidly and responsibly as possible toward therapies that deliver meaningful improvements in our children’s lives.
Small molecules
Small molecules are the traditional drugs most people are familiar with—the kinds you’d find in a pharmacy or medicine cabinet. While they don’t correct the underlying genetic cause of FOXP1 syndrome, they can help address downstream biological and functional effects of reduced FOXP1 activity, such as imbalances in signaling pathways or neurotransmission. There are two types of small molecules: repurposed and novel.
Repurposed Small MoleculesA drug already approved for another condition but identified as potentially beneficial for individuals with FOXP1 syndrome.
There is a faster path to getting these medications to our children either through off-label prescribing or indication expansion through the FDA.
Novel Small MoleculeA small molecule that has been identified as potentially beneficial for individuals with FOXP1 syndrome but has not yet been approved for any medical indication.
Developing such drugs is a more complex and time-consuming process than repurposing existing ones, as it requires full preclinical testing and clinical trials to establish safety and efficacy.
ASO upregulation
Antisense oligonucleotides, or ASOs, are tiny pieces of genetic “helpers” that can change how a cell reads instructions for making a protein. In upregulation therapy, the ASO helps the cell make more of a protein that’s missing or too low.
Gene therapy
Gene therapy is a way to give cells a healthy copy of a gene so they can make the right protein and work the way they should. A modified virus is used as a “delivery vehicle” to carry the new gene into the body’s cells. Once inside, the cells use this fresh set of instructions to start making the missing or corrected protein.
Gene editing
Gene editing directly repairs the body’s own DNA. Instead of adding a new gene, tools like CRISPR act like molecular scissors—finding the exact spot in the gene that’s not working and fixing it—so the cell can make the correct protein on its own.
Although gene editing has recently been used to treat a small number of diseases, it has not yet been successfully used to treat neurological conditions in humans. We are closely monitoring advances in this rapidly developing field and building relationships with leading researchers to explore whether these technologies could one day be applied to FOXP1 syndrome.