FRAXA is a charity which was set up in 1994 by parents whose children were affected by Fragile X. Its primary goal is to address the lack of funding in biomedical research related to the condition.
FRAXA started by investing in basic research into Fragile X. They raised funds from the FRAXA community and gave grants on a competitive basis to researchers. Once the gene and protein responsible for Fragile X were identified, FRAXA recruited neuroscientists to complement the work of the geneticists.
The missing protein was shown to play roles in learning, memory and brain plasticity and quickly became a hot topic in neuroscience (and still is to this day). FRAXA hoped that by knowing the gene and understanding the protein, an obvious drug target would be found. However, clinical trials kept failing and efforts to find a drug were confounded when it became apparent the role of the gene is far more complicated than once thought.
After 22 years of research, FRAXA had taken novel drug discovery routes as far as they could go. The disease was characterised at the molecular level, targets were validated and large scale international clinical trials were run with big pharma companies. None of this resulted in a drug that would make a meaningful difference to patients’ lives. FRAXA started to change its strategy. Instead of relying on academic labs they started using contract research organizations (CROs). But they were also open minded and prepared to try something new. In 2016, a friend of the CEO was in Cambridge and introduced him to Healx.