There are approximately 8,000 different types of rare diseases, and it is estimated that over 350 million people worldwide suffer from a rare disease. Yet, 95 percent of rare diseases have not one single FDA approved treatment. In fact, rare diseases is a large medical challenge and a serious public health problem. There is a lack of financial and market incentives to cure rare diseases, and only 326 new drugs were approved by the FDA and brought to market for rare diseases during the first 25 years of the Orphan Drug Act.
The Cambridge, UK-based startup Healx is determined to find new therapeutic solutions for patients with rare diseases by repurposing approved treatments. They combine advanced machine learning and computational biology techniques, together with scientific literature analysis, to identify novel drug application for rare diseases. The startup works closely with patient groups and charities to get a deep understanding of the cause of the disease, and use that information to generate drug candidates with a high probability of success. The most promising candidates are then tested experimentally and prepared for further clinical development, and the startup aim to complete its repurposing studies within six months.
Studies into rare medical conditions have failed to receive adequate funding, and repurposing already approved drugs could deliver new treatments at a fraction of the cost and in a much shorter timeframe. While new drugs usually take several years to get approved and cost billions, Healx’s innovative technology could cut that to months and at a fraction of the cost. Their close work with patients, and their passion to discover new treatments for these patients with few or in many cases no options, is inspirational and something that is seriously lacking in the industry. Healx show how precision medicine can change healthcare, while giving entire patient groups treatments that can significantly increase their quality of life.
Healx has previously been awarded ‘Cambridge Graduate Startup of the Year 2016’ and ‘Life Science Business of the Year 2015’. The startup recently announced they had secured £1.5 million in Series A round from Amadeus Capital, a global technology investor, and renowned healthcare entrepreneur Jonathan Milner. With this investment, Healx hope to expand their technology platform and accelerate their drug review process to find treatments for more devastating diseases.