Healx is a social enterprise focused on identifying next-generation drug repositioning opportunities for rare and orphan diseases. Our unique model places patient groups and charities central in the development process. We believe in working hand in hand with them to develop a cure together. Healx uses state-of-the-art technology from Cambridge University. The combination of machine learning with advanced ‘Omic analytics allows us to identify hidden links between drugs and diseases. This approach selects drug repositioning candidates with a much higher probability of success, which has been validated in a number of different disease areas. Visit the Healx website.
Horizon Discovery combines deep scientific experience in translational research with a precision gene-editing platform incorporating rAAV, CRISPR and ZFN technologies. Horizon supplies genetically-defined cell lines, in vivo models, custom cell line generation, molecular reference standards, and contract research services to over 1,000 academic, clinical and biopharmaceutical organizations
Sobi is a leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients and their families. Their research and product portfolio is primarily focused on haemophilia, inflammation and genetic and metabolic diseases. They market and make available across Europe, the Middle East, North Africa and Russia a portfolio of specialty and rare disease products for partner companies. They have world-class capabilities in protein biochemistry and biologics manufacturing development – validated by leading industry partners and manufacture the drug substance for the haemophilia treatment ReFacto AF/Xyntha® for the global market
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Patients with these life-threatening diseases often have no effective treatment options, and they and their families suffer with little hope. Our goal is to deliver medical breakthroughs where none currently exist. We are driven because we know people’s lives depend on our work.
As we continue to expand our operations into additional countries, Alexion is advancing the most robust rare disease pipeline in the biotech industry, with highly innovative product candidates in multiple therapeutic areas. Alexion is establishing a premier global metabolic rare disease franchise with two potential therapies in late-stage development for hypophosphatasia (HPP) and Lysosomal Acid Lipase Deficiency (LAL Deficiency, or LAL-D). In addition, as the global leader in complement inhibition, we are strengthening and broadening our portfolio of complement inhibitors across diverse platforms.
Illuminating Pathways – Changing Lives
Raptor Pharmaceuticals (NASDAQ: RPTP) is a commercial-stage, global biopharmaceutical company committed to developing and commercializing life-altering therapeutics for orphan diseases. Our focus is to help patients with rare, debilitating, and potentially fatal diseases by leveraging our deep understanding of cellular metabolic pathways to develop medicines that address serious unmet medical needs.
MRC Technology is an independent life science medical research charity committed to improving positive patient outcomes everywhere.
As a champion for human health, MRC Technology partners with academic, biotechnology, pharmaceutical and charity organisations to move promising medical research forward into viable and accessible patient treatments.
As a self-funded charity, income is reinvested to support translating research from the bench to patient. MRC Technology offers commercialisation and IP management skills and diagnostic and drug discovery expertise, specialising in small molecules and therapeutic antibodies.
MRC Technology projects have led to several approved drugs, changing the lives of countless patients by harnessing the potential of science. www.mrctechnology.org.
Shire is now the leading global biotechnology company focused on serving patients with rare diseases and other highly specialized conditions. These diseases are often misunderstood, undiagnosed and life-threatening. Our 22,000 employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.