From a drug discovery point of view, scientists need to see who they are fighting for and it is heartening to see the commitment they have to save a child’s life. Another speaker at the rare disease symposium, Dr. Guangping Gao from the University of Massachusetts Medical School showcased groundbreaking research on viral vectors of gene therapy. Apart from the brilliant science, he spoke with deep passion on getting to know patients with Canavan’s disease, another rare pediatric disease that is fatal. He truly believes that he can make a difference and save children afflicted with this deadly condition and his belief is inspirational to other drug discovery researchers. There are several examples of biotech companies that have been started by parents of children afflicted with rare disease – one example is Solid Biosciences which focuses on finding a cure for Duchenne’s muscular dystrophy. The company’s CEO, a former banker, founded the company in 2013 and the company recently raised $50 million in Series C funding to continue developing gene therapy for Duchenne’s muscular dystrophy. The goal of the company clearly goes beyond operating incomes and profits – they want a cure.
The future of rare disease drug discovery is looking bright primarily due to the strong voice from caregivers who actively start foundations to raise funding for cutting-edge research and improved clinical care. The momentum from patient families and foundations has not gone unnoticed by funding agencies and regulatory bodies and perhaps we can look forward to the day when a child diagnosed with any rare disease will have hope to win the battle.