Patients and families with genetic diseases that afflict one or very few patients in the world pose unique challenges to the healthcare industry. It is gratifying that the National Institutes of Health (NIH) and other institutions in the U.S. have begun to address identifying such patients and harnessing the power of modern genomics to diagnose and identify the molecular causes of these diseases. In fact, the Undiagnosed Disease Network, a consortium of medical schools, has led the way, but other medical schools not involved in the UDN are active as well.
With the efficiency of our antisense oligonucleotide (ASO) technology at Ionis, we can and are commercially addressing diseases that range from a thousand to many millions of patients, but I do not believe that an n of 1-10 patients can be treated using a commercial model.
So, for about two years, I conceived and crafted the non-profit n-Lorem Foundation, a charitable organization with a mission to apply the efficiency, versatility, and specificity of antisense technology to charitably provide experimental antisense oligonucleotides (ASO) medicines to treat patients with ultra-rare diseases. I have developed relationships with the UDN and worked with the U.S. Food and Drug Administration to make n-Lorem a reality. In the meantime, we provided advice and support to help four patients obtain treatment with experimental ASOs. We announced the launch of n-Lorem in January, with initial funding from Ionis, Biogen and Rosanne and me.
Progress since the launch has been gratifying. We are moving toward treating several patients and the response from the industry and potential donors has been wonderful. If you would like to learn more, please contact Tracy Johnson, executive director of the n-Lorem Foundation, tracy.johnson@nlorem.org.