The n-Lorem Foundation is dedicated to the principle that the individual is the indivisible unit of value. To be optimally fair to the many, we must be fair to the individual. To be generous to the many, we must be generous to the individual. For the human collective to achieve its maximum potential, the individual must have the opportunity to create maximum value. No single charitable organization can make a significant difference in finding a solution to the challenge of enhancing the health of human beings. But, at the n-Lorem Foundation, we have the technology to address a component of this most human challenge, diseases affecting only a few patients in the world.
Challenges of Treating Ultra-Rare Diseases
Patients with ultra-rare diseases present compelling unmet needs. Given the number of patients, the diversity of diseases, and costs of developing medicines for commercial purposes, meeting the needs of these patients through the usual pharma channels is financially impractical. While no single charitable institute can meet the needs of these patients, n-Lorem, in conjunction with the Undiagnosed Diseases Network (UDN), Ionis Pharmaceuticals, and the FDA, can bring hope and benefit to the underserved.
Using Antisense Technology to Treat Genetic Disease
Advances in molecular biology now allow for relatively inexpensive DNA sequencing that supports the identification of the genetic causes of many diseases. Some ultra-rare diseases are caused by a single genetic defect unique to only one or very few individuals. Antisense oligonucleotides (ASOs) are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of ASO drugs is that they can be developed rapidly, inexpensively, and are highly specific. With Ionis Pharmaceuticals, the leader in RNA-targeted therapeutics, backing n-Lorem, custom ASO drugs can be developed on a custom level for patients with ultra-rare genetic diseases.
The Mission of the n-Lorem Foundation
The mission of the n-Lorem Foundation is to apply the efficiency, versatility and specificity of antisense technology to charitably provide investigational ASO drugs to treat patients with ultra-rare diseases (n1-30 patients).
How the Foundation Works
With a seasoned leadership team and strategic partnerships, the n-Lorem Foundation provides the framework and funds for patient identification, ASO drug development, and clinical trial initiation. The UDN, working with Ionis, has already helped to get treatment for a few patients with ultra-rare diseases, demonstrating that the goals of the Foundation are not only feasible but achievable.