Foundation to custom design and provide “n-of 1” experimental ASO medicines to patients with extremely rare diseases for free
CARLSBAD, Calif., Jan. 8, 2020 /PRNewswire/ — The n-Lorem Foundation announced today its official launch as a nonprofit organization established to provide advanced, experimental RNA-targeted medicines free of charge for life to patients living with ultra-rare diseases. The n-Lorem Foundation is being established with an initial investment by founders Drs. Stanley and Rosanne Crooke. Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Biogen Inc. (Nasdaq: BIIB) are providing additional funding as the first corporate donors, along with support from individual contributors. The Foundation will be headed by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO and current executive chairman of the board at Ionis Pharmaceuticals. Dr. Crooke founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics.
“Patients who suffer from extremely rare genetic diseases, that is a total world-wide patient population of 1-10 or so patients, present many challenges to traditional approaches to diagnosis and treatment. Fortunately, advances in genomics today facilitate the potential to identify the molecular causes of these diseases. Genetic diagnosis, combined with advances in a new platform, antisense oligonucleotides (ASOs), provide an opportunity to efficiently create genetic medicines that are designed to correct these genetic defects. The n-Lorem Foundation is designed to couple advanced genomic diagnostics with antisense technology to charitably meet the therapeutic needs of these unique patients,” said Dr. Stanley T. Crooke, chairman and CEO of n-Lorem Foundation. “No single charitable foundation or drug discovery technology can meet the needs of all the patients who suffer from ultra-rare diseases, but having led the creation of the antisense platform, I believe that by bringing together the power of modern genomics and the antisense platform created by Ionis, we can responsibly meet the needs of some of these patients. Today is a beginning. I am deeply appreciative of contributions from Ionis, Biogen and the many others who have worked with me to begin this important effort. We look forward to keeping you informed about our progress.”
The n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients with ultra-rare diseases (1 to 10 patients) that are the result of a single genetic defect unique to only one or very few individuals. ASOs are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO and current executive chairman of the board of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics.
To learn more about the n-Lorem Foundation, visit nlorem.org. If you are interested in supporting the efforts of the n-Lorem Foundation, please contact Tracy Johnson at 760-552-7113 or [email protected].
SOURCE n-Lorem Foundation
Tracy Johnson, Executive Director