CARLSBAD, Calif., Mar 19, 2020 /PRNewswire/ — The n-Lorem Foundation announced today that the Korea Institute of Toxicology (KIT) has become the latest institute to join in support of the n-Lorem Foundation’s efforts to provide advanced, experimental RNA- targeted medicines free of charge for life to patients living with ultra-rare diseases. KIT, a world-class, government-funded research institute that evaluates the safety of medicine, bio-related products and chemicals, will provide support for a substantial amount of pre-clinical toxicity studies for n-Lorem. Toxicology studies are the most costly component in the process of providing experimental ASO therapies to patients with ultra-rare diseases.
“We are honored to have the opportunity to join the n-Lorem Foundation as a global partner. We truly hope that our contribution can serve as a steppingstone to allow the development of an effective treatment for individual patients with rare genetic diseases,” said Dr. Chang-Woo Song, president of the Korea Institute of Toxicology.”
“Although patients with extremely rare genetic diseases, such as those with only 1-to- 10 patients worldwide, present significant challenges to traditional treatment approaches, antisense oligonucleotides (ASOs) provide an opportunity to efficiently create genetic medicines designed to correct specific genetic defects,” said Dr. Stanley T. Crooke, chairman and CEO of n-Lorem Foundation. “The support that the Korean Institute of Toxicology has committed to the n-Lorem Foundation is deeply appreciated and will have a tremendous impact on the work we are doing for patients. KIT’s generous contribution adds to those from Ionis, Biogen and many others. Together and working to leverage the power of modern genomics and the antisense platform created by Ionis, we can responsibly meet the needs of some of these patients.”
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 Tracy.Johnson@nlorem.org.
SOURCE n-Lorem Foundation