New FDA guidance enhances n-Lorem’s ability to provide experimental antisense oligonucleotide (ASO) treatments to help desperate patients with ultra-rare mutations
Foundation grateful to FDA for expeditiously providing detailed and important guidance for new treatment approach
SAN DIEGO, Calif., May 3, 2021 (Business Wire) — n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients with ultra-rare diseases, today applauded important new guidance issued by the U.S. Food and Drug Administration (FDA) regarding the pre-clinical studies required for antisense oligonucleotide (ASO) treatment of patients with ultra-rare diseases. The constructive guidance is a positive and critical next step in meeting the needs of patients with ultra-rare mutations, and confirms the FDA’s stance that ASO technology is capable of helping “n-of 1” patients and is sufficiently advanced and understood to be subject to limited and expedited preclinical evaluation.
n-Lorem Foundation is the first and only charitable organization with a mission to provide personalized and potentially life-saving treatments to patients who have diseases caused by extremely unique mutations (1 to 30 patients worldwide) for free, for life. By accessing the ASO technology developed at Ionis Pharmaceuticals, n-Lorem helps qualifying patients receive individualized ASO treatments for their unique condition.
“At n-Lorem, we are deeply appreciative that the FDA has so rapidly provided clear and constructive guidance regarding the preclinical studies necessary to support treatment of patients with ultra-rare diseases with experimental personalized ASOs,” said Dr. Stanley T. Crooke, Founder, CEO and Chairman, n-Lorem Foundation. “We agree with the Agency that only ASOs based on chemical classes for which there is substantial experience should be developed under this guidance.”
The FDA’s guidance is significant, due to the nature of n-Lorem’s unique treatment approach, wherein the foundation works with a patients’ treating physician (sponsor investigator) to submit an investigator-sponsored IND to the FDA for an ASO medicine to treat a patient who has a severely debilitating, ultra-rare condition. The guidance is specific, clear and consistent with proposals made by n-Lorem, and will greatly facilitate rapid and efficient treatment of these needy patients.
Dr. Crooke added, “We believe that only when all stakeholders, including the FDA, work together collaboratively, can we maximize the number of patients that will benefit from experimental ASO treatment. We are grateful to the FDA for its support of efforts to help these patients and look forward to continuing to work with the Agency to assure the most streamlined process that is consistent with the creation of the highest quality ASOs possible, is adopted. With more that 25 patients moving toward treatment in our first year of operation, n-Lorem will continue to work with the FDA to efficiently bring ASO treatments to these needy patients while balancing prudent treatment risks. We thank all our donors and collaborators for their support, and are confident that many others will join us in this ‘call to arms’ to bring the immediate hope, rapid treatment and the power of ASO technology to bear for ‘n-of 1’ patients in desperate need.”
Click here to view the FDA guidance, “Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases.”
- Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Ultra-Rare Disease
- Watch: n-Lorem Foundation: Creating a Better Future for Ultra-Rare, One Patient at a Time
- Watch: n-Lorem Foundation’s 1st year of Progress
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 30 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. Follow us on Twitter, Facebook, LinkedIn and YouTube.