SAN DIEGO– n-Lorem, a nonprofit foundation, today noted important new guidance recently issued by the U.S. Food and Drug Administration (FDA) regarding both the manufacturing processes and stability testing required for antisense oligonucleotides (ASOs) to be administered to nano-rare patients and the clinical administration of ASO therapeutics for nano-rare patients. The guidance titled “IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations” discusses the clinical development requirements for developing ASO therapeutics for patients with unique gene variants, making them a nano-rare patient population. n-Lorem is the only non-profit foundation using a robust and proven ASO technology to discover and provide experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life.
“The FDA has now issued guidance defining preclinical, manufacturing and clinical administration of ASOs in nano-rare patients. These documents support the importance of ASO technology as an advanced and well-studied technology capable of meeting the needs of nano-rare patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “Operating in a regulatory environment in which clear preclinical, manufacturing and clinical regulatory guidance is available supports our efforts to discover, develop and provide free ASO medicines to patients for free, for life. Because nano-rare patients are often advanced in their disease upon diagnosis, efficient discovery efforts in these patients are critical. These guidances support our efforts to move as quickly as possible while assuring as optimal an ASO as possible and exposing patients to only prudent risks. We look forward to continuing to work with the FDA and other regulatory agencies in other jurisdictions to assure that there is a clear process that we can follow to bring experimental ASO treatments to these patients in such desperate need.”
“n-Lorem is blazing new ground never thought possible in efforts to help those patients with nano-rare diseases. The collaborative approach taken at the FDA for experimental ASO treatment is encouraging and will go a long way to supporting n-Lorem’s efforts to efficiently bring ASO treatments to nano-rare patients while prudently managing risks,” said Frank Sasinowski, J.D., M.P.H., M.S., n-Lorem Board of Directors.
