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n-Lorem Foundation Notes Latest in Guidance From FDA on Developing ASO Drug Products for Patients With Nano-rare Diseases

Guidance issued by FDA provides a supportive environment to develop experimental ASO medicines for patients with nano-rare disease
SAN DIEGO, Calif., January 6, 2022 (Business Wire) n-Lorem a non-profit 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.”

“I am honored to be joining the n-Lorem Advisory Council as its Chair, and look forward to working with like-minded advisors to support n-Lorem’s mission to bring experimental therapies to nano-rare patients,” said John Maraganore, Ph.D. “I feel that the patients served by n-Lorem are in desperate need of the best from all of us. I am honored to work again with Stan in achieving a high-quality and scalable approach for the nano-rare patient population. I am committed to do all that I can to enable n-Lorem to treat even more patients.”

“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.

Learn more about n-Lorem’s mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

  • 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

About n-Lorem

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 nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which 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. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 40 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO 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 TwitterFacebookLinkedIn and YouTube.

n-Lorem contact: 

Amy Williford, Ph.D.
Director of Communications
amy.williford@nlorem.org