n-Lorem’s nano-rare patients include adults and pediatrics with involvement in the central nervous system, kidneys and other organs

n-Lorem, a nonprofit foundation, announced today that four investigational new drug (IND) applications have been filed successfully with the FDA and n-Lorem has begun dosing on three nano-rare patients. n-Lorem anticipates dosing the fourth patient in early 2023. Each patient is receiving a personalized, experimental antisense oligonucleotide (ASO) medicine discovered and developed for that single patient. n-Lorem’s mission is to discover, develop and provide personalized ASOs for nano-rare patients (1 to 30 patients worldwide) for free, for life.

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Sally, Luke and Susannah (Photo: Business Wire)

“Today, we continue to deliver on our promise to discover, develop and provide personalized ASO medicines for our nano-rare patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO of n-Lorem Foundation. “Among the three patients treated this fall is Susannah, a beautiful 8 year old girl who suffers from a neurodegenerative nano-rare condition. Susannah is receiving a personalized, experimental ASO discovered and developed for her unique mutation.”

"The joy we get from being Susannah's parents, from watching her laugh and love and grow, is immeasurable. But the pain of having to watch her disease chip away at those hard-won skills is similar in its magnitude,” said Sally Jackson, Susannah’s mom. “We are cautious, to be sure, but newly hopeful that an experimental medicine developed just for Susannah could flip the switch on the progression of the degenerative disease which threatens every day to overtake the progress that our girl might otherwise make. The hope that the gains of the girl will come out ahead in this race is in itself the greatest gift."

“Our nano-rare patients differ in age, some are adults, some are teenagers, and some are infants, and they differ in the types of disorders that alter their lives. However, they all have two traits in common, they all have extraordinarily rare mutations in a single gene that causes severe illness and are in desperate need of treatment,” continued Dr. Crooke.

“At n-Lorem, we are industrializing the treatment of nano-rare patients by assuring that each step, beginning with the decision to treat a patient is of the highest quality possible, that each patient is treated with the optimal ASO, that the clinical exposure of patients to experimental ASO treatments is professionally managed, and we are scaling to meet the enormous need these patients represent. So, while we are thrilled to be treating three new patients, we have received more than 160 applications and approved ASO treatments for more than 80 patients in just 3 years, and we are confident those numbers and the number of patients treated will expand greatly in 2023,” continued Dr. Crooke.

“Nano-rare patients also present a unique opportunity to learn about health and disease, and we are obligated to learn maximally from each patient and our aggregate experience and share our learnings with all interested parties as rapidly as possible. In our first three years, we have created and optimized the process that leads to treatment for these patients and learned a great deal. We have already published peer reviewed manuscripts and many presentations. Next year, we look forward to sharing many new insights in peer reviewed manuscripts and in our first annual meeting for all interested parties,” concluded Dr. Crooke.

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.

• Subscribe: n-Lorem Patient Empowerment Program Podcast Series
• Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
• Watch: n-Lorem Foundation: Creating a Better Future for Nano-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 160 applications for treatment with more than 80 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 Twitter, Facebook, LinkedIn, YouTube and Instagram.

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