Treatment Research
MAPK8IP3 ASO
In 2025, The n-Lorem Foundation began treatment with an antisense oligonucleotide (ASO) medicine it discovered to target the MAPK8IP3 gene for a n-of-1 Clinical Trial. The trial is ongoing.
The n-Lorem Foundation exists to serve the nano-rare patient community. n-Lorem focuses on patients who have diseases caused by a single gene mutation that is found in approximately 30 or fewer patients in the world. Due to their extreme rarity, n-Lorem refers to these patients as nano-rare. n-Lorem chose this patient population because today there is no path to commercial approval or commercial model that can address the needs of such rare patients and because n-Lorem hopes that they can raise sufficient donations to treat as many nano-rare patients as they are able to. Already the need has exceeded their expectations.
The global scale of the nano-rare disease challenge is substantial. n-Lorem can do the seemingly impossible task of discovering, developing and providing a personalized, experimental ASO medicine that has been optimized to work for that individual patient. As a non-profit, n-Lorem completes this work for free and commits to providing free drug for the life of the patient. Within its first five years of operation, n-Lorem has demonstrated that its model works for nano-rare patients and that it is feasible to meet the needs for underserved patient population. As of March 2025, n-Lorem had accepted more than 160 patient programs, filed more than 25 investigational new drug applications (INDs) with the US FDA and treated more than 30 patients.
n-Lorem integrates a powerful, patient-driven mission and a deep understanding in ASO drug discovery and development with a robust, versatile technology that enables the discovery and development of optimized personalized experimental ASO medicines for nano-rare patients. n-Lorem has brought this mission to fruition through the establishment of an industrialized process that enables scalable, rapid, high-quality and cost-effective drug discovery and development and professional management of patients on treatment. As a non-profit, every added efficiency in the processes or new advancement by the n-Lorem team results in the Foundation’s ability to help more nano-rare patients.
Traditional drug discovery and development of a truly novel new medicine requires decades of time and billions of dollars. As such, it is unfeasible from both a time and cost perspective to conduct a traditional drug discovery approach for a personalized medicine that may only benefit a few patients. ASO technology directly targets the genetic cause of disease, is a proven drug discovery platform and offers a unique avenue to substantially decrease the cost and time needed to create a new medicine. In order to bring this idea to reality, n-Lorem worked closely with the US FDA to establish a path forward for the treatment of nano-rare patients. The combination of the efficiency, cost effectiveness and versatility of its ASO technology, plus the FDA’s guidance for only ASO technology that enables n-Lorem to mount a new drug discovery program to patient treatment in an average of 15-18 months Today, n-Lorem estimates that to discover, develop and manufacture drug product for the life of a patient is a bit more than $1 million. Most of that cost comes from studies done in animals to test an ASO’s safety. n-Lorem continues to identify ways in which the cost of each program can be reduced through its network of leading CROs and CMOs who share n-Lorem’s mission to help the nano-rare. n-Lorem does not do this work alone and has tapped into the expertise of leaders across the academic, healthcare, regulatory and pharmaceutical spaces. n-Lorem has established expert committees that provide expert advice throughout the entire patient process.
The challenges presented by nano-rare patients are far too complex for n-Lorem to solve even a fraction of the issues without funding support. Because of the rarity of this patient population, commercial efforts for drug discovery and development would not be feasible leaving the only viable solution a non-profit solution. n-Lorem has demonstrated that its non-profit processes work, its ASOs are the most optimized ASOs that can be made today and that patients who were once without hope have hope for a healthier future. While the mission has supported n-Lorem to raise sufficient funds to be successful to date, demand from the nano-rare patient community continues to outstrip n-Lorem’s ability to fundraise. With sufficient funding, n-Lorem could easily scale its operations in and outside of the US, enabling more nano-rare patients to benefit from its mission.
Nearly every new patient represents a novel ASO discovery and development program. The essence of science is uncertainty and the process of discovering a novel medicine is complex and arduous, even for ASOs. At n-Lorem, they have the expertise and experience coupled with a highly industrialized process that exceeds the productivity of even large pharmaceutical companies to bring hope and potential help to nano-rare patients. They believe that each patient deserves the very best ASO that they can discover, develop and provide to nano-rare patients for free, for life.
It is vitally important for n-Lorem to work with scientists and physicians to assure that patients are exposed to only optimized personalized ASO medicines. n-Lorem is lucky to have world-class renowned physicians supporting their patients and helping at every stage of the program’s development. If you would like to learn more about n-Lorem and help by supporting its mission, please click here.
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