n-Lorem Foundation Marks 5 Years of Hope for Nano-rare Patients
The n-Lorem Foundation, a pioneering nonprofit organization, is celebrating its fifth anniversary of delivering life-changing personalized experimental treatments to nano-rare patients—completely free of charge, for life. Founded with a bold vision to help patients suffering from ultra-rare genetic diseases, n-Lorem has proven that a nonprofit model can successfully bring individualized therapies to those who have long been overlooked by traditional drug development.
In its first five years, n-Lorem has treated more than 30 nano-rare patients using personalized antisense oligonucleotide (ASO) therapies. These experimental medicines are tailored to each patient’s unique genetic mutation, often in cases where no other treatment exists. The foundation’s work has already demonstrated that this patient-focused approach can produce significant clinical benefits—even in patients with severe, multi-system diseases—while maintaining an exceptional safety and tolerability profile.
“I am proud that, through n-Lorem, we’ve been able to bring real hope and therapeutic options to patients who had none,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman, and CEO of the n-Lorem Foundation. “The positive outcomes we’re seeing—across different genes and multiple organ systems—demonstrate not only the power of antisense technology, but also the potential of mutation-specific therapies to transform lives.”
To date, n-Lorem has received over 330 applications from patients and families seeking help. Of these, the foundation has accepted more than 160 for potential treatment, a testament to the growing demand within the nano-rare disease community. All investigational treatments are discovered and developed entirely in-house, with the foundation securing authorization to proceed on over 25 Investigational New Drug (IND) applications from four divisions of the U.S. Food and Drug Administration (FDA).
Despite the challenges of operating as a nonprofit in a traditionally for-profit industry, n-Lorem has built a sustainable and efficient model for developing individualized ASO therapies. According to Dr. Crooke, the foundation’s ability to industrialize its internal processes has been key to its success and will be crucial in scaling up to meet increasing demand.
“We’ve been incredibly fortunate to receive support from many sectors—philanthropy, biotech, and beyond,” he said. “But the need is growing faster than we ever expected. We now have the foundation in place to meet this growing demand, but we can’t do it alone. We invite others—scientists, donors, and advocates—to join us in this mission. Together, we can make a difference for thousands of patients, one life at a time.”
A major reason for the surge in demand is the increased accessibility of genomic sequencing, which is enabling more accurate and earlier diagnoses of rare and nano-rare conditions. As more patients are identified through genetic testing, the need for targeted, personalized therapies will continue to rise.
“Genomic testing is becoming more common, but for many families, it’s still considered a last resort,” said Dr. Crooke. “As this technology becomes more widespread, we anticipate even more applications for help—and we are preparing to meet that need.”
Beyond its scientific work, n-Lorem has also created a robust support network for nano-rare patients and their families. Through its Patient Empowerment Program, the foundation provides education, resources, and a sense of community to those facing the unique challenges of living with a nano-rare disease. Its efforts include an informative website, a regular podcast series, and the annual Nano-rare Patient Colloquium—a gathering that brings together patients, caregivers, physicians, and supporters to learn, share, and connect.
“We didn’t expect the need for treatment to be so overwhelming, or the necessity to expand our discovery and development operations so quickly,” Dr. Crooke added. “That we’ve been able to grow, adapt, and deliver meaningful outcomes on a nonprofit budget is extraordinary. But the most rewarding part is seeing the improvements in our patients’ lives—more than we could have ever hoped for.”
As it looks to the future, n-Lorem remains committed to its founding mission: delivering personalized, mutation-specific treatments to nano-rare patients who have no other therapeutic options. Through innovation, collaboration, and compassion, the foundation continues to change what’s possible for some of the most vulnerable people in medicine.
