n-Lorem Foundation Celebrates Five Years of Bringing Hope and Potential Treatments to Nano-rare Patients

Demand from the nano-rare community has been overwhelming, n-Lorem has filed more than 25 INDs that support the treatment of more than 30 patients

n-Lorem continues to seek additional financial support to expand and advance patient programs more rapidly

n-Lorem, a nonprofit foundation, is celebrating five years of success in establishing a unique non-profit model bringing personalized experimental ASO medicines to nano-rare patients for free, for life. Having treated more than 30 nano-rare patients, n-Lorem has demonstrated that a non-profit model in which patients are accepted irrespective of their financial means is feasible. n-Lorem has shown that the efficiency of ASO technology, coupled to the unique FDA guidance documents for nano-rare patients, can rapidly bring effective treatments to even the most advanced patients, with an excellent safety and tolerability profile, and profound clinical benefit observed in many patients.

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n-Lorem’s ASOs have been administered to more than 30 patients to date. Nearly all evaluable patients have achieved clinically significant benefit, despite the fact that many nano-rare patients are severely affected by their disease. Importantly, the safety and tolerability profile of n-Lorem ASOs is pristine in all treated patients.

“I am proud that, through n-Lorem, we have been able to bring hope and treatment to nano-rare patients today. The benefits we are observing in multiple organs and in multiple genes illustrate the power of antisense and the effectiveness of a mutation-directed therapeutic approach,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO, n-Lorem Foundation.

n-Lorem has received more than 330 applications and accepted more than 160 patients for potential treatment with personalized experimental ASOs discovered and developed completely in-house. n-Lorem has filed and received authorization to proceed for more than 25 investigational new drug (IND) applications with four divisions of the FDA.

“We are fortunate that we have many different types of environments where we can raise support, and we have been extremely successful in raising funding. However, demand from the nano-rare community has been overwhelming,” continued Dr. Crooke. “By industrializing our processes, we are positioned to meet an even larger demand in the future, but we cannot do this alone. We encourage others to learn more and join us in this effort to make a difference in the lives of thousands of patients, one patient at a time.”

Genomic sequencing is still often seen as a last resort for diagnosis. However, as it becomes more accessible, n-Lorem anticipates a growing demand as more patients are identified, which will, in turn, increase the need for personalized treatment options. With its unique position, n-Lorem is well-equipped to develop, discover, and deliver personalized experimental ASO medicines to patients with nano-rare conditions.

“Though we did not expect the extraordinary demand, we have responded by creating an organization that is delivering more than 10 INDs a year and advancing knowledge on nano-rare diseases. We believe that the knowledge gained from these unique patients can lead to an ever deeper understanding of health and disease while bringing hope and treatment to desperate patients and families,” continued Dr. Crooke.

In addition to developing experimental ASOs, n-Lorem has established a community for those affected by nano-rare conditions through its Patient Empowerment Program. Using its website, podcasts, and the annual Nano-rare Patient Colloquium, n-Lorem offers a platform for patients, families, physicians, and supporters to connect, learn, and share knowledge. This vibrant community is united by a common mission: to tackle the unique challenges faced by those with nano-rare diseases.

“What we didn’t expect was the demand for treatment from the nano-rare community to be so high and that we would need to grow our discovery capacity exponentially while also refining our processes. To be successful under those circumstances with limited funding is nothing short of remarkable. That we are seeing improvement in so many of our patients is more than we could have ever hoped for,” concluded Dr. Crooke.

Donations to help support n-Lorem’s ongoing work can be made through the foundation’s website: https://www.nlorem.org/donate/.

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 received over 330 applications for treatment with more than 160 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 and YouTube.

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 patients and families in need.

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