n-Lorem Has Industrialized the Treatment of Nano-rare Patients and Formed Key Committees

n-Lorem, a nonprofit foundation, outlines the steps the Foundation has taken to support the discovery, development and treatment of nano-rare patients (1-30 patients worldwide), for free, for life. By industrializing the process, forming oversight committees, and performing every step in the process with the highest quality, n-Lorem is committed to ensuring that each nano-rare patient is exposed to only prudent risk, has the best, most optimized experimental antisense oligonucleotides (ASO), and is managed professionally throughout the entire process.

"Nano-rare patients deserve to have the best team, the best technology and the best processes in place. At n-Lorem, we strive daily to make the lives of patients better, to help those who have had little hope or help. By industrializing our approach, we can ensure quality is embedded in every process, decision and experimental ASO that we discover and develop," said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO, n-Lorem Foundation. "We are fortunate to have experts across all areas of drug discovery and development engaged in our mission and supporting our efforts by serving on these committees. As we have advanced our patient programs, alongside, we have formed the committees necessary to provide unbiased oversight and counsel to our organization. These committees are important and necessary as we continue to build the highest quality organization we can."

Access To Treatment Committee (ATTC)

The Access to Treatment Committee (ATTC) was established in 2020 to provide an unbiased, thorough evaluation of the risk/benefit of treatment with an experimental ASO medicine for each nano-rare patient application that is submitted to n-Lorem. This committee is comprised of pharmaceutical executives, physicians, academic professors, patient advocates and a bioethicist. The ATTC reviews all applications and approves and prioritizes based on feasibility of developing an ASO treatment, the degree of potential benefit, practicality of treatment, the urgency of treatment, and the availability of physicians and institutions to treat each nano-rare patient.

Study Treatment and Assessment Review (STAR)

The STAR committee was established in 2022 to bring together n-Lorem and experts in clinical trial design, outcome measures and quantitative assessments to provide guidance on treatment goals and clinical outcome measures for each nano-rare patient. Nano-rare patients are unique in their disease and require individual treatment programs that capture the clinical endpoints that will matter the most to the patient and the evaluating physician. To be able to best assess an individual patient program, the STAR committee has experts with extensive clinical trial experience and that represent multiple medical disciplines.

Data Safety Monitoring Board (DSMB)

The DSMB was recently established to assure the continuing safety of nano-rare patients on treatment. The DSMB is comprised of highly recognized independent experts in ASO technology, in conducting and evaluating clinical trials and in drug development. This team of experts will provide safety oversight through interim monitoring of accumulating data. Each nano-rare patient on treatment is a clinical trial of one and the DSMB will be critical to provide an unbiased assessment of each patient and evaluate the appropriateness of the clinical endpoints in the patient's treatment plan.

To see the roster of experts that comprise n-Lorem's committees visit the n-Lorem website: Committees

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.

• 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 received over 200 applications for treatment with more than 90 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.