First-of-its-kind perspective from the n-Lorem Foundation provides a very broad review of n-Lorem’s experience in more than 300 patient applications and reports excellent safety profile and meaningful clinical benefit in treated patients receiving individualized ASO therapies

n-Lorem, a nonprofit foundation that discovers, develops and provides personalized experimental antisense oligonucleotide (ASO) medicines to patients with nano-rare mutations (1-30 known worldwide) for free, for life, announced today the publication in Nucleic Acids Research [https://doi.org/10.1093/nar/gkag504] that details its experience with the first 329 nano-rare patient applications for treatment and a summary of the results to date in nano-rare patients treated with experimental ASOs medicines provided by n-Lorem.

n-Lorem has created the largest safety database of nano-rare patients treated that includes more than 50 nano-rare patients treated with bespoke ASO medicines discovered and developed at n-Lorem, more than 300 doses administered across all routes of administration (subcutaneous, intravitreal, intrathecal), and more than 55 patient years of safety data. Amongst treated patients, one patient has been treated for longer than three years, six patients for longer than two years and 26 patients for more than one year. In all treated patients, n-Lorem has observed an excellent safety and tolerability profile with no ASO-related serious adverse events.

n-Lorem also highlighted the meaningful impact of its ASO medicines on individual patients. n-Lorem is observing improvements observed in treated nano-rare patients that include, significant reductions in seizures, substantial improvements in control of movements, reduction of neuropathic pain, improved autistic symptoms, recovery of development delays that were lost and acquisition of new skills such as walking independently, improved quality of life based on pre-defined endpoints and standard measures, stabilization of disease in ALS patients, halting of vision loss, and halting loss of kidney function.

“At n-lorem we are treating more than 50 nano-rare patients for free, for life and adding more patients every month. The systems and processes we have developed and the broad expertise in ASO technology has meant that we can meet our commitment to treat our patients with safe and effective ASOs,” said Stanley T. Crooke, Founder, Chairman and CEO of n-Lorem. “We are also thrilled with the benefits we are observing in nano-rare patients with disorders that manifest in the CNS, retina, and the kidney. Many of our patients begin treatment with very advanced diseases, many that have missed key developmental milestones, yet we continue to observe significant improvements in these patients, some responding even at very low doses. These observations demonstrate that many developmental delays can be recovered even a decade or more after the normal age of achievement of the milestone. Moreover, we show that patients can achieve milestones never achieved.”

The Nucleic Acids Research publication [https://doi.org/10.1093/nar/gkag504] titled “Addressing the Needs of Nano-rare Patients: the n-Lorem Experience” provides a comprehensive overview of the processes and systems that n-Lorem has industrialized that introduce efficiencies across the entire organization, highly sophisticated design algorithms that are a product of nearly 40 years of experience in creating, validating and advancing the technology, and scaling to meet a larger demand while maintaining high-quality standards. These processes include:

• Patient Selection and Assessment: Applications are rigorously reviewed for suitability, including genotype and phenotype analysis, organ-specific expertise and detailed clinical goals. Additional work is often needed to confirm that mutations are causative and to determine allele-selectivity requirements.
• ASO Design and Development: ASOs are rationally designed and extensively screened in patient-derived cells to ensure potency, selectivity and safety. For allele-selective and splicing-targeted ASOs, advanced sequencing and in vitro modeling are employed.
  • In this manuscript, the importance of n-Lorem’s systematic preclinical evaluation and screening industrialized processes contributes to the ability to identify only optimal ASOs for nano-rare patients. (Refer to Table 1)
• Clinical Trials and Safety Monitoring: Modified single-patient crossover designs are used due to disease severity and ASO durability. Extensive safety assessments and quarterly reviews by a Data Safety Monitoring Board ensure ongoing patient protection.

“The success of n-Lorem is a product of effectively creating an organization in which broad and deep knowledge of ASO technology is coupled to industrialized systems and deep experience in drug discovery and development and the management of patients being treated with experimental medicines and a deep commitment to treating patients safely,” continued Dr. Crooke.

Since launching in 2020, demand for n-Lorem’s bespoke medicines has far exceeded expectations, with neurological disorders comprising the majority of its programs, however n-Lorem has patients with retinal disease and amyloid kidney disease on treatment and applications span diverse age groups and genetic variants.

“At n-Lorem we are blazing new ground, we are gaining ever more important insights into the nature of extremely rare mutations, the phenotypes they may produce, how these rare diseases may progress and the impact of ASO treatments on patients with pathogenic rare mutations,” concluded Dr. Crooke. “Our patients also provide a unique opportunity to learn about health and disease altogether, because each of our patients has a single, identifiable variable - a causative mutation - and I am optimistic that over time the lessons we learn will change the way we think about health and disease altogether.”

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 440 applications for treatment with more than 240 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 X, Facebook, LinkedIn and YouTube.

To 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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We are treating more than 50 nano-rare patients for free, for life—and adding more patients every month.