SAN DIEGO – The editors of Nature Biotechnology recently published an editorial entitled “Ultra-precision medicine” in the June 2021 issue of Nature Biotechnology[i], highlighting new initiatives in n-of-1 drug development, as well as the rapid progress that n-Lorem Foundation, a nonprofit organization providing the ultra-rare disease community with free, lifetime supplies of individualized RNA-targeted medicines, has made since opening its doors in 2020.
The editorial underscores key points from n-Lorem Founder and CEO Dr. Stanley T. Crooke’s commentary recently published in Nature Biotechnology, “A call to arms against ultra-rare diseases[ii],” which details the extraordinary progress and model the foundation has created to serve patients with ultra-rare mutations through the development of individualized antisense oligonucleotide (ASO) treatments.
“We are very appreciative of Nature Biotechnology for their support in raising awareness within the scientific and medical communities about our efforts to create a non-profit model that can be scaled to meet the needs of many of these desperate patients with ultra-rare diseases,” said Dr. Stanley T. Crooke, Founder, CEO and Chairman, n-Lorem Foundation. “The extreme rarity (frequently a single patient) of these diseases poses extraordinary challenges that make treatment using a traditional commercial model extremely difficult. Today, n-Lorem and ASO technology represent the only immediate treatment opportunity for many of these patients. At n-Lorem, we are committed to collaborating with all stakeholders to maximize the benefit to patients and assure that the entire community benefits from what is learned. My hope is that many others will join us in this ‘call to arms’ to bring the immediate hope, rapid treatment and the power of ASO technology to bear for ‘n-of 1’ patients in need.”
The Nature Biotechnology editorial outlines several important points around n-Lorem’s progress and n-of-1 drug development initiatives, including the following direct excerpts:
- ‘Personalized medicine’ promised drugs tailored to individuals on the basis of their genetic makeup. While that never materialized, its scion ‘precision medicine’ offered drug development for groups of genetically stratified patients sufficiently large to generate a return on investment. For the millions of others who suffer from ultra-rare disease— conditions with a prevalence of <1 case per 50 000 population — most of commercial drug development simply forgot them. Now that situation looks set to change. [i]
- n-Lorem [is] an ambitious not-for-profit initiative with a mission to discover individualized experimental antisense oligonucleotide (ASO) treatments for patients with ultra-rare conditions arising from a single, often de novo, mutation (e.g., an indel, repeat or single point mutation). Since its founding last year, n-Lorem has built a network of public–private partnerships with Ionis Pharmaceuticals, Biogen, Ultragenyx, Charles River Labs, Covance, the Korea Institute of Toxicology and the US National Institutes of Health’s Undiagnosed Disease Network to discover and develop individualized ASO drugs collaboratively with clinical investigators in academic medical centers. The effort aims not only to register new ASO drugs for ultra-rare conditions, but also to supply these patients with drugs, free of charge for their entire lifetime. [i]
- ASO drugs are the modality being developed at n-Lorem. Their relatively mature and standardized chemistry, modular gapmer structure, specificity against a patient’s unique mutation, and ~10–12 month development time to prepare for first-in-human testing make them ideal candidates for individualized therapy. What’s more, they are cheap to make; 10 g of ASO is sufficient to treat a patient for life. [i]
- Progress has already been rapid. Since opening its doors, n-Lorem has already received 75 applications, accepted 16 and initiated 7 patient programs. That is 16 individuals who now can dare to hope.[i]
The editorial reflects several additional important factors in the ultra-precision drug development landscape, including legislative and regulatory considerations, clinical research, trial design and natural disease history factors and more. Learn more and read his full Nature Biotechnology editorial, “Ultra-precision medicine” here.
Click here to access Dr. Crooke’s Nature Biotechnology commentary, “A call to arms against ultra-rare diseases.” For more on n-Lorem’s mission, visit www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these desperate patients and families.
- Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Ultra-Rare Disease
- Watch: n-Lorem Foundation: Creating a Better Future for Ultra-Rare, One Patient at a Time
- Watch: n-Lorem Foundation’s 1st year of Progress
About n-Lorem Foundation
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 patients with ultra-rare diseases (1 to 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. ASOs 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. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO, and former executive chairman of the board 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. To learn more about the n-Lorem Foundation, visit www.nlorem.org, and follow us on Twitter, Facebook, LinkedIn and YouTube.
[i] Ultra-precision medicine. Nat Biotechnol (2021). https://doi.org/10.1038/s41587-021-00967-8
[ii] Crooke, S.T. A call to arms against ultra-rare diseases. Nat Biotechnol (2021). https://doi.org/10.1038/s41587-021-00945-0
Tracy Johnson, Executive Director