n-Lorem has industrialized the process to scale up to meet substantial demand with major efforts underway to streamline all aspects of its process
n-Lorem is already delivering on its promises to provide individualized experimental ASOs and with additional funding can significantly increase the number of patients to be treated in 2023
SAN DIEGO, Calif., Mar. 16, 2023, (Business Wire) n-Lorem, a nonprofit Foundation, celebrates three years of progress in discovering, developing and providing experimental antisense oligonucleotide (ASO) medicines to nano-rare patients (1 to 30 patients with the same single gene mutation worldwide) for free for life.
Learn more about the exciting progress n-Lorem has made by reading Stan Crooke’s annual letter and visit n-Lorem’s new website.
“What n-Lorem is doing is extraordinary and unique. There are no treatment options for nano-rare patients, many of whom will progress and succumb to their disease without therapeutic intervention. n-Lorem’s mission is so critical to these patients, their families and to the health care system, which has thus far underserved this patient population,” said Michael Hayden, MBChB, Ph.D., Killam Professor of Medical Genetics at University of British Columbia and member of n-Lorem Board of Directors. “I am honored to be a part of this mission and to support the great work that the team at n-Lorem is doing every day to return hopes and dreams to families, one family, one nano-rare patient at a time.”
“Our progress in three years is astounding and is evident in all facets of the Foundation. What n-Lorem is doing has never even been considered possible until the technology developed under my leadership was invented, advanced and validated. Industrializing the process ensures that all steps are undertaken with the highest quality and assures that each patient treated is only treated with an optimal ASO,” said Stanley T. Crooke, M.D., Ph.D., Founder, chairman and CEO, n-Lorem Foundation.
Since inception n-Lorem has:
- Received and reviewed over 160 applications and accepted more than 80 patients;
- Filed four INDs in late 2022 and began treatment on three new patients by the end of the year. The fourth patient to be treated soon;
- Established a network of more than 30 partners that provide support in all areas of drug discovery, development and manufacturing;
- Received U.S. Food and Drug Administration (FDA) guidances that provide a supportive regulatory environment for ASO treatment of nano-rare patients;
- Industrialized the process of discovering, developing and providing only optimal ASOs to n-Lorem patients;
- Launched its Patient Empowerment Program that includes an educational podcast with more than 20 episodes released;
- Built its own laboratory space that has more than tripled the Foundation’s drug discovery efforts;
- Shared learnings broadly in the community through presentations and publications;
- Reduced per-patient costs of drug discovery, development and manufacturing; and
- Built out its leadership team to support this rapid growth.
“Though I am encouraged by the support we have received to date, the demand has outstripped all expectations and the funds we have been able to raise,” concluded Dr. Crooke. “Our immediate challenge is to raise sufficient funds to expand and begin to meet today’s demand, then show that our model is sustainable. These are formidable challenges, but we know that n-Lorem is the best hope for these unserved patients.”
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 needy patients and families.
- Subscribe to n-Lorem’s Patient Empowerment Program, a podcast series for nano-rare patients
- Watch: n-Lorem Foundation: Offering Hope and Help to Nano-rare Patients
- Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
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 has assisted in the development and treatment of 14 nano-rare patients and received over 160 applications for treatment with more than 80 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.
Amy Williford, Ph.D.
Director of Communications