Foundation received 50 applications from patients with diseases caused by unique mutations seeking personalized treatments, and is moving forward with nearly 20 applicants, greatly exceeding acceptance rate expectations
SAN DIEGO, Calif., Jan. 26, 2021 — n-Lorem Foundation, the first and only nonprofit organization whose mission is to create experimental antisense oligonucleotide (ASO) treatments for patients who have diseases caused by extremely rare mutations (1-30 patients worldwide) for free, for life, celebrates its first anniversary and the extraordinary progress made in 2020 toward its mission of offering immediate hope and rapid treatment to the millions of patients affected by ultra-rare mutations.
In its first 12 months of operation, n-Lorem Foundation received applications to treat 50 patients, is proceeding to treat nearly 20 of those patients and helped two clinical investigators obtain experimental ASO treatments for nine patients – greatly exceeding applicant and acceptance rate expectations. Leveraging the powerful ASO technology developed at Ionis Pharmaceuticals, the foundation has established quality processes so that only appropriate patients are treated with the highest quality, most effective ASO solution. Importantly, this personalized method of treatment can be scaled up to meet the needs of every qualifying patient. The foundation also made significant progress in attracting new donations and broadening awareness to assure that needy patients, and their physicians, know of n-Lorem.
“Though each patient may have a unique mutation, we now know that there are millions of such patients in the world, and that number grows rapidly as we genetically characterize more and more patients,” said Dr. Joseph Gleeson, pediatric neurologist, Rady Children’s Hospital, who is working closely with n-Lorem to help treat some of his patients with ultra-rare conditions. “Because n-Lorem is providing experimental ASOs for free, for life, families can focus solely on the care of their loved ones.”
To meet the needs of as many patients as possible, n-Lorem is developing a broad network of collaborations to assure that n-of-1 patients receive the highest quality experimental ASO treatments as rapidly as possible, at the lowest cost per patient, to ensure every dollar donated is used as efficiently as possible.
“As exciting as our progress is, we know that we are just beginning and that we face significant challenges, but we are in awe of the extraordinary support we have received and the efforts of the FDA to help meet the needs of these patients and families,” said Dr. Stanley Crooke, founder, chairman and CEO of n-Lorem Foundation. “We fully expect that the number of applications will increase significantly as more patients and physicians become aware of n-Lorem, and we look forward to helping treat patients with diseases of the eye, lung, liver and kidney and those with neurological diseases.”
“I felt like I was fighting for so many years for my son, especially in the beginning, and now I’m watching all of these amazing people show up and work on this personalized treatment for one child that could potentially benefit and change the course of his life and our family’s life. Just that is enough – it’s the best feeling, it’s hope,” said Kelley Dalby, mother of Connor, who suffers from an ultra-rare neurodevelopmental disease and is in the process of receiving personalized ASO treatment through n-Lorem.
Given that n-Lorem is blazing new ground never considered as regulations were developed, the foundation greatly appreciates the U.S. Food and Drug Administration’s (FDA) commitment to patients with ultra-rare diseases and the initial guidance focused on ASO treatment of these patients. n-Lorem looks forward to continuing to work with the FDA to develop definitive guidance that facilitates meeting the needs of these patients with the highest possible quality.
“On behalf of the patients we serve and the many more we hope to help in the future by providing experimental ASO treatments free, for life, we thank the continuing support of our founding donors – Ionis Pharmaceuticals, Biogen and the Crookes – as well as the numerous new donors, members of the Access to Treatment Committee, board members, advisory committee members, and volunteers who have provided support,” said Tracy Johnson, Executive Director, n-Lorem Foundation. “A special thank you to our collaborators whose support is essential in meeting the needs of our patients, including Ionis Pharmaceuticals, Biogen, Charles River Labs, Covance by Labcorp, Illumina and the Korea Institute of Toxicology (KIT).”
The 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 10 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 current 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.
If you are interested in supporting the efforts of the n-Lorem Foundation, please contact Tracy Johnson at 760- 552-7113 or Tracy.Johnson@nlorem.org.
Please consider giving to n-Lorem to bring hope, possibility and treatment options to these desperate patients and families.