SAN DIEGO, Calif., October 18, 2022, (Business Wire) — n-Lorem, a nonprofit foundation,  announced today that it has surpassed its 2022 fundraising goal of $8 million with more than $17 million in committed cash in 2022. As a non-profit, this support is critically important for n-Lorem’s mission to discover and develop personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

“On behalf of our patients, we are grateful for the committed support from our partners, our individual donors and granting foundations that have come together with a purpose to provide better futures to nano-rare patients. The tremendous need for n-Lorem has greatly exceeded our expectations. It is now clear that there are many more nano-rare patients than we projected. As we bring hope and potential help to these patients, many more find n-Lorem,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO of n-Lorem Foundation. “While we are extremely appreciative of our supporters, we need more help, more donors, more support to meet a growing demand. We are continually exploring ways to increase our capacity for research and development, bring in more funding and create processes that ensure the highest quality at each and every step while scaling up to meet the growing demand for treatment.”

“What n-Lorem has done in less than three years is remarkable. It is evident that n-Lorem’s charitable approach can be sustainable, which is exceptional news for nano-rare patients today,” said Michael Hayden, MBChB, Ph.D., Killam Professor of Medical Genetics at University of British Columbia and n-Lorem Board of Director. “n-Lorem’s non-profit model is first-in-class. It is institutionalized with quality as the highest priority at each step and is scalable to meet the needs of many nano-rare patients. Although these patients may be the only patient in the world with their unique gene mutation, there are likely to be tens of thousands of nano-rare patients. Each patient requires a therapeutic approach that is designed only for them. That a foundation would take on this challenge is astonishing.”

The n-Lorem mission aligns with the heart of the pharmaceutical industry and many companies involved in the drug discovery, development and manufacturing industry are substantial supporters of n-Lorem. To date, n-Lorem has partnered with over 30 companies, all of whom are the best of the best in the industry and share n-Lorem’s mission to provide help to the patients who need it the most, today. Each partner is providing in-kind or monetary support and brings a team of dedicated employees who are committed to providing the highest quality at every step in the process. With more than 70 patients accepted into its program, n-Lorem and its partners work together to discover, develop and provide a personalized medicine that has been designed and optimized for a single patient.

To date, n-Lorem has more than 75 donors and supporters, each and every one is helping a nano-rare patient, today. This year, n-Lorem added four new major donors that support n-Lorem’s efforts in particular disease areas.

• Silence ALS initiative between n-Lorem and Columbia University focused on nano-rare ALS patients was supported by an initial funding donation of $400,000 by Target ALS
• Wolverine Foundation and n-Lorem partnered to expedite research and ASO discovery for allele-specific gene targets associated with neurodevelopmental disease. n-Lorem received $3 million and could receive an additional $5.5 million as the collaboration proceeds and research targets are achieved.
• Solve FSHD and an anonymous donor provided n-Lorem more than $1.7 million to support n-Lorem’s work on expanding the understanding of FSHD2, a rare form of muscular dystrophy.
• An anonymous donor pledged $8.5 million to support n-Lorem’s discovery and development efforts to provide experimental ASOs for eight different nano-rare patients.

We hope that you will consider joining us. Without your support, we could not do what we do. It is vital for us to continue our mission of providing nano-rare patients and their families with an opportunity for a better life, renewed hope and brighter dreams,” concluded Dr. Crooke.

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 

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 has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 40 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.

n-Lorem contact:

Amy Williford, Ph.D.
Director of Communications
[email protected]

We cannot do
this alone

We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.