n-Lorem Provides Update on Its Remarkable Progress to Deliver Hope and Potential Help to More Than 100 Nano-rare Patients

n-Lorem, a nonprofit Foundation, outlines the exceptional progress the Foundation has made toward its mission to discover, develop and provide treatment to nano-rare patients (1-30 patients worldwide), for free, for life. Already ten investigational new drug (IND) applications have been filed with the FDA and n-Lorem has dosed six nano-rare patients with plans to dose additional patients within the coming months.

"As 2023 draws to a close, I am excited about the positive impact our mission is having in the nano-rare community today," said Stanley T. Crooke, M.D., Ph.D., Founder, chairman and CEO, n-Lorem Foundation. "The outstanding progress we have made this year in fulfilling our commitment to nano-rare patients builds momentum within the community for even greater successes for nano-rare patients in the future."

Since inception n-Lorem has:

• Received and reviewed over 225 applications and accepted more than 100 patients;
• Filed ten INDs with four different divisions of the FDA;
• Began treatment on six nano-rare patients;
• 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 39 episodes released and more than 10,000 downloads;
• Built out its main laboratory space more than tripling the Foundation's drug discovery efforts and recruited top tier scientists;
• Shared learnings broadly in the community through first annual nano-rare patient colloquium, presentations and publications;
• Reduced per-patient costs of drug discovery, development and manufacturing; and
• Built out its leadership team to support this rapid growth.

Learn more about the exciting progress n-Lorem has made by reading Stan Crooke's annual letter and visit n-Lorem's website.

• Read: Year-end Annual Letter

"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: n-Lorem Patient Empowerment Program Podcast Series
• Watch: n-Lorem Hope is Possible
• Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
• Read: USA Today Article on Nano-rare and the Future of Medical Care

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 received over 225 applications for treatment with more than 100 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.