n-Lorem Foundation Announces the 50th Nano-rare Patient Receives Personalized ASO Medicine

No ASO-related serious adverse events and an excellent overall safety and tolerability record

One patient treated for more than three years and 24 patients treated for >1 year

SAN DIEGO--(BUSINESS WIRE)--n-Lorem, a nonprofit foundation, announced today that the 50^th nano-rare patient has been treated with a personalized, experimental antisense oligonucleotide (ASO) medicine discovered, developed and provided by n-Lorem. n-Lorem has shown that the efficiency of ASO technology, coupled to the unique FDA guidance documents for nano-rare patients, can rapidly bring effective treatments to even the most advanced patients.

n-Lorem has observed an excellent safety and tolerability profile across all treated patients to date. Across more than 260 doses administered through multiple routes of administration, no ASO-related serious adverse events have been observed. These data represent over 45 patient-years of cumulative safety experience and form the largest dataset of nano-rare patients treated with experimental ASO therapies. To date, the foundation has one patient who has been treated for three years and many more who are approaching one and two years on treatment.

“When I founded n-Lorem, I thought it would be a significant success to delay progression of diseases that are caused by nano-rare mutations. In fact, in most patients we are documenting significant improvements, reacquisition of development skills lost, acquisition of new skills and improvement in quality of life for most patients with neurodevelopmental/neurodegenerative diseases,” said Stanley T. Crooke, Founder, Chairman and CEO of n-Lorem. “Our success showcases the potential of ASO treatments of genetic diseases.”

n-Lorem has received more than 440 applications and accepted more than 240 nano-rare patients for potential treatment with personalized experimental ASOs discovered and developed at by n-Lorem. n-Lorem has filed and received authorization to proceed for more than 45 investigational new drug (IND) applications with five divisions of the FDA. Nearly all evaluable patients have achieved clinically significant benefit, despite the fact that many nano-rare patients are severely affected by their disease.

“The greater than 90% success rate and excellent safety and tolerability profile we have reported emphasize the importance of deep expertise in ASO technology, our industrialized processes and thorough ASO discovery and development process that assure that each patient is treated with an optimal, safe and effective ASO,” concluded Dr. Crooke.

Patients accepted into the n-Lorem program receive treatments from n-Lorem for free, for life. They are not expected to contribute to or fundraise for the development of the therapy. All treatments are developed thanks to monetary and in-kind donations made by people, foundations and companies dedicated to advancing the treatment of nano-rare diseases.

n-Lorem’s work was recently highlighted at the first CNBC Cures Summit, a landmark event dedicated to bringing together the most influential leaders, policymakers and investors in biotechnology to accelerate the future of medicine through breakthroughs in rare disease research. CNBC Cures is a new initiative launched in January to help raise awareness of rare diseases and improve patient outcomes for people living with them. Go here to view the panel ‘Redefining What’s Possible’ that includes Dr. Stan Crooke.

To 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 patients and families in need.

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 received over 440 applications for treatment with more than 240 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 X, Facebook, LinkedIn and YouTube.

To 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 patients and families in need.