n-Lorem to share its progress at its Second Annual Nano-rare Patient Colloquium beginning today Oct. 30 

n-Lorem has made tremendous progress over the last five years to develop optimized, personalized ASO medicines for nano-rare patients and provide them for free, for life.  

SAN DIEGO, Calif., Oct 30, 2024, (BUSINESS WIRE) n-Lorem, a nonprofit foundation, will assemble patients, physicians, partners and supporters today and tomorrow, Oct. 30 & 31, 2024, for its second annual Nano-rare Patient Colloquium.  In this public meeting, n-Lorem will provide valuable information to the nano-rare community, bring together those who support n-Lorem’s mission of discovering optimal treatments for nano-rare patients, give nano-rare patients a voice and highlight the industrialized processes n-Lorem has implemented to discover, develop and ultimately treat nano-rare patients with an optimized ASO for that individual patient.  The meeting is being held at the Cambridge Marriott and includes more than 40 panelists and presenters and approximately 500 in-person and virtual attendees, all of whom are dedicated to finding treatment for nano-rare patients.  Live streaming of the event can be accessed by visiting n-Lorem’s website: www.nlorem.org. 

“Our first annual meeting in 2023 was an overwhelming success, and we expect that this year’s event will be even more informative.  We have a number of patients who have been treated for longer than a year, with our longest treated patient for nearly two years.  Today we treat diseases of the liver, kidney, eye and CNS and have observed positive clinical changes from ASO treatment in patients with nano-rare diseases that are expressed in different organs,” shared Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO, n-Lorem Foundation.  “We look forward to sharing these observations today and continuing our discussions from last year on how to help more nano-rare patients, providing a voice to our nano-rare community and hearing from our industry partners, academic clinical investigators, expert advisors and supporters of nano-rare.” 

To date, n-Lorem has evaluated more than 300 applications to treat and of those has accepted more than 140 nano-rare patients for treatment.  n-Lorem has initiated treatment for 15 patients, with approximately 25 patients projected to be treated by the end of 2024.  Details of n-Lorem’s progress and the current challenges facing treating nano-rare patients will be presented at n-Lorem’s second annual Nano-rare Patient Colloquium, which is being hosted by Biogen on Oct. 30-31 in Cambridge, Mass.  

“When we founded n-Lorem in 2020, we assumed that we would process about 10 to 20 applications for treatment by 2025. We exceeded that goal in the first year and now have processed more than 300 applications for treatment,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO, n-Lorem Foundation. “This demand is reflective of the need for hope among those who are nano-rare. They have historically been overlooked by the healthcare system, leaving them isolated and debilitated and placing extraordinary stress on their family and supporters. We offer the hope they have been looking for, the hope physicians have been looking for to help their patients.” 

Registration for the Patient Colloquium is currently open, and participants can attend in-person or virtually. 

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 300 applications for treatment with more than 140 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. 

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.  

n-Lorem contact:

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

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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.