n-Lorem Foundation Organizes Third Annual Nano-rare Patient Colloquium, Highlighting Scientific and Clinical Advancements Achieved Since Organization’s Founding

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SAN DIEGO--(BUSINESS WIRE)--Oct 2, 2025--

n-Lorem, a nonprofit foundation, announced today that it will hold its third annual Nano-rare Patient Colloquium in Cambridge, Mass., where n-Lorem leaders and experts in the field will share updates on research being conducted to find treatments for patients with nano-rare diseases. The Colloquium, which is being hosted Oct. 20-21 by Biogen, will include >40 panelists and presenters, a scientific poster session, reception and an art auction (bidding is happening now) with more than 480 registrations to date.

“This meeting is a reflection of our commitment to create a community for nano-rare patients and to enhance awareness of the needs of nano-rare patients and their families. I believe that this year’s event will be even more impactful than the last two years. We have more than 35 patients being treated with ASOs discovered for them. With one patient treated almost three years and many approaching two years in treatment we are beginning to learn about the long-term value of bespoke ASO treatment in nano-rare patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, chairman and CEO, n-Lorem Foundation. “Thanks to the extraordinary demand we have experienced, this meeting will share important lessons about nano-rare patients and the effects of bespoke treatment in these patients. These lessons are relevant not only to nano-rare patients but, over time, may revolutionize the way we think about health and disease altogether.”

This event includes two full days of presentations and panels, providing a broad overview of n-Lorem’s efforts for the nano-rare including:

• Progress in building a knowledgeable community of and for nano-rare patients
• Clinical learnings from pioneer patient on treatment
• Lessons about developmental delays and whether development delays can be reversed with treatment
• Lessons learned about the safety of bespoke ASOs discovered and developed via the rigorous and industrialized processes invented by n-Lorem
• New advances in antisense oligonucleotide (ASO) technology created at n-Lorem

Rescue 7: Firefighters for Patients will be on-site during the Colloquium to hang out with families and entertain children with firefighting-themed activities while caregivers attend the event.

Registration is free and participants can attend in-person or virtually. Interested parties can access the event through via a live stream and more information on the Colloquium can be found on n-Lorem’s website.

Presentations and panel discussions at the Colloquium will be videotaped and available on n-Lorem’s website several weeks after the event.

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 380 applications for treatment with more than 200 nano-rare patients approved and more than 30 patients on treatment. 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, visit us at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to nano-rare patients in need.

View source version on businesswire.com:https://www.businesswire.com/news/home/20251002216594/en/

CONTACT: n-Lorem Contact:

Amy Williford, Ph.D.

Executive Director of Communications and Donor Relations

[email protected]

KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA MASSACHUSETTS

INDUSTRY KEYWORD: RESEARCH GENETICS PHILANTHROPY BIOTECHNOLOGY HEALTH FUND RAISING PHARMACEUTICAL FOUNDATION SCIENCE

SOURCE: n-Lorem Foundation

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PUB: 10/02/2025 09:05 AM/DISC: 10/02/2025 09:04 AM

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