n-Lorem Foundation Convenes First Annual Nano-rare Patient Colloquium to Share Accomplishments and Learnings in Developing Optimized ASO Therapies for Nano-rare Patients

n-Lorem, a nonprofit foundation, will assemble patients, physicians, partners and supporters tomorrow, October 12, 2023, for its first-annual Nano-rare Patient Colloquium to provide valuable information to the nano-rare community, bring together those who support n-Lorem's mission to finding treatments for nano-rare, give nano-rare patients and families a voice and provide a public platform to illustrate the high-quality process n-Lorem uses to advance from an accepted patient program to treating with an optimized ASO for that patient. Hosted at Biogen's headquarters in Cambridge, Mass., the meeting includes more than 30 panelists and presenters and more than 250 in-person and virtual attendees, all of whom are dedicated to finding treatments for people with nano-rare illnesses (30 or fewer patients affected by a single gene mutation).

"At this first-of-its-kind meeting, we are pleased to share our progress, our aspirations and the important lessons we are learning that we believe will reshape how we think about health and disease," shared Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO, n-Lorem Foundation. "This forum provides voice to our nano-rare community, and we look forward to hearing from our pioneering patients and their families, physicians, patient advocates and our partners."

"With every nano-rare patient, we have an extraordinary opportunity to learn more about the unique nature of these genetic-based diseases," continued Dr. Crooke. "What we discover from a single patient helps us advance care for all, and we owe it to our patients to learn as much as possible about these illnesses. We believe that if we provide a new future to one patient, one family at a time, we change the world."

The event includes a full day of presentations and panels, providing a broad overview of n-Lorem's efforts for nano-rare, including:

• Patient diagnostic journeys
• Partner and supporter panels discussing why they have joined our community of care
• Physicians highlighting the challenges and rewards of treating nano-rare patients
• Patient advocates who are committed to driving reform
• Behind the scenes: n-Lorem discovery and development decision making to ensure only the most optimized ASO is moved forward
• n-Lorem's holistic approach to empowering patients and families

"While ASO technology sounds simple, it is not and, in this meeting, we hope to provide insight into our extensive discovery process to identify an optimal ASO medicine and our drug development decisions, which are supported by years of expertise and experience," concluded Dr. Crooke.

Interested parties can access the event through via a live stream tomorrow 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 in the coming weeks.

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 225 applications for treatment with approximately 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.

To learn more about n-Lorem's mission, visit www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these patients and families in need.