n-Lorem and EspeRare announce European collaboration to expand access to individualized ASO Therapies for Rare Genetic Diseases

SAN DIEGO and GENEVA, Jan. 13, 2026 /PRNewswire/ -- The n-Lorem Foundation and the EspeRare Foundation announced today a strategic collaboration to expand access in Europe to n-Lorem's antisense oligonucleotide (ASO) therapies for patients with rare genetic diseases. The collaboration aims to address the regulatory, operational, and diagnostic barriers that limit access to personalized genetic medicines outside the United States. The partnership will initially focus on pursuing treatment of a small number of nano-rare patients who reside in Switzerland, with the goal of establishing a scalable model to support subsequent expansion across the European Union.

EspeRare is committed to the development of treatment options for overlooked patients, rooted in a patient-first ethos and deep engagement with patient communities. Leveraging long-standing expertise in navigating regulatory pathways and building multi-stakeholder networks across health systems, including regulators, ethics committees and academic medical centers, EspeRare will co-develop fit-for-purpose access frameworks for n-Lorem's ASO therapies built upon scientific rigor, patient safety, and robust ethical oversight.

n-Lorem has led the discovery and development of personalized experimental ASO medicines and currently treats the largest number of nano-rare patients globally. n-Lorem's industrialized process is grounded in rigorous science, a deep understanding of ASO technology, expertise and experience discovering and developing ASOs to create and provide the most optimal ASO to each individual patient.  To date, n-Lorem has operated under FDA authorization, expanding access internationally requires tailored clinical and regulatory solutions that respect local regulatory frameworks while maintaining n-Lorem's standards for patient safety and scientific rigor. 

With this collaboration, EspeRare will work closely with n-Lorem to support the identification of European patients who may benefit from n-Lorem's individualized ASO therapies that are already being used to treat nano-rare patients safely under FDA authorization and to help enable the regulatory and clinical pathways required for treatment within Swiss and European academic medical centers. This includes coordination with treating physicians and institutions, alignment with country-specific regulatory and ethics requirements, and integration of advanced genomic techniques to ensure a precise understanding of each patient's underlying biology. Consistent with n-Lorem's mission, patient safety remains paramount and will be upheld through rigorous scientific review, independent ethical oversight, and careful clinical decision-making at each stage of the process. As an initial focus, the collaboration will concentrate on establishing the clinical and regulatory pathways needed to enable patient access in Switzerland, which will then be used as a framework to establish similar pathways throughout Europe.

"At n-Lorem, we are committed to enabling the treatment for as many patients as possible using our established industrialized ASO platform.  We recognized the expansive need of the nano-rare community globally and feel that this collaboration represents an important step toward extending n-Lorem's mission to patients beyond the United States," said Sarah Glass,Ph.D., Chief Operating Officer of the n-Lorem Foundation. "Individualized ASO therapies can only reach patients if the underlying regulatory and clinical frameworks are capable of supporting them. EspeRare's experience navigating European regulatory systems and enabling access to complex rare disease therapies makes them an ideal partner as we work to responsibly expand access for European patients."

"EspeRare was founded to close the gap between scientific possibility and real-world patient access," said Caroline Kant, Executive Director of EspeRare. "Far too often, innovation stops at the border of accessibility. That is why we are determined to bring our expertise and network to help extend the reach of n-Lorem's pioneering ASO platform beyond the United States. Together, we aim to deliver groundbreaking treatments to families who urgently need them and to redefine what's possible in precision medicine."

The collaboration will be governed by a steering committee with representatives from both organizations to coordinate regulatory strategy, clinical engagement, and program execution. The parties anticipate a multi-year collaboration reflecting a shared commitment to building durable, Europe-focused pathways for individualized genetic medicines and to reducing inequities in access for patients with ultra-rare conditions.  As an initial focus, EspeRare anticipates supporting the initiation of treatment within the first year for up to three Switzerland-based patients who are amenable to ASO therapies already under clinical evaluation in n-Lorem nano-rare patients.

About n-Lorem Foundation

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 400 applications for treatment with more than 200 nano-rare patients approved and more than 40 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.
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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.

About the EspeRare Foundation
EspeRare is a nonprofit organization dedicated to advancing therapies for patients with rare diseases through strategic partnerships, translational infrastructure, and patient-centered programs. After a decade of removing drug development barriers that prevent promising science from reaching historically overlooked patients, EspeRare has become a pioneer in prenatal and personalized therapies for rare diseases. Combining biotech agility with a mission-driven model, EspeRare bridges the translational "valley of death" by aligning patient communities, academics, commercial partners, and regulators to advance treatments and support equitable access. Based in Geneva, it leverages its position at the crossroads of science, health diplomacy and the life science industry to drive R&D and structure collaborations that unlock innovative therapies and maximize global patient impact.

Visit www.esperare.org to learn more about our mission and if you're inspired by what we do, consider supporting us. Your generosity fuels the breakthroughs these families have been waiting for.

Media Contacts
n-Lorem Foundation
Amy Williford, Ph.D.
Vice President, Foundation Development and External Relations
amy.williford@nlorem.org

EspeRare Foundation
Ryan Taft
taft.ryan@esperare.org

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