n-Lorem and EspeRare Join Forces to Expand Access to Personalized ASO Treatments for Rare Genetic Diseases in Europe

A New Era in Rare Disease Treatment

The collaboration between n-Lorem Foundation and EspeRare Foundation marks a significant advance in the world of personalized medicine for rare genetic disorders. Announced on January 13, 2026, this strategic partnership aims to broaden access to antisense oligonucleotide (ASO) therapies developed by n-Lorem across Europe. With an emphasis on addressing regulatory, clinical, and diagnostic barriers, the initiative seeks to bring hope to patients struggling with rare genetic conditions.

Addressing the Needs of Nano-Rare Patients

The partnership initially targets a small group of patients identified as 'nano-rare' in Switzerland, with plans to create a replicable model that can expand to other EU countries. Nano-rare diseases, caused by unique genetic mutations, often leave patients without any viable treatment options. This collaboration aims to bridge that gap, leveraging n-Lorem's strength in providing tailored ASO treatments, and EspeRare's expertise in navigating European regulatory landscapes.

EspeRare has a rich history of developing therapeutic options for neglected patient communities, prioritizing their needs above all. By working closely with health authorities, ethics committees, and universities, EspeRare will help shape customized access frameworks grounded in scientific rigour, patient safety, and robust ethical governance.

Scientific Innovation Meets Patient-Centric Approaches

N-Lorem stands at the forefront of developing personalized ASO therapies and currently treats the largest cohort of nano-rare patients globally. Their protocols, established under FDA regulations, need to be adapted for European contexts, ensuring that patient safety and scientific integrity remain paramount. With their collaboration, EspeRare and n-Lorem are working towards identifying European patients who can benefit from treatments already being safely administered under FDA approval.

This involves a careful coordination with healthcare providers to align with local regulatory and ethical requirements while employing advanced genomic technologies for thorough patient assessments. n-Lorem is committed to ensuring patient safety throughout the treatment process, maintaining rigorous scientific evaluations and independent ethical oversight.

Collaborative Framework for Future Acclaim

Sarah Glass, Ph.D., Chief Operating Officer of n-Lorem, stated, "We are committed to enabling as many patients as possible to gain access to treatments through our ASO platform. The global nano-rare community has enormous needs. This collaboration is a critical step in expanding our mission beyond the United States." The success of these therapies depends on the robustness of clinical and regulatory frameworks to support their administration.

Caroline Kant, CEO of EspeRare, remarked on the technological advancements bridging patients with effective therapies, underscoring the need to improve access to promising innovations that often get lost in complex healthcare systems. "This partnership aims to make cutting-edge therapies available to families in urgent need, redefining what precision medicine means for those affected by rare diseases."

A steering committee comprising representatives from both organizations will govern the collaboration, coordinating regulatory strategies and clinical engagement. The goal is to create sustainable care pathways in Europe for personalized gene therapies, thereby reducing access inequities for patients with ultra-rare diseases.

An Ambitious Start

Initially, EspeRare intends to support treatment initiation for up to three eligible nano-rare patients located in Switzerland during the first year. These patients will be treated with ASO therapies that are currently under clinical evaluation in the U.S. This model could eventually be expanded to other EU nations, establishing a framework for future collaborations and treatment accessibility.

About n-Lorem Foundation

Founded with the aim of utilizing antisense technology, n-Lorem is a non-profit organization dedicated to providing free experimental ASO treatments to nano-rare patients. These individuals, who are exceedingly rare—ranging from one to 30 cases globally—often lack any therapeutic options. N-Lorem has made strides in providing hope to these patients and smoothing the development of treatments tailored precisely to their unique genetic profiles. To date, they have processed over 400 treatment requests, with more than 200 approved cases and about 40 patients currently undergoing treatment.

About EspeRare Foundation

EspeRare is committed to advancing solutions for rare diseases through strategic partnerships, translational research infrastructure, and patient-focused programs. Their mission includes dismantling the barriers that prevent promising scientific innovations from reaching historically neglected patients. With more than a decade of experience, EspeRare combines the flexibility of biotech with a social mission to ensure equitable access to vital therapies.

Together, these two organizations represent a beacon of hope for the future of personalized medicine in Europe’s healthcare landscape.