n-Lorem Enters Collaboration with EspeRare to Expand Access to Individualized ASOs for Rare Genetic Diseases

Rare Daily Staff

The n-Lorem Foundation and the Geneva-based EspeRare Foundation said they have entered a strategic collaboration to expand access in Europe to n-Lorem’s antisense oligonucleotide 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 treating a small number of nano-rare patients who live in Switzerland, with the goal of establishing a scalable model to support subsequent expansion across the European Union.

EspeRare will leverage its long-standing expertise in navigating regulatory pathways and building multi-stakeholder networks across health systems, including regulators, ethics committees, and academic medical centers. It will co-develop fit-for-purpose access frameworks for n-Lorem’s ASO therapies built on scientific rigor, patient safety, and 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. To date, the organization has operated under FDA authorization, but expanding access internationally requires tailored clinical and regulatory solutions that respect local frameworks while maintaining n-Lorem’s standards for patient safety and scientific rigor.

“We recognize 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, 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.”

Photo: Sarah Glass, chief operating officer of the n-Lorem Foundation