Gene Therapy Pioneers Forge Path to Europe for Nano-Rare Patients

GENEVA, SWITZERLAND – January 13, 2026 – In a move that could redefine access to personalized medicine, two non-profit foundations from opposite sides of the Atlantic have joined forces to bring highly individualized genetic therapies to European patients with ultra-rare diseases. The San Diego-based n-Lorem Foundation, a leader in developing bespoke antisense oligonucleotide (ASO) treatments, and the Geneva-based EspeRare Foundation, experts in navigating Europe's complex regulatory landscape, have announced a strategic collaboration to break down the barriers that have largely confined these life-changing therapies to the United States.

The partnership aims to create a viable pathway for treating European patients suffering from "nano-rare" diseases—conditions caused by a single genetic defect that may affect as few as one to 30 individuals worldwide. For these patients, traditional pharmaceutical development is commercially unfeasible, leaving them with little to no hope for treatment. This new alliance intends to change that, beginning with a pilot program in Switzerland.

A Partnership of Pioneers

The collaboration unites two organizations with complementary expertise. The n-Lorem Foundation, founded by RNA-therapeutics pioneer Dr. Stanley T. Crooke, has created what it calls an "industrialized process" for discovering and developing experimental ASO medicines. ASOs are short strands of modified DNA designed to precisely target and correct the genetic errors underlying a specific patient's disease. Operating under U.S. Food and Drug Administration (FDA) authorization, n-Lorem has become the world's largest provider of these charitable, individualized treatments, having received over 400 applications and currently treating more than 40 patients.

While n-Lorem provides the technological engine, EspeRare supplies the crucial navigational charts for Europe. For a decade, the Swiss foundation has specialized in bridging the translational "valley of death," where promising science often fails to become real-world treatment due to regulatory and financial hurdles. EspeRare builds networks connecting patient communities, academic researchers, and regulatory bodies to advance therapies for overlooked patient populations.

"Individualized ASO therapies can only reach patients if the underlying regulatory and clinical frameworks are capable of supporting them," said Dr. Sarah Glass, Chief Operating Officer of the n-Lorem Foundation. "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."

This sentiment is echoed by EspeRare's leadership, who see the partnership as a direct fulfillment of their mission.

"Far too often, innovation stops at the border of accessibility," stated Caroline Kant, Executive Director of EspeRare. "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."

Navigating the European Regulatory Labyrinth

Translating n-Lorem's FDA-authorized model to Europe is the central challenge the collaboration seeks to solve. The continent's regulatory landscape for Advanced Therapy Medicinal Products (ATMPs)—a category that includes gene and cell therapies like ASOs—is a patchwork of centralized EU rules and specific national laws. While the European Medicines Agency (EMA) has a centralized procedure for ATMPs, early access mechanisms like Compassionate Use Programs (CUPs) or Named Patient Programs (NPPs) are governed by individual member states.

This is where EspeRare's expertise becomes critical. The foundation will co-develop "fit-for-purpose" access frameworks built on n-Lorem's standards of scientific rigor and patient safety. This involves intricate coordination with national authorities like Swissmedic in Switzerland, local ethics committees, and academic medical centers. The collaboration will likely leverage existing but complex pathways, such as the "hospital exemption," which allows hospitals in some jurisdictions to prepare and use ATMPs for individual patients without a full marketing authorization under strict conditions. The interpretation of this exemption, however, varies significantly across the EU, underscoring the need for a carefully tailored, country-by-country strategy.

Switzerland provides a strategic starting point. Its regulatory body, Swissmedic, has a specialized division for ATMPs and is actively working to align its legislation with EU standards, potentially creating a smoother path for future expansion. Furthermore, the country's world-class academic hospitals provide the necessary infrastructure for advanced genomic diagnostics and clinical care.

From Swiss Pilot to Pan-European Precedent

The collaboration's initial focus is to establish the clinical and regulatory pathways needed to treat up to three nano-rare patients in Switzerland within the first year. These patients will have conditions amenable to ASO therapies that are already being safely administered to patients in the U.S. under n-Lorem's FDA programs. This pilot phase is not merely about treating a few individuals; it is about creating a robust, replicable model.

The long-term vision is to use the Swiss framework as a blueprint for expansion across the European Union. A joint steering committee with representatives from both foundations will oversee the multi-year effort, coordinating regulatory strategy and clinical engagement. The goal is to build durable, Europe-focused pathways that reduce the profound inequities in access for patients with the rarest of conditions.

However, scaling this model presents its own hurdles. Each EU member state has distinct healthcare systems, funding mechanisms, and health technology assessment (HTA) bodies that evaluate a therapy's cost-effectiveness. Securing reimbursement for highly individualized, and often costly, treatments will require navigating 27 different systems. Furthermore, recent proposals to revise EU pharmaceutical legislation have raised concerns among patient groups that the bar for approving rare disease treatments based on small, single-arm trials—often the only option for nano-rare conditions—could be raised.

Despite these challenges, the partnership represents a significant step forward. By combining a proven therapeutic platform with deep regulatory expertise, n-Lorem and EspeRare are not just importing a technology; they are attempting to engineer a new paradigm for global access to precision medicine, offering tangible hope to families who have long been told that their disease is too rare to treat.