EspeRare and the n-Lorem Foundation have launched a strategic partnership to bring individualized antisense oligonucleotide (ASO) therapies to patients with nano-rare genetic conditions in Europe.
The collaboration will begin with a pilot phase in Switzerland, aimed at unlocking the regulatory, diagnostic, and clinical pathways needed to enable access to these transformative treatments. Initial efforts will focus on a small group of carefully selected Swiss patients already matched to individualized ASO therapies developed by n-Lorem and authorized for use by the FDA in the United States.
“We are thrilled to have Dr. Ryan Taft leading this initiative for EspeRare,” said Caroline Kant, Executive Director and Co-founder of the EspeRare Foundation. “His deep understanding of genomic medicine and experience at the intersection of science, regulation, and patient access are central to our efforts to responsibly expand this new therapeutic paradigm in Europe. We are equally grateful to Lifehouse, whose commitment to advancing care for children affected by severe rare neurodevelopmental disorders is instrumental in enabling this work.”
n-Lorem has pioneered a groundbreaking model in precision medicine by designing and providing individualized ASO therapies for patients with nano-rare genetic conditions—typically affecting only a handful of individuals worldwide. While these therapies are currently developed and accessible exclusively in the United States, the collaboration with EspeRare aims to explore how similar therapeutic pathways could be enabled in Europe, through careful adaptation to the specific regulatory, ethical, and clinical frameworks of both the Swiss and the European context.
EspeRare brings over a decade of experience navigating complex regulatory environments and collaborating with academic hospitals, ethics committees, and public authorities. In this collaboration, EspeRare’s role is to facilitate regulatory coordination, engage key stakeholders, and help develop sustainable access pathways, while clinical oversight and patient care will remain entrusted to accredited medical institutions.
“This program is focused on expanding access to cutting-edge therapies, and improving rare disease patient lives as quickly as possible,” said Dr. Taft. “Together with n-Lorem, we’re building the bridge between its individualized ASO platform, and the systems required to deliver these treatments to patients outside the United States.”
This initiative is supported by Lifehouse, whose commitment to advancing care for children affected by severe rare neurodevelopmental disorders has been instrumental in enabling this work.
This collaboration reflects EspeRare’s broader mission to bridge scientific innovation and equitable access for overlooked patient populations, while ensuring that new therapeutic paradigms and precision medicine are introduced responsibly and sustainably.
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