News

EspeRare is honoured to contribute to the scientific session of the Senate of the Swiss Academy of Medical Sciences (SAMS/ASSM) in Bern on 18 June 2026.

As part of the mini-symposium “New therapies and rare diseases ”, which will also feature contributions from EspeRare’s partners Dr Roxane van Heurck of Geneva University Hospitals and Prof. Vladimir Katanaev of the University of Geneva, Caroline Kant, Executive Director of EspeRare, will give a presentation entitled:

“From molecular diagnosis to precision-medicine pathways for children with severe rare genetic diseases”

The session will bring together complementary perspectives on emerging therapeutic opportunities for rare genetic diseases, including antisense oligonucleotides, clinical implementation, and the broader transition toward individualized precision-therapy pathways.

Her presentation will address how molecular diagnosis can become the starting point for a personalized treatment path and what clinical, regulatory and access infrastructure is needed to make this possible in Switzerland.

EspeRare will share lessons from its partnership with n-Lorem Foundation and Geneva University Hospitals (HUG) to help enable access to individualized antisense oligonucleotides therapies for Swiss patients. The presentation will also highlight EspeRare’s collaboration with Prof. Vladimir Katanaev at the University of Geneva and Life House to explore personalized drug repositioning approaches for children with severe genetic neurodevelopmental diseases. 

Together, these initiatives illustrate EspeRare’s commitment in helping translate molecular diagnosis into responsible precision-medicine pathways for children with rare genetic diseases.

Learn more about the Swiss Academy of Medical Sciences: https://www.samw.ch/en.html

The Health Ethics & Policy Lab at ETH Zurich is hosting a lecture on April 23 at 2:00 p.m. titled “Treating Diseases Before Birth: The Next Frontier in Precision Medicine for Rare Diseases,” featuring Caroline Kant and Sébastien Mazzuri. 
Through the ETHix SERIES, the Health Ethics & Policy lab of the ETH Zurich aims to regularly bring some of the leading thinkers in Bioethics, Health Policy and Digital Ethics to a wider audience.

 Learn more and register here: https://www.linkedin.com/events/7442240065340149761/

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.

Read the full press release