n-Lorem and EspeRare Unite to Enhance Access to Rare Disease Therapies in Europe
The n-Lorem Foundation and EspeRare Foundation have announced a strategic collaboration aimed at enhancing access to specialized antisense oligonucleotide (ASO) therapies for patients suffering from rare genetic disorders in Europe. This partnership seeks to address and surmount the existing regulatory, clinical, and diagnostic barriers that currently limit access to personalized gene therapies outside of the United States.
Initially, the partnership will target a select group of patients with what are termed 'nano-rare' disorders in Switzerland, aspiring to develop a replicable and transferable model that could eventually extend to other European Union countries. EspeRare focuses on the development of therapeutic options for underrepresented patient groups, placing their well-being at the core of its mission and maintaining close relationships within impacted communities. By leveraging its expertise in navigating European regulatory frameworks and establishing connections with various health stakeholders—including regulatory authorities, ethics committees, and university hospitals—EspeRare will collaborate with n-Lorem to create tailored access models grounded in scientific rigor, patient safety, and solid ethical governance.
As a leader in the development of customized and experimental ASO therapies, n-Lorem currently treats the largest number of patients worldwide with rare nano-disorders. Their industrialized approach is built on substantial scientific expertise, comprehensive knowledge of ASO technologies, and years of experience creating tailored therapies for each patient. Currently, n-Lorem operates under FDA approval, and expanding international access necessitates customized regulatory and clinical solutions that consider local conditions while upholding n-Lorem’s high safety and scientific standards.
Through this collaboration, EspeRare will closely work with n-Lorem to identify European patients who could benefit from ASO therapies, which have already been safely administered to patients with rare diseases within the FDA approval framework. This includes coordinating with healthcare professionals and institutions, ensuring compliance with national regulatory and ethical standards, and integrating advanced genomic technologies to create a detailed understanding of each patient’s unique biology. The mission of n-Lorem emphasizes patient safety, which will be safeguarded throughout the entire process via rigorous scientific evaluations, independent ethical oversight, and informed clinical decision-making.
Dr. Sarah Glass, Chief Operating Officer of the n-Lorem Foundation, stated, “We at n-Lorem are committed to providing therapy access through our ASO platform to as many patients as possible. The nano-rare community has a tremendous need worldwide. This collaboration marks a critical step in expanding our mission beyond the USA. Personalized ASO therapies can only reach patients when the clinical and regulatory environments allow for it. EspeRare’s expertise in European approval systems makes it the ideal partner for responsibly extending access for patients in this region.”
Caroline Kant, director of the EspeRare Foundation, remarked, “EspeRare was founded to bridge the gap between biomedical advancements and actual patient access to therapies. Therapeutic innovation often fails at the hands of complex and fragmented health systems. Thus, we aim to leverage our expertise and network to extend n-Lorem’s ASO platform beyond the USA. Together, we aspire to make cutting-edge therapies available to families who desperately need them, redefining the possibilities of precision medicine.”
The collaboration will be overseen by a steering committee comprising representatives from both organizations, responsible for coordinating regulatory strategy, clinical engagement, and program execution. The partners aim for a multi-year collaboration to emphasize their shared commitment to building sustainable treatment pathways for personalized gene therapies focused on Europe, while reducing inequalities in treatment access for patients with ultra-rare diseases. Initially, in the first year, EspeRare plans to support the treatment of up to three patients in Switzerland who are anticipated to benefit from ASO therapies currently under clinical evaluation for n-Lorem patients with nano-rare diseases.
The n-Lorem Foundation is a non-profit organization that utilizes the effectiveness, versatility, and specificity of antisense technologies to provide free experimental antisense oligonucleotide (ASO) therapies to patients with rare diseases caused by unique gene mutations; these patients are primarily comprised of extremely rare cohorts, often numbering between one and thirty individuals, who frequently lack treatment options. Founded with the mission to inspire hope for these patients through the development of individualized ASO therapies—short strands of modified DNA precisely targeting the transcripts of defective genes to correct anomalies—n-Lorem has received over 400 treatment requests to date, approved over 200 patients with rare diseases, and currently has over 40 patients undergoing treatment.
Founded by Dr. Stanley T. Crooke, M.D., Ph.D., the former president and CEO of Ionis Pharmaceuticals, n-Lorem is a leader in RNA-based therapies known for its innovative approach.
To learn more about the mission of n-Lorem, visit nlorem.org. We welcome donations to help offer hope, future prospects, and treatment options to patients with rare nano-diseases in urgent need of assistance.
EspeRare is a non-profit organization dedicated to advancing therapies for rare diseases through strategic partnerships, translational research infrastructure, and patient-centered programs. For over a decade, EspeRare has been working to eliminate barriers that prevent promising scientific innovations from reaching patients lacking adequate care. The organization has emerged as a pioneer in developing prenatal and personalized therapies for rare diseases. By blending the agility of a biotechnology company with a socially responsible approach, EspeRare bridges the critical translational research phase, connecting patient organizations, academic researchers, industry partners, and regulatory authorities to drive therapies forward and promote equitable access. Based in Geneva, EspeRare harnesses its unique position at the intersection of science, health diplomacy, and life sciences to craft impactful collaborations that serve patients worldwide.
Learn more about our mission at esperare.org. If you are inspired by our work, please consider supporting us; your generosity enables the progress that so many families are awaiting.
Initially, the partnership will target a select group of patients with what are termed 'nano-rare' disorders in Switzerland, aspiring to develop a replicable and transferable model that could eventually extend to other European Union countries. EspeRare focuses on the development of therapeutic options for underrepresented patient groups, placing their well-being at the core of its mission and maintaining close relationships within impacted communities. By leveraging its expertise in navigating European regulatory frameworks and establishing connections with various health stakeholders—including regulatory authorities, ethics committees, and university hospitals—EspeRare will collaborate with n-Lorem to create tailored access models grounded in scientific rigor, patient safety, and solid ethical governance.
As a leader in the development of customized and experimental ASO therapies, n-Lorem currently treats the largest number of patients worldwide with rare nano-disorders. Their industrialized approach is built on substantial scientific expertise, comprehensive knowledge of ASO technologies, and years of experience creating tailored therapies for each patient. Currently, n-Lorem operates under FDA approval, and expanding international access necessitates customized regulatory and clinical solutions that consider local conditions while upholding n-Lorem’s high safety and scientific standards.
Through this collaboration, EspeRare will closely work with n-Lorem to identify European patients who could benefit from ASO therapies, which have already been safely administered to patients with rare diseases within the FDA approval framework. This includes coordinating with healthcare professionals and institutions, ensuring compliance with national regulatory and ethical standards, and integrating advanced genomic technologies to create a detailed understanding of each patient’s unique biology. The mission of n-Lorem emphasizes patient safety, which will be safeguarded throughout the entire process via rigorous scientific evaluations, independent ethical oversight, and informed clinical decision-making.
Dr. Sarah Glass, Chief Operating Officer of the n-Lorem Foundation, stated, “We at n-Lorem are committed to providing therapy access through our ASO platform to as many patients as possible. The nano-rare community has a tremendous need worldwide. This collaboration marks a critical step in expanding our mission beyond the USA. Personalized ASO therapies can only reach patients when the clinical and regulatory environments allow for it. EspeRare’s expertise in European approval systems makes it the ideal partner for responsibly extending access for patients in this region.”
Caroline Kant, director of the EspeRare Foundation, remarked, “EspeRare was founded to bridge the gap between biomedical advancements and actual patient access to therapies. Therapeutic innovation often fails at the hands of complex and fragmented health systems. Thus, we aim to leverage our expertise and network to extend n-Lorem’s ASO platform beyond the USA. Together, we aspire to make cutting-edge therapies available to families who desperately need them, redefining the possibilities of precision medicine.”
The collaboration will be overseen by a steering committee comprising representatives from both organizations, responsible for coordinating regulatory strategy, clinical engagement, and program execution. The partners aim for a multi-year collaboration to emphasize their shared commitment to building sustainable treatment pathways for personalized gene therapies focused on Europe, while reducing inequalities in treatment access for patients with ultra-rare diseases. Initially, in the first year, EspeRare plans to support the treatment of up to three patients in Switzerland who are anticipated to benefit from ASO therapies currently under clinical evaluation for n-Lorem patients with nano-rare diseases.
About n-Lorem Foundation
The n-Lorem Foundation is a non-profit organization that utilizes the effectiveness, versatility, and specificity of antisense technologies to provide free experimental antisense oligonucleotide (ASO) therapies to patients with rare diseases caused by unique gene mutations; these patients are primarily comprised of extremely rare cohorts, often numbering between one and thirty individuals, who frequently lack treatment options. Founded with the mission to inspire hope for these patients through the development of individualized ASO therapies—short strands of modified DNA precisely targeting the transcripts of defective genes to correct anomalies—n-Lorem has received over 400 treatment requests to date, approved over 200 patients with rare diseases, and currently has over 40 patients undergoing treatment.
Founded by Dr. Stanley T. Crooke, M.D., Ph.D., the former president and CEO of Ionis Pharmaceuticals, n-Lorem is a leader in RNA-based therapies known for its innovative approach.
To learn more about the mission of n-Lorem, visit nlorem.org. We welcome donations to help offer hope, future prospects, and treatment options to patients with rare nano-diseases in urgent need of assistance.
About EspeRare Foundation
EspeRare is a non-profit organization dedicated to advancing therapies for rare diseases through strategic partnerships, translational research infrastructure, and patient-centered programs. For over a decade, EspeRare has been working to eliminate barriers that prevent promising scientific innovations from reaching patients lacking adequate care. The organization has emerged as a pioneer in developing prenatal and personalized therapies for rare diseases. By blending the agility of a biotechnology company with a socially responsible approach, EspeRare bridges the critical translational research phase, connecting patient organizations, academic researchers, industry partners, and regulatory authorities to drive therapies forward and promote equitable access. Based in Geneva, EspeRare harnesses its unique position at the intersection of science, health diplomacy, and life sciences to craft impactful collaborations that serve patients worldwide.
Learn more about our mission at esperare.org. If you are inspired by our work, please consider supporting us; your generosity enables the progress that so many families are awaiting.
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