#Switzerland #Geneva #EspeRare #n-Lorem #ASO Therapies

n-Lorem and EspeRare's Strategic Collaboration for Rare Genetic Diseases Access

In a groundbreaking development, the n-Lorem Foundation and EspeRare Foundation have announced a strategic collaboration that aims to enhance access to antisense oligonucleotide (ASO) therapies across Europe for patients with rare genetic disorders. This partnership aims to address the various challenges that hinder access to personalized genetic treatments outside of the United States.

The collaboration’s initial focus will be on providing treatment for a select group of 'nano-rare' patients residing in Switzerland. Nano-rare patients are defined as those with diseases caused by a single genetic defect that affects very few individuals, typically between one to thirty patients globally. With such small numbers, these patients often face significant treatment barriers due to the lack of available therapies.

EspeRare Foundation is dedicated to creating treatment options for neglected patient populations. Their mission resonates deeply with that of n-Lorem, which specializes in the discovery and development of individualized ASO medicines. Together, they plan to eliminate regulatory, operational, and diagnostic barriers to access, especially in Europe’s complex healthcare landscape.

EspeRare's extensive experience in navigating the intricacies of regulatory frameworks, ethics committees, and health systems positions them as an ideal partner for n-Lorem. As part of their collaborative efforts, the organizations will develop tailored access frameworks built on a foundation of scientific integrity, patient safety, and strong ethical governance.

N-Lorem has pioneered the use of personalized experimental ASO medications and currently holds the record for providing treatments to the largest collection of nano-rare patients worldwide. Their operational processes are built on rigorous scientific principles and a deep understanding of ASO technology, allowing them to create optimized therapies for individual patients. Up to now, n-Lorem has operated under FDA regulations in the U.S., and as they look to expand into Europe, they are committed to developing clinical and regulatory solutions that prioritize patient safety and scientific validity located within the local regulatory frameworks.

Working collaboratively, EspeRare and n-Lorem plan to identify European patients capable of benefitting from ASO therapies that already have FDA authorization. This includes coordinating with healthcare providers and institutions to ensure compliance with local regulations and ethical expectations. Moreover, they aim to integrate advanced genomic methodologies to achieve a thorough understanding of the biological underpinnings of each patient's condition.

As they look to expand their efforts, the two organizations intend to start facilitating treatment in Switzerland as a template for broader implementation throughout the European Union. The collaboration will be governed by a steering committee comprising members from both foundations to ensure alignment on regulatory strategies, clinical engagement, and program executions.

Sarah Glass, Ph.D., the Chief Operating Officer of the n-Lorem Foundation, stated, "Our goal is to enable as many patients as possible to access our state-of-the-art ASO therapies. This partnership is crucial in addressing the needs of the nano-rare community globally. We acknowledge that individualized ASO therapies can effectively reach patients only when the underlying frameworks enable such treatments."

Reflecting on their commitment, Caroline Kant, the Executive Director of EspeRare, noted, "The fundamental mission of EspeRare is to bridge the gap between scientific advancements and real-world patient needs. We recognize the urgency for innovation that transcends geographical boundaries, and we are determined to leverage our capabilities to ensure that groundbreaking treatments reach families in need across Europe."

Overall, this collaboration signifies a significant advancement in the movement towards equitable access to cutting-edge treatments for ultra-rare conditions. With plans to start patient treatments within the first year, the future trajectory of this strategic alliance holds much promise for the rare disease community in Europe. This joint effort marks an essential step in ensuring that the benefits of scientific innovation do not stop at borders but translate into tangible healthcare solutions.

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About n-Lorem Foundation

The n-Lorem Foundation is a non-profit organization that harnesses the power of antisense technology to provide experimental ASO medicines for nano-rare patients with unique genetic disorders. Founded by Dr. Stanley T. Crooke, who led Ionis Pharmaceuticals to the forefront of RNA-targeted therapeutics, the foundation aims to bring hope to individuals and families facing extremely rare conditions by creating highly specific and effective treatment options.

About EspeRare Foundation

EspeRare is a nonprofit organization committed to developing therapies for patients suffering from rare diseases through strategic collaborations and patient-centered programs. Since its inception, EspeRare has established itself as a frontrunner in advancing personalized and prenatal therapies while ensuring that promising scientific discoveries reach those who need them most. For more information, visit EspeRare's website.