Partnership to advance research in rare neurodevelopmental disorders

Image via BUDDHI Kumar SHRESTHA on Unsplash

Servier, an independent international pharmaceutical company governed by a foundation, and n-Lorem Foundation, a non-profit organisation established to provide experimental medicines to patients with high unmet needs in rare diseases, are entering into a multi-target research collaboration to develop antisense oligonucleotide (ASO) therapeutic approaches to address rare neurodevelopmental disorders.

n-Lorem research teams will utilise their ASO technology platform to engineer preclinical candidates, which Servier will advance into clinical  development. ASOs are designed to target disease-causing RNA and could enable highly personalised approaches to treat genetic disorders. This approach unlocks a new frontier in medicine: addressing the root cause of disease and offering the potential to tackle previously intractable neurological conditions.

This collaboration aims to extend the reach of precision genetic medicine to patients living with rare neurological disorders, many of whom currently have no approved treatment options. The partnership also represents another step toward achieving Servier’s 2030 ambition of developing innovative treatments for people living with rare neurological diseases, where unmet medical need remains, including refractory epilepsy, genetically driven autism spectrum disorders, leukodystrophies, peripheral neuropathies, movement disorders, and neuromuscular diseases.

Nitza Thomasson, global head of R&D Neurology at Servier, said, “We are proud to partner with n–Lorem, sharing the same commitment to advancing personalised ASOs and bringing meaningful therapies to people living with rare genetic neurological disorders who have few, if any, treatment options available today. This partnership builds on a shared belief in open, multidisciplinary collaboration grounded in strong science to accelerate research and deliver new therapies for patients. By combining n-Lorem’s pioneering expertise in antisense technology with Servier’s development capabilities, we aim at accelerating new approaches for patients who have been waiting for far too long.”

Stanley T. Crooke, founder, chairman and CEO of n-Lorem, said, “We welcome Servier to our growing list of supporters and partners and look forward to contributing to Servier’s commitment to patients with rare neurological disorders and to advancing ASO technology. This is truly a win-win for patients with rare diseases.”

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