Servier and the n‑Lorem Foundation have launched a multi‑target collaboration to develop antisense oligonucleotide (ASO) therapies for rare genetic neurodevelopmental disorders, expanding Servier’s rare neurology pipeline and advancing precision RNA‑targeted medicines for nano‑rare patients.
Written By: Sana Khan, BPharm
Reviewed By: Pharmacally Editorial Team
Servier and the n-Lorem Foundation have entered a multi-target research collaboration to develop antisense oligonucleotide (ASO) therapies for rare genetic neurodevelopmental disorders. The partnership combines n-Lorem’s individualized ASO platform with Servier’s neurology development expertise to advance RNA-targeted therapies for patients with severe genetic conditions who have few or no approved treatment options.
Expanding Rare Neurology Pipeline
Under the partnership, n-Lorem will identify and engineer preclinical ASO candidates, while Servier will advance selected programs into clinical development. The collaboration strengthens Servier’s rare neurology portfolio, part of its broader strategy to expand rare disease assets by 2030 and enhances n-Lorem’s capacity to deliver therapies for patients affected by highly individualized genetic mutations.
ASO Technology Targets Root Cause of Disease
Antisense oligonucleotides are short synthetic nucleic acid sequences that bind disease-causing RNA and modulate gene expression. Their ability to target specific genetic mutations makes them particularly relevant in neurology, where conventional drug development often struggles to address the needs of ultra-rare patient populations.
The technology offers a precision medicine approach that can directly address the underlying molecular drivers of disease, creating opportunities to develop therapies for disorders that previously had limited treatment prospects.
Focus on High-Need Indications
The collaboration will prioritize conditions with significant unmet medical need, including refractory epilepsies, genetically driven autism spectrum disorders, leukodystrophies, peripheral neuropathies, movement disorders, and neuromuscular diseases. These programs will remain individualized and preclinical in scope, reflecting n-Lorem’s mission to support patients who often fall outside traditional drug development pathways.
Addressing Nano-Rare Patient Needs
n-Lorem specializes in developing individualized ASO medicines for nano-rare patients, a population typically affected by genetic mutations found in fewer than 30 individuals worldwide. These patients frequently lack access to approved therapies and are often overlooked by conventional commercial drug development programs.
To date, n-Lorem has received more than 440 applications for treatment consideration and approved over 240 nano-rare patient programs. Through the collaboration, Servier provides a pathway for advancing promising ASO candidates beyond early research and into formal clinical development.
Expanding Access to Genetic Medicines
Nitza Thomasson, Global Head of R&D Neurology at Servier, highlighted the shared commitment to advancing personalized ASO therapies for patients with rare genetic neurological disorders. She noted that combining n-Lorem’s antisense expertise with Servier’s development capabilities could help accelerate new treatment approaches for underserved patient populations.
Stanley T. Crooke, founder, chairman, and chief executive officer of n-Lorem, described the agreement as an important step in advancing antisense technology while expanding treatment opportunities for patients with rare diseases. He also emphasized the partnership’s potential to increase the number of patients who can benefit from n-Lorem’s ASO programs.
Translating Research into Patient Impact
The collaboration establishes a framework for generating multiple ASO candidates across rare neurodevelopmental disorders, with Servier responsible for advancing promising programs into clinical testing. As precision genetic medicine continues to evolve, the partnership could expand access to individualized RNA-targeted therapies for patient populations historically underserved by conventional drug development models.
The agreement also supports Servier’s 2030 objective of expanding its rare neurology pipeline across genetically defined neurological disorders and reinforces the growing role of antisense technology in addressing areas of significant unmet medical need.
Reference
servier-partnership-n-lorem_PR.pdf
Servier and n-Lorem Foundation join forces to advance research in rare neurodevelopmental disorders
About the Writer
Sana Jamil Khan (LinkedIn) is a B. Pharm graduate with a strong interest in medical writing and scientific communication. Her work focuses on interpreting clinical research, exploring developments in pharmaceutical science, and presenting complex medical information in a clear and accessible manner. She is particularly interested in topics related to human clinical studies, drug safety observations, and emerging therapeutic research.