BioMarin and n-Lorem collaborate on ReNU syndrome
Analysis based on 7 articles · First reported Jul 27, 2026 · Last updated Jul 28, 2026
The collaboration positions BioMarin to enter a new therapeutic area for a rare genetic disorder with significant unmet need, potentially expanding its pipeline. The agreement may enhance BioMarin's growth prospects and attract investor interest in rare disease biotechnology.
BioMarin Pharmaceutical Inc. and n-Lorem Foundation announced a strategic collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a rare neurodevelopmental condition caused by variants in the RNU4-2 gene. The investigational ASO targets the RNU4-2 (n.64_65insT) variant, estimated to account for approximately 75% of ReNU syndrome cases. ReNU syndrome was discovered in 2024 by an international team led by Dr. Nicola Whiffin at the University of Oxford and Dr. Ernest Turro at the Icahn School of Medicine at Mount Sinai. There are currently no approved medicines for the condition. Under the agreement, BioMarin and n-Lorem will conduct preclinical studies and collaborate to select a lead candidate for clinical studies. BioMarin will lead development for the wider ReNU syndrome community, while n-Lorem will initiate individualized clinical trials for a subset of patients. The global population with ReNU syndrome is estimated at approximately 100,000.
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