Under the agreement, BioMarin and n-Lorem will collaborate to advance an investigational ASO candidate targeting the RNU4-2 (n.64_65insT) variant, which is estimated to account for approximately 75% of ReNU syndrome cases. ReNU syndrome was first discovered in 2024 by an international team of geneticists led by Dr. Nicola Whiffin at the University of Oxford's Big Data Institute and Dr. Ernest Turro at the Mt. Sinai Icahn School of Medicine. There are currently no approved medicines that address the underlying cause of disease. Both BioMarin and n-Lorem will conduct preclinical studies and collaborate to select the lead candidate to move forward in clinical studies.