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BioMarin Pharmaceutical Enters Global Exclusive License Agreement And Collaboration With n-Lorem Foundation To Develop A First-In-Disease, Antisense Oligonucleotide Medicine For People Living With ReNU Syndrome

BenzingaJul 27, 2026, 1:10 PMBenzinga Newsdesk

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

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benzinga
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Jul 27, 2026, 1:10 PM
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Jul 27, 2026, 1:10 PM
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