BioMarin, n-Lorem partner to develop antisense drug candidate for ReNU syndrome
BMRN•ReNU syndrome market context
ReNU syndrome, identified in 2024, is projected to affect about 100,000 people globally, implying a larger-than-typical rare-disease commercial opportunity.
Preclinical work to select a lead candidate
Both groups will run preclinical studies to select a lead candidate for clinical testing. No approved therapies address the disease’s genetic cause.
BioMarin and n-Lorem begin early-stage collaboration
BioMarin entered an early-stage research collaboration with the n-Lorem Foundation to develop an investigational antisense oligonucleotide for ReNU syndrome.
The program targets the RNU4-2 (n.64_65insT) variant, estimated to represent about 75% of cases; BioMarin holds a global exclusive license.
