BioMarin, n-Lorem partner to develop antisense drug candidate for ReNU syndrome

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


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. BioMarin Pharmaceutical Inc. published the original content used to generate this news brief via PR Newswire (Ref. ID: 202607270905PR_NEWS_USPR_____SF12241) on July 27, 2026, and is solely responsible for the information contained therein.