Late last month BioMarin announced a new pre-clinical candidate for Duchenne, BMN 351. BMN 351 is a third-generation antisense oligonucleotide therapy under investigation for exon 51 skipping that is designed to target a distinct site that regulates exon 51 splicing. It is delivered intravenously (IV), or directly into a vein.
This is a new pre-clinical candidate for BioMarin, who in 2014, acquired drisapersen. In 2016, clinical development was discontinued after discussions with regulatory authorities. Since that time, BioMarin has continued pre-clinical studies on various compounds and therapeutic approaches.
We are pleased that BioMarin has continued to pursue novel therapeutics in Duchenne, and look forward to learning more about BMN 351.
Read the Clinical Program update from BioMarin here.