BMN 351 / BioMarin - LARVOL DELTA

Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy. (PubMed, Nucleic Acid Ther) - "BMN 351 also improved gait scores and clinical and anatomical muscle pathology parameters compared with vehicle-treated hDMDdel52/mdx mice. The pharmacologic activity and safety of BMN 351 warrant further nonclinical and clinical development."

Journal • Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • HSP90AA1

BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy. (PubMed, Nucleic Acid Ther) - "Liver samples demonstrated findings consistent with ASO accumulation, to which mice are considered especially sensitive compared to humans and other non-clinical species. These results support further non-clinical and clinical development of BMN 351."

Journal • Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • HSP90AA1

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: BioMarin Pharmaceutical

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

The antisense oligonucleotide BMN 351 durably ameliorates dystrophic phenotypes in a mouse model of exon 51–skip-amenable Duchenne muscular dystrophy (WMS 2023) - No abstract available

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Form 8-K BIOMARIN PHARMACEUTICAL For: Feb 25 (Streetinsider.com) - "BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies are underway with BMN 351, an oligonucleotide therapy that has demonstrated a high-level of protein expression in pre-clinical DMD models. The Company intends to determine timing of a potential IND filing at the end of the year based on results of ongoing IND-enabling studies."

Preclinical • Duchenne Muscular Dystrophy