Entrada trial skipping exon 45

CureDuchenne provided early funding to Entrada Therapeutics, who has announced that they received authorization in the United Kingdom (UK) to initiate a Phase 1/2 multiple ascending dose clinical trial in individuals with Duchenne amenable to skipping exon 45.  They plan to start that trial in Q3 2025.  This is the second clinical exon-skipping program for Entrada, who has authorization for exon 44 skipping trials in the UK, Europe and United States. 

Read the press release:  https://ir.entradatx.com/news-releases/news-release-details/entrada-therapeutics-receives-authorization-united-kingdom-1

What are Exons?

Exons are segments of a gene that encode for protein. The DMD gene, which is responsible for the production of the key muscle protein dystrophin, is one of the largest human genes known and contains 79 individual exons.  
 
Mutations, such as deletions, duplications, or point mutations in the DMD gene, may prevent the production of dystrophin, leading to the progressive muscle degeneration seen in Duchenne muscular dystrophy (DMD). Treatments like exon skipping aim to allow the production of a truncated but functional dystrophin protein.