Posted by Michael Wonder on 15 Jun 2026
US FDA grants orphan drug and rare paediatric disease designations to GEn1E Lifesciences’ dual signal modulator for the treatment of Duchenne muscular dystrophy
9 June 2026 - GEn1E Lifesciences today announced that the US FDA has granted both orphan drug designation and rare paediatric disease designation to GEn-1123 for the treatment of Duchenne muscular dystrophy.
The dual designations mark important regulatory milestones for GEn1E and support the advancement of GEn-1123, a novel oral dual signal modulator designed to target key inflammatory and degenerative drivers of Duchenne muscular dystrophy progression.
