Posted by Michael Wonder on 26 Jun 2025
Precision BioSciences receives FDA rare paediatric disease designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy
25 June 2025 - Precision BioSciences today announced that the US FDA has granted rare paediatric disease designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy.
PBGENE-DMD is Precision’s development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near-full length dystrophin protein within the body to improve functional outcomes.
