GA-004

GA-004 is an adeno-associated virus (AAV)-based gene therapy candidate being developed by Genosera for the treatment of Merosin-Deficient Congenital Muscular Dystrophy Type 1A (MDC1A). MDC1A is a severe congenital muscular dystrophy caused by mutations in the *LAMA2* gene, which encodes the laminin-α2 protein essential for muscle fiber stability and basement membrane integrity. GA-004 utilizes a gene replacement or augmentation strategy, potentially employing a 'micro-laminin' approach to deliver a truncated version of the *LAMA2* gene that fits within the packaging constraints of AAV vectors. The therapy aims to restore functional laminin-α2 expression to stop disease progression and potentially reverse existing muscle damage. The program originated from research conducted at the Center for Gene Therapy at Nationwide Children's Hospital and is currently in the IND-enabling preclinical stage.

Other names

GA-004 MDC1AGA004 MDC1AGA 004 MDC1AMerosin-Deficient Congenital Muscular Dystrophy Type 1A gene therapy