GA-001

GA-001 is a dual-action gene therapy candidate developed by Genosera for the treatment of Wolman Disease, a severe form of Lysosomal Acid Lipase Deficiency (LAL-D). Utilizing an adeno-associated virus (AAV) vector, the therapy employs a patent-pending bicistronic construct to deliver both a gene replacement component (to address the underlying enzyme deficiency) and a muscle-building component designed to restore muscle mass and strength. The research for this therapy originated from the Center for Gene Therapy at Nationwide Children's Hospital. GA-001 is currently in the preclinical and IND-enabling stage, with a focus on stopping disease progression and reversing existing damage.

Other names

GA-001 + LAU2LAU2-Genosera-gene therapyLAU-2-Genosera-gene therapyLAU 2-Genosera-gene therapy