Therapeutic programs

Genetic diseases

InvivoGen Therapeutics is developing the pCpG plasmids for the treatment of various genetic diseases by gene therapy. The goal is to replace the defective gene by delivering within the target organ a pCpG plasmid expressing the wild-type gene. pCpG plasmids advantages are a strong and sustained expression of the transgene and the absence of immunostimulatory activity.

Three therapeutic programs will be iniated:

Treatment of Duchenne muscular dystrophy

This X-linked inherited disorder leads to progressive muscle fiber necrosis and death during the third decade of life. Duchenne Muscular Dystrophy arises from mutations or deletions within the dystrophin gene, the longest human gene. Our strategy focuses on the replacement of the defective gene by the hydrodynamic delivery to the muscle of a pCpG plasmid expressing the full-length dystrophin cDNA under the control of a muscle-specific promoter.

Treatment of cystic fibrosis

Cystic fibrosis (CF) is a chronic genetic disease of the body's mucus glands caused by a mutation in the cftr gene. This progressive disease leads to breathing difficulties and frequently to death. Our approach is to restaure the activity of the cftr gene by transfer through intranasal instillation of a pCpG plasmid expressing the wild-type gene.

Treatment of hemophilia

Hemophilia is an X-linked genetic disease caused by mutations in the blood clotting factors VIII (hemophilia A) or IX (hemophilia B). Our startegy is to produce the defective factor in the liver by injecting directly in the organ a pCpG plasmid carrying the wild-type gene under the control of a strong liver-specific promoter.