How GENV-002 (AAV.GAA and AAV.Cas9) works
High Affinity Delivery
We utilize AAV vectors with high target tissue affinity to deliver optimized transgenes that hold the potential to serve as medicines to treat inherited diseases.
Scientific and Regulatory Strategies
Our Scientific Team has over 28 years of experience with AAV gene therapy, from vector design to transgene optimization. Our Regulatory Team has a track record of FDA approvals and has worked on over 100 programs ranging from IND to NDA and BLA. Together our Scientific and Regulatory Teams work collaboratively with our investigators conducting contract and sponsored research to assess the efficacy and safety of our gene therapy product candidates.
Pompe disease, part of a group of diseases called lysosomal storage disorders, is a genetic disorder caused by loss of functional acid alpha-glucosidase (GAA). In healthy cells, this enzyme breaks down glycogen to glucose in the lysosomes. This is an important biochemical pathway that ensures muscle tissues in the body function correctly. In patients with Pompe disease, the lack of GAA leads to glycogen buildup in the lysosomes. This results in glycogen leakage and damage to muscle fibers, progressively weakening the heart and other muscles, including those involved with breathing.
GENV-002 is a gene therapy approach using adeno-associated virus (AAV). It consists of one AAV carrying a healthy human GAA gene (“AAV.GAA Donor”) and an AAV carrying a Cas9 nuclease and a guide RNA for cleavage of the endogenous GAA genomic sequence (“AAV.Cas9”). When GENV-002 is administered, it targets the liver, resulting in functional GAA in circulation. This “liver depot” approach has been shown to reverse the biochemical abnormalities of Pompe disease in the heart and skeletal muscle, as well as suppress immune responses to GAA.
