AAV9-miniCOL4A5

AAV9-miniCOL4A5 is an adeno-associated virus serotype 9 (AAV9) vector-based gene therapy carrying a human COL4A5 minigene (miniCOL4A5). It is being developed through a collaborative research project between the University of Manchester, Duke University, and Four Points Innovation (a private company wholly-owned by affiliates of Deerfield Management). The therapy is designed to treat Alport syndrome, a rare genetic kidney disorder characterized by progressive loss of kidney function, hearing loss, and ocular abnormalities caused by mutations in the COL4A3, COL4A4, or COL4A5 genes. Because the full-length COL4A5 gene exceeds the packaging capacity of standard AAV vectors, AAV9-miniCOL4A5 utilizes a shortened 'minigene' construct. The therapy delivers the functional miniCOL4A5 gene to podocytes, restoring type IV collagen expression and assembly in the glomerular basement membrane, thereby preserving kidney function and extending survival.

Other names

AAV9-miniCOL4A5 gene therapyAAV-9-miniCOL4A5 gene therapyAAV 9-miniCOL4A5 gene therapyAAV9-miniCol4a5AAV-9-miniCol4a5AAV 9-miniCol4a5miniCOL4A5miniCOL-4A5miniCOL 4A5