We are a clinical stage company developing a compelling portfolio of innovative AAV-based genetic medicines for people with serious rare neuromuscular diseases.

Pompe is caused by mutations in the gene that encodes the enzyme GAA, which is responsible for metabolizing glycogen within the lysosome of the cell. Dysfunction or absence of functional GAA results in toxic accumulation of lysosomal glycogen. Gene replacement therapy candidate AT845 utilizes an AAV8 vector to deliver a functional copy of the gene to transfect and express GAA directly in tissues affected by the disease.

DMD is caused by mutations in the dystrophin gene, which encodes the protein dystrophin, a structural protein involved in maintaining muscle cell integrity. AAV-antisense candidates AT702, AT751 and AT753 utilize vectorized exon skipping to induce cells to skip over faulty or misaligned sections of the genetic code, which results in the expression of a more complete, functional protein.