AVRO - Avrobio: Leading The Gene Therapy Field In Fabry's Disease

Avrobio (AVRO) recently reported impressive data for its gene therapy in Fabry's disease (see the table below).

Fabry’s disease overview

Fabry’s disease is the most common lysosomal storage disease. It is an X-linked inborn error due to mutations in the alpha-Gal-A gene, cause a deficiency of the enzyme lysosomal hydrolase alpha-galactosidase A, AGA, that causes the accumulation of globotriaosylceramide (Gb3) in various organs like the heart (cardiomegaly, heart failure, coronary artery disease), nerves (neuropathic pain), kidneys (proteinuria), gastrointestinal system (diarrhea, abdominal pain), skin (telangiectasias and angiokeratomas) and CNS (stroke) in the classic presentation. Approx.