1 February 2019


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ADN-LYS is a preclinical product candidate in discovery-preclinical phase which ADIENNE is developing as a biosimilar (generic biologic drug) ERT for the treatment of Gaucher Disease (“GD”). GD is the most prevalent lysosomal storage disorder, a severe congenital metabolic disease that results from defects in lysosomal function due to absence or malfunction of glucocerebrosidase.

Clinical manifestations of Gaucher Disease include splenomegaly, hepatomegaly, anemia, thrombocytopenia, skeletal pathology (susceptibility to fractures), and pulmonary hypertension/infiltration. Treating the disease by substituting the deficient enzyme prevents the development of these typical disease manifestations.

Other Molecules in Development