Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the
human glucocerebrosidase gene (GCD), which have been mapped to chromosome 1 q21-q31, leading
to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the
accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage
system. This accumulation leads to the visceral manifestations of hepatosplenomegaly, anemia
and thrombocytopenia, as well as to the skeletal features and less frequently also to lung
involvement.

prGCD is a plant cell expressed recombinant glucocerebrosidase enzyme for the treatment of
Gaucher disease. Expression of proteins in plant cell culture is highly efficient, does not
require post-expression modification of the protein, and is not susceptible to contamination
by agents such as viruses that are pathological to humans.

prGCD safety will be observed in this treatment protocol of patients with non-neuronopathic
Gaucher disease who require enzyme replacement therapy. Eligible patients will receive
intravenous (IV) infusions of prGCD every two weeks. The dose of prGCD will be equal to each
patient's previous imiglucerase dose before reduction or discontinuation due to shortage. The
infusions will be administered at the selected medical center.

Inclusion Criteria:

- Males and females, 18 years or older

- Diagnosis of Gaucher disease treated historically with imiglucerase

- Able to provide written informed consent

Exclusion Criteria:

- Currently taking another experimental drug for any condition

- History of allergy to carrots

- Previous infusion reaction suspected to be allergic in nature to Cerezyme® or
Ceredase® or receiving premedication to prevent infusion reactions

- Allergy to beta-lactam antibiotics

- Presence of any medical, emotional, behavioral or psychological condition that in the
judgment of the Investigator would interfere with the patient's compliance with the
requirements of the study.