Target Selection
The target product, BChE, was selected based on the suitability of plant
cell culture operation for small to moderate drug indications, such as
chemical or biological defense stockpiles and rare disease treatment.
The target production level for rrBChE is 25 kg per year. With a single
dose at 400 mg, this corresponds to production of 62,500 doses of rrBChE
annually. The production level was estimated on the basis of stockpile
generation and emergency deployment. Many orphan diseases would require
similarly small production capabilities, such as alpha-1-antitrypsin
deficiency, which affects approximately 100,000 people in the US
(Stoller & Aboussouan, 2009), or amyotrophic lateral sclerosis (ALS),
commonly known as Lou Gehrig’s disease, which affects approximately
30,000 people in the US (Miller & Appel, 2017).
The large-scale biomanufacturing facility designs are based on
laboratory-scale data. However, process design inputs assume modest
improvements in culture performance and downstream recovery based on
anticipated process optimization work to be done as part of scale up to
pilot and commercial manufacturing.