Appeal No. 2005-2739 Page 2
Application No. 09/966,893
degeneration . . . [and] accumulation of macromolecules in various tissues and organs of
the body causing these organs to function less efficiently, resulting in progressive
deterioration . . . and eventually death.” Specification, page 2.
“A number of [lysosomal storage disorders] have been treated using enzyme
replacement therapy and several clinical trials are ongoing in this area. For example,
α-Galactosidase A has been used to treat Fabry disease and glucocerebrosidase has
been used to treat Gaucher Disease” (id., page 6). Table 1 of the specification lists
dozens of lysosomal storage disorders and their associated enzyme deficiencies, as well
as supporting references.
“Insect cells . . . used for expression of foreign proteins, typically via infection with
a recombinant baculovirus, accomplish most of the same post-translational modifications
as mammalian cells, including phosphorylation, N- and O- linked glycosylation, acylation,
disulphide cross-linking, and oligomeric assembly.” Id., page 2. “Production of foreign
protein in insect cells is generally considered [to be] more cost effective and efficient . . .
[than production in] mammalian cell[s]” (id., pages 1-2), but the differences in post-
translational modifications between insect and mammalian cells are not well understood
(id., page 2). According to appellants, “[t]hese differences and their ill-defined nature are
generally considered a disadvantage of producing proteins in insect cells” (id.).
“The therapeutic activity of proteins . . . used to treat lysosomal storage disorders
is attributed primarily to the lysosomal activity of such proteins in macrophages.” Id.,
page 9. “The present invention is based on the discovery that proteins produced in
insect cells by standard baculovirus expression systems are glycosylated in a unique
way which makes them susceptible to uptake by macrophages via mannose receptors
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