New orphan disease therapies from the proteome of industrial plasma processing waste- a treatment for aceruloplasminemia.

ABSTRACT: Plasma-derived therapeutic proteins are produced through an industrial fractionation process where proteins are purified from individual intermediates, some of which remain unused and are discarded. Relatively few plasma-derived proteins are exploited clinically, with most of available plasma being directed towards the manufacture of immunoglobulin and albumin. Although the plasma proteome provides opportunities to develop novel protein replacement therapies, particularly for rare diseases, the high cost of plasma together with small patient populations impact negatively on the development of plasma-derived orphan drugs. Enabling therapeutics development from unused plasma fractionation intermediates would therefore constitute a substantial innovation. To this objective, we characterized t

SUBMITTER: Zanardi A 

PROVIDER: S-EPMC10828504 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature