Publications by authors named "B J Rup"
Introduction: Patients with hemophilia A treated with coagulation Factor VIII (FVIII) products are at risk for developing anti-FVIII antibodies. The ABIRISK Consortium aimed to provide knowledge on the formation and detection of anti-drug antibodies against biopharmaceutical products, including FVIII. Accordingly, standardized and validated assays for the detection of binding (total) and neutralizing antibodies are needed.
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- A survey by the Therapeutic Product Immunogenicity community explored immunogenicity risk assessment strategies used by participants before clinical development, spanning 5 years and focusing on in silico algorithms and in vitro assays.
- Key findings showed a trend towards using advanced tools like high-throughput in silico algorithms, human immune cell-based assays, and proteomics for effective risk assessments.
- Participants also indicated that these tools not only supported early development phases but also informed clinical strategies and improved bioanalysis efficiency.
The purpose of this article is to illustrate how performance of an immunogenicity risk assessment at the earliest stage of product development can be instructive for critical early decision-making such as choice of host system for expression of a recombinant therapeutic protein and determining the extent of analytical characterization and control of heterogeneity in co- and post-translational modifications. Application of a risk-based approach for a hypothetical recombinant DNA analogue of a human endogenous cytokine with immunomodulatory functions is described. The manner in which both intrinsic and extrinsic factors could interact to influence the relative scale of risk associated with expression in alternative hosts, namely Chinese hamster ovary (CHO) cells, Pichia pastoris, Escherichia coli, or Nicotinia tabacum is considered in relation to the development of the investigational product to treat an autoimmune condition.
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- Pegunigalsidase alfa is a new enzyme replacement therapy for Fabry disease designed to improve the duration of its effects and lower the risk of immune reactions compared to existing treatments.
- In a study with symptomatic adults, dosage levels of 0.2, 1.0, and 2.0 mg/kg were tested over 12 months, showing significant reductions in harmful substances in kidneys and stable renal function.
- The therapy demonstrated a range of plasma half-lives and low rates of severe side effects, with most adverse events being mild or moderate, suggesting it could be a significant improvement in treating Fabry disease.
Plants are a promising alternative for the production of biotherapeutics. Manufacturing in-planta adds plant specific glycans. To understand immunogenic potential of these glycans, we developed a validated method to detect plant specific glycan antibodies in human serum.
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