Immunogenicity Considerations for Therapeutic Modalities Used in Rare Diseases. (3rd December 2022)

• Record Type: Journal Article
• Authors: Fathallah, Anas M.; Oldfield, Philip; Fiedler‐Kelly, Jill; Ramadan, Abdulraouf
• Other Names: Ahmed Mariam A. guestEditor.; Bhattacharya Indranil guestEditor.
• Is Part Of: Journal of clinical pharmacology. Volume 62(2022)Supplement 2
• Page Start: S110
• Page End: S118
• Publication Date: 2022-12-03
• Subjects: ASO -- gene therapy -- immunogenicity -- protein replacement therapy -- rare diseases; Pharmacology -- Periodicals; Pharmacology -- Periodicals; Pharmacology, Clinical -- Periodicals; 615.1
• DOI: 10.1002/jcph.2166
• Languages: English
• ISSNs: 0091-2700

Abstract

Abstract: New therapeutic modalities carry with them great promise for the treatment of rare diseases. They also present unique development challenges including immunogenicity, which can impact the safety and efficacy of those new modalities. In this review, an overview of the basic function of the immune system and its possible interaction with new therapeutic modalities is presented. A juxtaposition of immunogenicity in the rare disease space versus traditional clinical programs is hereby being proposed. A clinical pharmacology viewpoint of immunogenicity, proposed approaches to account for immunogenicity in clinical data, bioanalytical considerations, and effects of route of administration and production changes on immunogenicity are discussed.