A high-throughput test to detect C.E.R.A. doping in blood

C.E.R.A., a continuous erythropoietin receptor activator, is a new third-generation erythropoiesis-stimulating agent (ESA) that has recently been linked with abuse in endurance sports. In order to combat this new form of doping, we examined an enzyme-linked immunosorbent assay (ELISA) designed to detect the presence of C.E.R.A. in serum samples. The performance of the assay was evaluated using a pilot excretion study that involved six subjects receiving C.E.R.A. Validation data demonstrated an excellent reproducibility and ensured the applicability of the assay for anti-doping purposes. To maximize the chances of detecting the drug in serum samples, we propose the use of this specific ELISA test as a high-throughput screening method, combined with a classic isoelectric focusing test as a confirmatory assay. This strategy should make C.E.R.A. abuse relatively easy to detect, thereby preventing the future use of this drug as a doping agent

Immunogenicity Assessment of AAV-based Gene Therapies: An IQ Consortium Industry White Paper

Immunogenicity is critical to AAV-based gene therapy during discovery and development. The cross industry working groups organized by International Consortium for Innovation and Quality in Pharmaceutical Development (IQ) have focused on various aspects of critical questions related to AAV-based gene therapy. This white paper summarizes the immunogenicity potential and risks associated with recombinant AAV-based GTs based on current clinical experiences and provides a framework for bioanalytical approaches related to the immunogenicity assessment of rAAV-based GTs. The paper provides a comprehensive overview of immunogenicity risks associated with rAAV GTs, as well as describes the methodologies for monitoring pre-existing and treatment boosted/or induced immune responses (humoral vs cellular). In addition, we discuss current clinical mitigation strategies deployed to reduce immunogenicity upon administration of rAAV-based G