Gene therapies have demonstrated to be life-changing therapies for many devastating diseases. The drug development path is not always straightforward and regulatory guidance is often vague about the details of some requirements. One of the requirements in clinical development is to monitor vector shedding to control the potential environmental risk associated with the therapy. The assay read-out is qualitative, providing the sponsor and investigator with the relevant information about vector-shedding clearance after therapy. A sensitive and reliable method is needed in several different matrices, usually including blood, plasma, faeces, semen, urine and saliva. This webinar will cover unique requirements and challenges in developing vector shedding assays like the AAV shedding assay, with applications.
Join this webinar for a review of best practices for developing a suitable qPCR-based vector shedding assay and how that data can be used and interpreted during the clinical study.
Johannes Stanta, PhD, Global Director of Molecular & Cellular Biology (MCB), Celerion
Johannes Stanta, PhD, works at Celerion as the Global Director of Molecular & Cellular Biology (MCB) being in charge of the Lincoln, NE and Zürich, Switzerland MCB laboratories at Celerion, INC. In this role, Johannes strengthens Celerion’s service offering with GLP and GCP compliant molecular and cell biology testing to support the development of new modality therapies, such as cell and gene therapies. He previously worked at Freeline Therapeutics as Director of Bioanalysis where he has the overall bioanalytical responsibility of several gene therapy clinical trials, including the companion diagnostic analytical and clinical development. He previously worked in senior scientific leadership roles in bioanalysis and clinical laboratory at LabCorp and Hammersmith Medicines Research. He received his PhD from the University of Cambridge, focusing on the discovery of diagnostic biomarkers for neuropsychiatric disorders. He has hands-on industry experience in small and large molecule bioanalytical assay development, validation and sample analysis, supporting clinical and non-clinical programs and is also leading the European Bioanalysis Forum’s Cell and Gene Therapy interest group where Bioanalytical labs across Europe share their bioanalytical challenges and best practices.
Who Should Attend?
Senior/Principal Scientists and Associate/Senior Directors within the following research areas:
What You Will Learn
Attendees will discover:
The unique requirements for shedding assays and it’s context of use
The challenges in developing a suitable assay with different matrices
The regulatory expectations associated with a shedding assay
Celerion, a global leader in early clinical research services offers a unique combination of medical expertise, clinical operations experience and scientific excellence that gives our clients the confidence to make fast, accurate decisions about their drug development path. For fifty years, Celerion has leveraged the latest operational concepts and technologies to execute safety/tolerability, pharmacokinetic and pharmacodynamics studies in highly controlled clinic environments. These include first-in-human dose escalation, drug-drug interaction, cardiac safety, bioequivalence and bioavailability, metabolism and excretion studies as well as pharmacokinetic evaluations in patients with impaired renal or hepatic function. Celerion offers feasibility, data management, biostatistics, clinical monitoring and bioanalytical services. Our founding mission is to help our clients get their drugs to market quickly, so that they touch the lives of our family, friends and people in need around the world. For more information, please visit www.celerion.com.