eBook: Expedited programs for gene and cellular therapies

9 Mar 2023

Cell, gene and tissue-based therapies have the potential to tackle some of the most problematic diseases, including rare genetic conditions and those without treatment options. For these therapies, interactions with regulators at an early stage of development can aid the approval process, thus delivering treatment to patients. However, due to regulatory authorities regularly issuing new guidelines that help with the interpretation of therapy regulations, it is integral to stay up to date with evolving standards and best practices. In this eBook, we will review expedited regulatory pathways and the best practices for the successful implementation of them as well as how to select a CRO for gene therapy studies.

CONTENTS INCLUDE:

COMMENTARY: Regulatory Considerations in Gene and Cellular Therapy Development
PERSPECTIVE: Five Criteria to Use When Selecting a CRO for a Gene Therapy Study
INTERVIEW: 60 seconds with Kimberley Buytaert-Hoefen: cell and gene therapy
WHITE PAPER: 2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 – Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62)