Related Experiment Video
Updated: Jul 6, 2025
Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production
Published on: August 18, 2023
Cell and Gene Therapies: Challenges in Designing Extractables and Leachables Studies and Conducting Safety
Adeyma Arroyo1, Petra Booij2, Getachew Woldemariam3
1Genentech, Inc., a member of the Roche Group, South San Francisco, CA 94080, USA.
Abstract:
Over the past decade, Cell and Gene Therapies (C>) have been an emerging therapeutic area with more than twenty C> drug products approved and over 1000 registered trials. The remarkable progress in these modalities brings new challenges for scientists who evaluate manufacturing and storage materials, including risk assessments for extractables and leachables (E&L). Establishing a business process to qualify materials for these applications is an important risk mitigation strategy in support of these assessments. Process validation verifying process performance and product quality requirements using qualified materials also ensures that leachables from the materials do not result in an impact to process and product. The authors provide an overview of available guidelines and publications relevant to E&L risk assessments that can be used to support ex vivo C> products, highlighting gaps and standardization needs in the areas of biocompatibility and extractables conditions. Finally, the authors present leachable testing strategies, relevant to the specific manufacturing and storage conditions of C> products, and safety assessment considerations for organic and inorganic chemical entities.
Insights
Cell and Gene Therapies (C>) require rigorous material risk assessments for extractables and leachables (E&L). This study reviews guidelines and proposes testing strategies to ensure C> product safety and efficacy.
Area of Science:
- Biotechnology
- Pharmaceutical Manufacturing
- Regulatory Science
Background:
- Cell and Gene Therapies (C>) represent a rapidly growing therapeutic field with numerous approved products and ongoing clinical trials.
- The expansion of C> necessitates robust evaluation of manufacturing and storage materials, particularly concerning extractables and leachables (E&L).
- Establishing formal business processes for material qualification is crucial for risk mitigation in C> development.
Purpose of the Study:
- To provide an overview of existing guidelines and publications relevant to E&L risk assessments for ex vivo C> products.
- To identify gaps and areas needing standardization in biocompatibility and extractables conditions for C> materials.
- To present practical leachable testing strategies and safety assessment considerations tailored to C> manufacturing and storage.
Main Methods:
- Literature review of current guidelines and publications on E&L risk assessments in C>.
- Analysis of standardization needs in biocompatibility and extractables testing conditions.
- Development of proposed leachable testing strategies and safety assessment approaches for C>.
Main Results:
- Identified key guidelines and publications applicable to E&L risk assessments for ex vivo C>.
- Highlighted significant gaps and the need for standardization in biocompatibility and extractables testing specific to C>.
- Outlined relevant leachable testing strategies and safety assessment considerations for organic and inorganic compounds in C>.
Conclusions:
- A structured approach to material qualification and E&L risk assessment is vital for C> development.
- Standardization in testing methodologies and conditions is required to ensure the safety and efficacy of C> products.
- The proposed strategies offer a framework for managing E&L risks in the complex C> manufacturing and storage environments.
Related Concept Videos
00:59Gene Therapy
01:20Overview Of Cell Separation And Isolation