Reducing Risk for Gene Therapy Commercial Manufacturing
In biotherapeutic development today, a critical element of commercialization is reducing manufacturing risk. Serving as many patients as possible, as quickly and efficiently as possible, requires a particular focus on three key areas:
· Manufacturing
· Process Development
· Quality Control
Andelyn’s long, pioneering history with gene therapy modalities and adeno-associated virus (AAV) therapies enables researchers to leverage its legacy of expertise to accelerate and scale development. Researchers at Nationwide Children’s Hospital developed Zolgensma, the first systemic gene therapy approved by the FDA.
Andelyn’s CDMO services are structured around four corepillars:
· Starting Materials: including research, tox, and GMP plasmid manufacturing
· Development: including process development, tech transfer, and process optimization
· Manufacturing: including research, tox, and GMP of adherent and suspension processes of up to 2,000 L
· Fill/Finish: including program-specific fill volumes and vial configurations, CZ vials, and primary labeling and packaging
Leveraging Critical Control Strategies to Optimize Late-Stage Manufacturing
Andelyn has positioned itself for agility by conducting most of its testing in-house and establishing redundancy for certain equipment across its development activities and GMP manufacturing. For example, Andelyn uses the same bioreactor platforms in its process development lab and manufacturing facility, enabling one-for-one tech transfer of a process. The proximity of its Ohio facilities also allows teams to collaborate closely during tech transfer and build a cross-functional understanding of how an application performs in both settings. Its manufacturing activities, which encompass adherent and suspension platforms and its fill/finish capabilities round out a comprehensive approach to end-to-end scale-up and commercialization. Its quality assurance organization oversees every phase of production in these facilities, and its regulatory support experts are on hand to verify every stage of scale-up.
Andelyn’s proven industrialization platform is built on decades of experience in development and GMP manufacturing. It leverages a Design of Experiments (DoE)-based approach to optimize production parameters, enabling Andelyn to establish a platform that maintains titers across multiple scales, from 125 mL flasks to 2,000 L bioreactors. With the flexibility to accommodate multiple serotypes and to deliver consistently high yields, recovery, and purity across scales, Andelyn can ensure seamless scaling from preclinical to commercial.
Process development and scale-up present technical challenges that require comprehensive strategies. At Andelyn, operators use the ICH Q8, Q9, and Q10 guidelines as the foundation for product development and commercialization. Andelyn then works closely with clients to develop a strategy tailored to their Process Performance Qualification (PPQ) or Biologics License Application (BLA) goals. Andelyn's phase-appropriate approach to Q8 includes developing a comprehensive process map and description, establishing an analytical testing strategy, assessing raw materials, outlining a raw material strategy, performing Process Failure Mode and Effects Analysis (PFMEA), and establishing process controls. For Q9, Andelyn focuses on performing risk assessment, establishing risk visibility across the organization, and creating a business continuity plan. Finally, its Q10 approach is supported by a common electronic platform for managing change control, deviations, and documentation, along with quality culture initiatives, environmental monitoring, internal audits, supplier management, training, and quality council.
Ultimately, Andelyn creates a strong manufacturing paradigm for its clients by following a quality-by-design approach that leverages modular and customized platforms, high-throughput technologies, and robust analytics. By employing FMEA studies, analytical method qualification, scale-down models, intermediate and media stability, and DoE studies, Andelyn focuses on critical parameters to help clients achieve the optimal end product and manufacture it consistently across scales in an expedited, cost-effective manner.
Download the white paper below.
In biotherapeutic development today, a critical element of commercialization is reducing manufacturing risk. Serving as many patients as possible, as quickly and efficiently as possible, requires a particular focus on three key areas:
· Manufacturing
· Process Development
· Quality Control
Andelyn’s long, pioneering history with gene therapy modalities and adeno-associated virus (AAV) therapies enables researchers to leverage its legacy of expertise to accelerate and scale development. Researchers at Nationwide Children’s Hospital developed Zolgensma, the first systemic gene therapy approved by the FDA.
Andelyn’s CDMO services are structured around four corepillars:
· Starting Materials: including research, tox, and GMP plasmid manufacturing
· Development: including process development, tech transfer, and process optimization
· Manufacturing: including research, tox, and GMP of adherent and suspension processes of up to 2,000 L
· Fill/Finish: including program-specific fill volumes and vial configurations, CZ vials, and primary labeling and packaging
Leveraging Critical Control Strategies to Optimize Late-Stage Manufacturing
Andelyn has positioned itself for agility by conducting most of its testing in-house and establishing redundancy for certain equipment across its development activities and GMP manufacturing. For example, Andelyn uses the same bioreactor platforms in its process development lab and manufacturing facility, enabling one-for-one tech transfer of a process. The proximity of its Ohio facilities also allows teams to collaborate closely during tech transfer and build a cross-functional understanding of how an application performs in both settings. Its manufacturing activities, which encompass adherent and suspension platforms and its fill/finish capabilities round out a comprehensive approach to end-to-end scale-up and commercialization. Its quality assurance organization oversees every phase of production in these facilities, and its regulatory support experts are on hand to verify every stage of scale-up.
Andelyn’s proven industrialization platform is built on decades of experience in development and GMP manufacturing. It leverages a Design of Experiments (DoE)-based approach to optimize production parameters, enabling Andelyn to establish a platform that maintains titers across multiple scales, from 125 mL flasks to 2,000 L bioreactors. With the flexibility to accommodate multiple serotypes and to deliver consistently high yields, recovery, and purity across scales, Andelyn can ensure seamless scaling from preclinical to commercial.
Process development and scale-up present technical challenges that require comprehensive strategies. At Andelyn, operators use the ICH Q8, Q9, and Q10 guidelines as the foundation for product development and commercialization. Andelyn then works closely with clients to develop a strategy tailored to their Process Performance Qualification (PPQ) or Biologics License Application (BLA) goals. Andelyn's phase-appropriate approach to Q8 includes developing a comprehensive process map and description, establishing an analytical testing strategy, assessing raw materials, outlining a raw material strategy, performing Process Failure Mode and Effects Analysis (PFMEA), and establishing process controls. For Q9, Andelyn focuses on performing risk assessment, establishing risk visibility across the organization, and creating a business continuity plan. Finally, its Q10 approach is supported by a common electronic platform for managing change control, deviations, and documentation, along with quality culture initiatives, environmental monitoring, internal audits, supplier management, training, and quality council.
Ultimately, Andelyn creates a strong manufacturing paradigm for its clients by following a quality-by-design approach that leverages modular and customized platforms, high-throughput technologies, and robust analytics. By employing FMEA studies, analytical method qualification, scale-down models, intermediate and media stability, and DoE studies, Andelyn focuses on critical parameters to help clients achieve the optimal end product and manufacture it consistently across scales in an expedited, cost-effective manner.
Download the white paper below.
In biotherapeutic development today, a critical element of commercialization is reducing manufacturing risk. Serving as many patients as possible, as quickly and efficiently as possible, requires a particular focus on three key areas:
· Manufacturing
· Process Development
· Quality Control
Andelyn’s long, pioneering history with gene therapy modalities and adeno-associated virus (AAV) therapies enables researchers to leverage its legacy of expertise to accelerate and scale development. Researchers at Nationwide Children’s Hospital developed Zolgensma, the first systemic gene therapy approved by the FDA.
Andelyn’s CDMO services are structured around four corepillars:
· Starting Materials: including research, tox, and GMP plasmid manufacturing
· Development: including process development, tech transfer, and process optimization
· Manufacturing: including research, tox, and GMP of adherent and suspension processes of up to 2,000 L
· Fill/Finish: including program-specific fill volumes and vial configurations, CZ vials, and primary labeling and packaging
Leveraging Critical Control Strategies to Optimize Late-Stage Manufacturing
Andelyn has positioned itself for agility by conducting most of its testing in-house and establishing redundancy for certain equipment across its development activities and GMP manufacturing. For example, Andelyn uses the same bioreactor platforms in its process development lab and manufacturing facility, enabling one-for-one tech transfer of a process. The proximity of its Ohio facilities also allows teams to collaborate closely during tech transfer and build a cross-functional understanding of how an application performs in both settings. Its manufacturing activities, which encompass adherent and suspension platforms and its fill/finish capabilities round out a comprehensive approach to end-to-end scale-up and commercialization. Its quality assurance organization oversees every phase of production in these facilities, and its regulatory support experts are on hand to verify every stage of scale-up.
Andelyn’s proven industrialization platform is built on decades of experience in development and GMP manufacturing. It leverages a Design of Experiments (DoE)-based approach to optimize production parameters, enabling Andelyn to establish a platform that maintains titers across multiple scales, from 125 mL flasks to 2,000 L bioreactors. With the flexibility to accommodate multiple serotypes and to deliver consistently high yields, recovery, and purity across scales, Andelyn can ensure seamless scaling from preclinical to commercial.
Process development and scale-up present technical challenges that require comprehensive strategies. At Andelyn, operators use the ICH Q8, Q9, and Q10 guidelines as the foundation for product development and commercialization. Andelyn then works closely with clients to develop a strategy tailored to their Process Performance Qualification (PPQ) or Biologics License Application (BLA) goals. Andelyn's phase-appropriate approach to Q8 includes developing a comprehensive process map and description, establishing an analytical testing strategy, assessing raw materials, outlining a raw material strategy, performing Process Failure Mode and Effects Analysis (PFMEA), and establishing process controls. For Q9, Andelyn focuses on performing risk assessment, establishing risk visibility across the organization, and creating a business continuity plan. Finally, its Q10 approach is supported by a common electronic platform for managing change control, deviations, and documentation, along with quality culture initiatives, environmental monitoring, internal audits, supplier management, training, and quality council.
Ultimately, Andelyn creates a strong manufacturing paradigm for its clients by following a quality-by-design approach that leverages modular and customized platforms, high-throughput technologies, and robust analytics. By employing FMEA studies, analytical method qualification, scale-down models, intermediate and media stability, and DoE studies, Andelyn focuses on critical parameters to help clients achieve the optimal end product and manufacture it consistently across scales in an expedited, cost-effective manner.
Download the white paper below.
