Pioneering Gene Therapy Facility Design for CHOP’s Clinical Vector Core

Genesis AEC played a central role in the pioneering Clinical Vector Core (CVC) project for the Children’s Hospital of Philadelphia (CHOP), which aimed to revolutionize the production and accreditation of clinical-grade viral vectors, specifically Adeno-associated virus (AAV) and Lentivirus (LV) vectors, crucial for transformative medical treatments during Phase I/II clinical trials. Our expertise spanned crucial aspects.

Genesis AEC’s Crucial Role in Transforming Clinical Vector Production

Firstly, we provided meticulous guidance on adhering to Current Good Manufacturing Practice (cGMP) regulations. This ensured that the facility was meticulously designed and operated in accordance with stringent quality and safety standards. Notably, our team’s expertise extended to layout and design, where we orchestrated the Heating, Ventilation, and Air Conditioning (HVAC) systems to create controlled environments essential for these viral vectors. Our commitment to adhering to cGMP guidelines led us to execute a comprehensive risk assessment, which anticipated potential challenges and resulted in a robust operational framework aligned with regulatory compliance.

Ensuring cGMP Compliance in a Cutting-Edge Gene Therapy Facility

A distinctive facet of our contribution was the creation of a comprehensive Type C package, presented to the FDA’s CBER division. Through meticulous attention to detail, our team effectively communicated the project’s compliance and merits, playing a pivotal role in securing approval. The CVC project was not without unique design challenges. The subterranean setting of the facility introduced complexities affecting construction, design, and environmental controls, which our adept team navigated to ensure seamless operations. Moreover, the integration of the CVC within the surrounding vivarium demanded innovative solutions and meticulous planning to ensure coexistence.

Securing FDA Approval and Presentation to the FDA’s CBER Division

Despite these intricate challenges, the project was a resounding success. The CBER swiftly granted approval post the Type C presentation, underscoring the project’s excellence and adherence to regulations. In essence, our involvement in the ambitious Clinical Vector Core project showcased our adeptness in tackling intricate design challenges, meticulous adherence to regulatory guidelines, and our role in catalyzing transformative medical advancements.

This project was a recipient of the ISPE Facility of the Year Award (FOYA).

Glossary

• cGMP (Current Good Manufacturing Practice): Regulatory guidelines that ensure products are produced and controlled to set quality standards.
• AAV (Adeno-associated virus) & LV (Lentivirus) Vectors: Types of viruses used to deliver genes into cells during gene therapy.
• Type C Package: A regulatory submission to the FDA that addresses specific questions and concerns about a product or process.
• CBER (Center for Biologics Evaluation and Research): A center within the FDA that regulates biological products for human use.