Meet the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia: A 2021 Facility of the Year Honorable Mention Award Winner

The 2021 Facility of the Year Award (FOYA) Honorable Mention category recognizes projects that did not win a specific category but were clearly successful projects that overcame significant challenges in planning, execution, and delivery.

The Raymond G. Perelman Center for Cellular and Molecular Therapeutics is one of three Honorable Mention Award winners this year. The other two are Biocon Biologics and Locus Biosciences.

The Raymond G. Perelman Center for Cellular and Molecular Therapies (CCMT) located at the Children’s Hospital of Philadelphia (CHOP) particularly appealed to the judges because of its impact on the future of cell and gene therapy facilities. In addition to being a thought-provoking example of integrating R&D, manufacturing, and treatment in one environment, the facility delivered a high-quality design with strong infrastructure capabilities and redundancy. A bonus was the ability to leverage the existing city and campus infrastructure, thus improving resiliency and cost while also putting shelled, but exiting, space to use.

FOYA Judges

Saving Young Lives

Children’s Hospital of Philadelphia (CHOP) is the nation’s first hospital devoted exclusively to the care of children. CHOP’s Research Institute’s advanced breakthrough treatments and innovations have changed lives and pushed pediatric scientific knowledge forward. Families from all over the world bring their children to CHOP when they are facing complex medical conditions.

The CCMT is dedicated to the manufacturing of viral vectors in support of Phase I and Phase II clinical trials in compliance with current Good Manufacturing Practice (cGMP). The CCMT aims to be in the forefront of pediatric medicine by fostering a multidisciplinary approach to leading edge research endeavors that will lead to a variety of applications in the treatment of inherited and acquired disorders.

Fitting new manufacturing and research space into an already existing space is always a challenge and fitting manufacturing suites, designed to produce personalized gene therapies, into an existing building was no exception. But architects, engineers, and scientists involved with the project worked together to ensure the quality of the design and the safety of the drugs manufactured in it. The project team developed a rigorous risk assessment program where risks were categorized by numerical values based on risk to a product and severity of the effect. The analysis provided them with a document they were able to reference throughout the design process and use to demonstrate the reliability of the project to the FDA. This analysis and assessment was crucial to the success and design of a facility that maximizes reliability for generating human drug products.

The facility’s systems are capable of maintaining 100 percent uptime and designers did not leave anything to chance – the team included extra redundancy for power supply to the facility. If the building’s existing humidification systems fail, a redundant chilled water system is ready. The team was also able to use existing infrastructure and add additional layers of back-up systems throughout.

The Raymond G. Perelman Center for Cellular and Molecular Therapeutics has manufactured viral vectors in support of over thirty clinical trials in ten countries with more than 550 patients treated. These ground-breaking scientific clinical trials include treatments for hemophilia, chronic lymphoid leukemia, retinal degenerative diseases, and pancreatic cancer.

Over the past ten years, CHOP researchers have seen the demand for viral vectors for human use increase. Proximity to the basic research being done at our hospital has given the CCMT an advantage in being able to develop personalized gene therapies and get them to patients immediately. To support this rapid translation from laboratory bench to hospital bedside, the CCMT has the resources to manufacture a wide range of viral and non-viral vectors to transfer genes of interest and the capacity to separate and manipulate human cells to make gene therapy trials possible.

The facility was also designed to support multiple trials simultaneously. The HVAC system was designed to handle multiple campaigns in the facility and separated room supply and exhaust streams can be sealed so individual suites can be decontaminated as needed. CCMT does not have to shut down the whole facility during decontamination but can seal off specific rooms.

As with any successful institution, staff recruitment and retention are critical components to the success of CCMT. To that end, the project team designed the space to be appealing to today’s workforce while still meeting rigorous manufacturing and safety requirements. High ceilings and hundreds of windows give the facility a light and airy feeling while helping staff navigate from area to area. Designers also added elements of wellness and humanity throughout the sterile facility, including lines of sight that allow workers inside and outside of the sterile and laboratory areas to stay connected. The application of splashes of color personalize the otherwise sterile looking manufacturing suites.

“In this facility, we make the tools – the vectors – that scientists use to deliver cell and gene therapies, bringing dramatic precision medicine treatments to patients,” said Madeline Bell, President and CEO, Children’s Hospital of Philadelphia. “The new facility is allowing us to continue our mission in the growing field of gene therapy and supporting the new generation of gene therapy clinical trials.”

Nominate Your Facility for the 2022 FOYA Program!

Apply for a chance to join this prestigious list of innovative game-changers and let ISPE honor your organization as a 2022 FOYA program winner. Applications must be submitted by 19 November 2021.

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