From a standing start in 2018, the BioPhorum Cell & Gene Therapy (C>) Phorum has made huge progress on a range of initiatives aimed at supporting the faster delivery of C> products to market. These projects will lower business and supply chain risk, and accelerate the adoption of technical solutions.
Exploiting BioPhorum’s knowledge base
A crucial part of the Phorum’s success is how it accesses the wealth of knowledge built up by the wider BioPhorum membership—knowledge that is of interest to anyone establishing a new C> production line.
For example, it has pulled together a summary of work completed by other Phorums, e.g. Drug Substance, Fill Finish and Technology Roadmapping. This includes links to more than 30 BioPhorum documents and details of more than 30 C> Phorum documents and work areas.
One of the current topics receiving some focus is the future of C> multi-product facility design, which was one of the subjects discussed at a face-to-meeting in September 2019 in North Carolina, US. This had a great turnout with 53 members from 17 companies, most of whom also made a site visit to see a C> facility and share information and thoughts about its design and operation.
Site visit – bluebird bio
The tour was hosted by bluebird bio, which is at the forefront in gene therapy and so the facility fits really well with the Phorum’s CG&T membership.
Following an $80m investment, the leading-edge, 125,000ft2 facility includes warehousing and quality control testing laboratories. The site was opened in March 2019 and contains three suites capable of producing lentiviral vector (LVV) at 200l volumes. The LVV will be used for the company’s gene and cell therapies to treat multiple myeloma.
We visited two separate microbiology labs and the warehouse that has ambient temperature, cold room and -20oC storage spaces. We were also able to view three manufacturing suites capable of producing three separate products in parallel.
Our host made the tour an incredibly open and two-way experience. Each department visited was led by a specialist who answered questions and discussed how to solve facility design problems. The company also asked for feedback on its facility. This openness was a highlight of the tour and mirrored the approach of the BioPhorum team.
“I found the C> Phorum meeting in North Carolina to be extremely interesting and useful,” said Sridhar Pennathur, Senior Director/Fellow, Biopharmaceutical Development at AstraZeneca-Biologics. “It was great to meet and interact with colleagues from various companies as it gave me an opportunity to learn from their experiences. The highlight of the meeting for me had to be the tour of the bluebird bio facility. The staff there were so generous with their time and willingness to share their knowledge.”
We then held a face-to-face meeting where a headline topic was multi-product facility design. This is a hot topic in the industry and dovetailed perfectly with the bluebird bio site visit.
C> is still a relatively new area to be commercialized and companies are considering how to successfully manufacture multiple products/batches using the complex processes of C>. Many are therefore looking at designing new facilities or modifying existing sites to enable C> manufacture while future-proofing their designs.
The members around the table were very keen to discuss and share experiences on this topic—a sign of their interest was that many discussions could not be completed in the time available. All ideas and thoughts were carefully documented and a future action list drawn up for further work within the BioPhorum teams—none of these valuable discussions will be lost.
Two presentations generated a lot of debate. The first was a case study on the design concept for a viral vector manufacturing facility, the second covered the challenges in a C> manufacturing facility using mock scenarios. This was followed by breakout groups on segregation and changeover, containment and protection, and construction design.
We also discussed environmental health and safety (EHS) and biosafety, which is critical for facility design. This centered on the importance of the safety of operators, support staff and emergency responders—but focused on commercial-scale production, which is where standards and regulations lag behind industry’s release of new products.
“The face-to-face meeting is a valuable platform for industry peers to get together and share common challenges, possible solutions and best practices,” said Sarah Yuan, Vice President, Process & Analytical Development at bluebird bio. “At the September meeting, we covered topics ranging from virus and cellular processes to facility design and EHS. The organizers employed a number of discussion formats to ensure all delegates were engaged throughout the two-day sessions.”
The group also reviewed a planned article, Environmental Health and BioSafety Risk Assessment Guidance for Commercial Scale Cell and Gene Therapy Manufacturing, detailing the EHS areas to be considered when establishing a new manufacturing facility.
Future actions include producing a summary of multi-product facility design considerations for C> (encompassing mock scenarios for C> facility designs) and a summary of the critical risks and potential mitigation actions for different C> facility types. Discussions on identifying the best solutions for the whole sector will continue throughout 2020.
For more information about the Cell & Gene Therapy Phorum, and how to become a member, please contact Helen Hay firstname.lastname@example.org