As your cell or gene therapy moves toward commercialization, supply chain readiness is critical. But what does it take to develop a successful supply chain strategy to support a commercial launch? Orchard Therapeutics and Be The Match BioTherapies® have first-hand experience.
As they worked together in the year leading up to commercial readiness, Orchard and Be The Match BioTherapies found several strategies were key to adapting the supply chain from one used to support clinical studies to one for a commercial supply chain.
Partnering with a supply chain vendor with cell and gene therapy experience
For autologous cell and gene therapies, the drug product is not off-the-shelf. A patient’s cells need to be extracted, cryopreserved and then shipped to a manufacturing facility in a temperature-controlled state. Once the patient cells are gene-modified, they are again cryopreserved and shipped back to the treatment center for infusion into the patient. Chain of identity and chain of custody must be maintained throughout the process.
Speed, reliability, traceability and temperature-controlled transport are crucial.
“When it comes to manufacturing and delivering hematopoietic stem cell (HSC) gene therapy in the commercial setting, we are still in the early days. All of us are navigating this new world, so collaborating and sharing expertise are critical to moving this field forward,” said Aileen Baquiran, Vice President, Global Supply Chain and Business Operations, Orchard Therapeutics.
Orchard Therapeutics partnered with Be The Match BioTherapies. Be The Match BioTherapies supports:
- Cell harvest center qualification via auditing, training development and mock shipment training
- Delivery of patient-harvested cells to the manufacturing site
- Delivery of the gene-corrected cells back to the treatment center
“We’ve benefited from Be The Match BioTherapies’ scale, supply and vendor infrastructure,” Baquiran explained. She added, “Their many years of experience have been valuable for us as we work to maintain an efficient, high-quality and personalized supply chain.”
Focus on scalability of processes for use in commercial launches
When they began their partnership during clinical studies, both companies had an eye toward commercial launch of HSC gene therapies in Orchard’s pipeline.
“As we support the implementation of clinical infrastructure, designing processes and developing documentation, our goal is to be able to leverage a lot of that work in the commercial launch. Of course there are real differences, but it lays a groundwork that can create efficiencies for the commercial infrastructure,” explained Chris McClain, Senior Vice President, Sales and Business Development, Be The Match BioTherapies.
Constant communication between teams focused on commercial readiness
Orchard collaborated closely with the Be The Match BioTherapies team as they prepared for commercial launch.
“There is a lot of planning that goes into a commercial launch to make sure we have designed a solution that can work. It needs to work not only in a normal environment, but as COVID-19 has shown us, one where you need to improvise,” McClain said.
The teams discovered new challenges in the commercial environment that weren’t a challenge in the clinical study environment.
“Orchard is pioneering in the field as one of the earliest gene therapy companies to garner European approval, and constant communication during that period was critical. The work we did with Orchard over the course of a year provided important insights for our future work in the cell and gene therapy field. We learned a lot from each other in terms of the challenges migrating from a clinical to a commercial solution,” McClain added.
Existing knowledge of a cell harvest center’s capabilities and processes
Orchard has leveraged Be The Match BioTherapies’ infrastructure for the qualification and training of their cell harvest and treatment centers—including conducting site assessments, quality system audits, mock shipments and logistics training for sites.
“Be The Match BioTherapies already has experience working with our Qualified Treatment Centers, so when we partner with these providers, we already have the knowledge we need and we’re not starting from scratch. That has helped us ramp up quickly,” Baquiran shared.
Orchard has continued to use Be The Match BioTherapies’ established process for qualifying the cell harvest and treatment centers so they know their product will be handled in the way they had intended.
“The site qualification process has become an internal playbook of sorts for us. We are following it for all our European treatment centers. I think the process has been fairly well received by the centers with whom we’ve partnered, and we are confident with the level of diligence demonstrated throughout the process as our QTCs conduct necessary preparations to administer treatment,” Baquiran added.
A shared commitment to patients
One element of a successful partnership that often gets overlooked is the shared passion for patient care.
“With the complexity of the cell and gene therapy supply chain, sometimes you have to move mountains to overcome challenges and sustain the supply chain. We’ve had that shared commitment with Orchard from the very beginning,” McClain said. He added, “That passion for patient care has allowed us to take on big challenges together and work to surmount them in support of patients and their families.”
Put our experience to work for you
Anticipating common setbacks and actively planning for their solution requires a specialized and collaborative approach with your supply chain partner. We have more than 30 years of experience building tools and processes to successfully manage the variability of the cell and gene therapy supply chain.