Blood centers have the donors, resources and community footprint drug sponsors need to solve supply chain issues around cell and gene therapies.
CAR T cell therapies are growing their foothold beyond cancer and into autoimmune indications. Gene editing therapies, meanwhile, offer one-and-done treatments for sickle cell disease and have proven value in rarer diseases as well.
But as cell and gene therapy (CGT) transitions from treating small cohorts at flagship academic centers to larger populations, a fundamental bottleneck has emerged: the industry’s reliance on academic medical centers and bespoke donor infrastructure for initial cell collection and delivery simply does not scale.
Whether the product is autologous (made from the patient’s own cells) or allogeneic (from a donor), the first step in any CGT pathway is cell collection, typically via leukapheresis to isolate specific immune cells that will be genetically modified or expanded in manufacturing. Exclusively using academic medical centers for this step is expensive, inefficient and exclusionary.
Biopharma companies are increasingly discovering that expanding CGTs into late-phase development and commercial use demands resources and workflows that academic hospitals were not built to provide at scale. Furthermore, for many—if not most—eligible patients, the nearest such center is a significant distance away.
For these reasons, our industry group, the Blood and Cells Advocacy Roster (BCAR), is working to help companies scale their cell and gene therapies by plugging into the proven existing infrastructure of blood centers, which have the donors, resources and community footprint drug sponsors need.
Biopharma’s non-scalable model
Companies with products in the CGT space consistently describe challenges scaling in the commercial setting. Several CGT companies have publicly struggled to build sustainable commercialization for therapies. Galapagos, for example, described its difficulty setting up manufacturing infrastructure, in particular around decentralized CAR T production. Bluebird bio, meanwhile, sold to private equity firms last year for just $50 million, despite having multiple gene therapies on the market and having once been valued at some $10 billion in 2018.
The unique challenge in this space comes down to a simple biological reality: CGT workflows begin with people. Historically, the “first mile” of CGT manufacturing—collecting and preparing patient cells—has been handled by clinical research teams at academic medical centers, reflecting its roots in bone marrow transplantation and the earliest manufacturing of cell therapies.
But with CGTs growing on the market and more moving into later clinical stages, larger and chronic indications, and first-line settings, we need a new model.
What works for a Phase 1 or an ultrarare disease is not repeatable writ large. Academic medical centers alone cannot support scale, geographic access or cost efficiency, and they risk becoming overly stretched if expected to support delivery beyond the research stage they are designed to enable.
Blood centers’ ready-made infrastructure
There is established infrastructure that is ready to fill the gap. Biopharma companies often aren’t aware that blood centers are already suited for purpose, operating at the intersection of clinical collection, regulatory compliance, quality systems and community access.
Some of these facilities and teams have experience with collection of patients’ cellular starting material, suitable for autologous workflows. Some have experience managing donor recruitment, screening, logistics and the intricacies of collecting starting material for allogeneic therapies. Their community footprint promotes patient access and lends itself to donor diversity, a condition necessary for allogeneic therapy feasibility.
Blood centers have been performing leukapheresis for years for marrow and organ transplantation and therapeutic apheresis. As a result, they are prepared to manage donor recruitment, mobilization, consent, cell collection, product traceability and documented quality control at every step, translatable to the equally regulated CGT space.
More recently, blood centers have begun demonstrating how their capabilities transfer to CGT, performing autologous and allogeneic cell collection and processing under rigorous quality regimes that align closely with good manufacturing practice (GMP) and good clinical practice (GCP) expectations.
Leveraging these services allows clinical sites to focus on patient care and drug delivery instead of managing complex manufacturing inputs, simplifying clinical site expansion and increasing patient access without new investment. Decentralizing collection and some processing can bring therapies closer to patients.
Further, blood centers have invaluable experience with standardized protocols and quality systems in a decentralized system that can enable faster tech transfer.
Overlooked no longer
The biopharma industry has only infrequently interacted with blood centers as therapeutic logistics and manufacturing partners. Organizations like BCAR are seeking to change that paradigm by formalizing cross-sector collaboration, bringing together blood and tissue collection networks, technology providers and biotherapy stakeholders to push the narrative on advanced therapy access and infrastructure. There is growing recognition that blood centers can be essential to scaling CGTs without compromising quality or regulatory compliance.
Blood centers have quietly positioned themselves for a CGT transition, leveraging advances in technology to make their ability to support a decentralized model even more robust. Modern IT systems and quality platforms now enable real-time tracking of patient, donor and product data across distributed workflows, mitigating the risk that once deterred biopharma from integrating third-party sites. In fact, this kind of logistical sophistication sits much more comfortably within networks of blood centers that are constantly engaging to ensure a nationally available blood supply than at academic institutions where local priorities drive focus.
Blood centers can function as collaborators—for example, helping develop new donor recruitment strategies with allogeneic therapy developers or supporting patient collections in community hospitals or in their existing fixed site donation facilities.
Not all blood centers have the same capabilities and fully developed service lines. They do, however, have an initial framework for supporting these therapies.With growing demand, building onto the infrastructure of these organizations remains the fastest and cheapest path to a distributed network.
BCAR is helping build a precompetitive, mission-driven ecosystem, with members representing blood centers, technology providers, equipment manufacturers and data platforms operating in complementary roles. This collaboration can support what CGT developers need: optimization focused on interoperability, standardization and patient access. Tapping into this existing infrastructure will allow hospitals and biopharma alike to move faster, treat more patients and reduce the amount of required capital investment.
We are at the very early stages of building an ecosystem that will someday parallel our delivery of prescription pharmaceuticals. Going forward, we must be creative, fast and frugal—using existing resources as much as possible and growing into new roles to serve the community.
