How Clinical Supply Is Shaping The Next Generation Of Cell And Gene Therapies

By Rachel Grabenhofer, Chief Editor, Clinical Supply Leader

Star Trek seeded the public’s imagination with the idea that cell and gene therapies could reverse disease, repair mutations, and even alter what it means to be human. (And yes, Star Wars also gave us the healing power of The Force; Star Trek just happens to be the more relevant analogy here. My sci-fi loyalties are evenly divided.)

What the series never explored was the messy reality between possibility and practice: manufacturing, infrastructure, patient access, and the clinical supply challenges that connect them. Those operational considerations are just as critical to patient impact as the science itself, and they're shaping the next chapter of the story.

Over the last decade, cell and gene therapies have advanced from scientific promise to approved treatments, and this year has brought several reminders of that evolution. Just this month (July 2026), the U.S. Food and Drug Administration (FDA) expanded the approved use of Casgevy (exagamglogene autotemcel) to children as young as two with sickle cell disease; it was previously approved for those 12 and older.

Clinical trials confirm the autologous gene therapy is effective – and for families, it’s the medical miracle they’ve hoped for. Delivering that outcome, however, requires considerable coordination.

Personalized Therapies Meet Clinical Supply Reality

Briefly: a patient's hematopoietic stem cells are collected, genetically edited using CRISPR/Cas9, manufactured as a personalized therapy, and ultimately returned to the same patient as a one-time treatment. Every dose is patient-specific, requiring precise coordination across collection, manufacturing, logistics, and treatment scheduling.

According to the Casgevy product website, stem cell collection may require multiple visits over the course of a week (or weeks). From there, the therapy moves through a months-long manufacturing process (up to six months) – where chain-of-identity, chain-of-custody, and temperature-control requirements must be maintained throughout.

Administering the treatment is equally intensive. Patients are admitted to the hospital for chemotherapy conditioning before they receive the therapy, after which they may remain hospitalized for four to six weeks while clinicians monitor their recovery. These requirements limit their access to facilities with the specialized infrastructure and expertise necessary to support the care delivery process.

Despite these practical challenges, the transformational potential of the therapy continues to drive efforts to extend its reach. “Making this therapy available to younger patients opens a critical window for intervention and gives these children a meaningful chance at a healthier future,” said Megha Kaushal M.D., acting deputy director of the Office of Therapeutic Products in the FDA’s Center for Biologics Evaluation and Research (CBER).

The FDA's approval reflects a broader push to deliver advanced therapies to patients sooner. But as more individuals become eligible for treatment, it becomes clear that regulatory approval is only the beginning. Manufacturing capacity, treatment infrastructure, and therapy delivery models ultimately determine how many patients can access these therapies — and how quickly.

Precision Medicine's Supply Challenge: One Patient, One Product

The Casgevy example is not unique. Similar challenges are shaping the CAR-T landscape and other advanced therapies. And amid headlines about approvals and innovation, a more practical question remains: how will these therapies actually reach patients? According to Tatyana Matveeva, Ph.D., the answer has less to do with the science than one might assume.

Matveeva is the director of cGMP operations for cell therapy and regenerative medicine at Harvard Medical School and Massachusetts General Hospital. In a recent Clinical Supply Leader interview, she argued that many of the biggest barriers to advanced therapies are operational as much as they are scientific. Geography, treatment-site limitations, cryogenic storage requirements, and specialized infrastructure continue to restrict patient access.

“As the distance increases between a patient and a certified center that can administer therapy – one with the right infrastructure and cold storage – the odds of receiving treatment drop sharply,” she explained. "This is fundamentally a supply chain and infrastructure problem.”

Her observation highlights a growing reality across advanced therapies: manufacturing and access constraints are becoming just as important as clinical efficacy. The challenge is no longer simply to develop these therapies – it’s also to deliver them.

Those constraints are influencing where innovation is headed. This was evident in discussions during the May 2026 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), which focused on in vivo cell engineering and lipid nanoparticle technologies that could simplify manufacturing and delivery models for advanced therapies.

Similar work is appearing in the literature, where researchers are exploring manufacturing innovations and alternative production models aimed at improving scalability, simplifying logistics, and expanding patient access.

Together, these developments suggest something larger is underway. While manufacturing, distribution, and patient access remain essential to successful therapies, they are playing a growing role in therapeutic design. As a result, clinical supply is evolving from a function focused on execution to one that helps inform design.

In a separate conversation, Jonathan Anderson, Ph.D., described how this movement is already underway. Anderson is a scientific program officer and associate professor at the University of California, Davis, School of Medicine. He specializes in the clinical translation, regulatory strategy, and commercialization of advanced cell and gene therapies, bridging the gap between academic biomedical innovation and real-world application.

As Anderson elaborated, perhaps the clearest evidence of this shift can be found in CAR-T therapies – where operational constraints are helping drive the next generation of treatment approaches.

CAR-T's Growth Is Testing Clinical Supply Models

The current CAR-T treatment model requires collecting cells from the patient, modifying them, then returning them for administration, according to Anderson. "Let’s say I have cancer. I go in for apheresis to take blood and cells from my body. These are then shipped to a manufacturing facility where viral vectors are used to genetically engineer the cells,” Anderson explains.

Safety, quality and other tests are performed on the engineered cells before they are frozen and shipped back to the medical center. “Then I’m back in the infusion chair,” he says.

This approach creates a highly individualized manufacturing and distribution process – or as Anderson puts it, very “bespoke.” But such a model comes with significant operational consequences. "It’s one patient, one product. That’s not very efficient in terms of cost of goods, distribution, delivery, and all the mechanistic factors involved in that pipeline," he explains.

Because each treatment is manufactured for a specific patient, every collection session, manufacturing run, quality review, shipment, and infusion must be carefully coordinated. So, as patient volumes grow, the challenge becomes less about producing an individual therapy and more about executing that model at scale.

To date, approved CAR-T therapies have been used primarily to treat blood cancers, but researchers are expanding their focus, according to Anderson. "The two main categories starting to come on deck through clinical trials are autoimmune diseases and solid tumors – so that’s a much, much larger patient population,” he says.

As CAR-T therapies are explored in larger patient populations, the limitations of current supply models become increasingly difficult to ignore. According to Anderson, those pressures are helping reshape how advanced therapies are designed, manufactured, and brought to patients.

The Clinical Supply Barriers Between Approval and Access

Even when a therapy is available, patients still need access to centers equipped to deliver it safely. "Only a few specialized medical centers can handle that," Anderson emphasizes. “Patients have to be within a two-hour drive, or so, of the health center that has the expertise,” he explains.

This means that a therapy with the potential to reach a million patients might effectively reach only ~100,000 – a mere fraction.

From a business perspective, expanding access also increases the addressable market size. “So, there's financial incentive to alleviate all the pain points as much as possible,” says Anderson.

One reason these therapies are limited to specialized centers is that they don't fit neatly into traditional clinical workflows, according to Anderson. "Physicians don't want something that's a pain to execute. They want something that fits into their current clinical workflow," he says.

Unlike conventional therapies, many cell and gene therapies require cryogenic storage, specialized handling procedures, dedicated infusion centers, and highly trained clinical staff. As Anderson notes: "It's not dry storage. It's cumbersome liquid nitrogen that needs to be consistently refilled."

For clinical supply teams, those requirements extend far beyond product distribution, affecting site readiness, administration logistics, and ultimately which patients can realistically access treatment.

Designing Therapies Around Clinical Supply Realities

Increasingly, those constraints are prompting researchers to rethink the treatment model itself. According to Anderson, one emerging approach is to perform key aspects of the genetic engineering process inside the patient, potentially reducing reliance on highly individualized manufacturing and delivery networks.

“What's most exciting is in the lipid nanoparticle space,” Anderson says. “You can load the particles with different types of therapeutic modalities and inject them into the patient – and they target the correct cell type or tissue.”

Achieving that level of precision remains a major challenge, according to Anderson, but the technology has gained considerable momentum as developers build on mRNA platforms that have proven successful in other applications, including vaccines.

From a manufacturing and clinical supply perspective, the appeal is clear. Therapies built on this approach can be manufactured more easily and at a greater scale, reducing cost of goods, simplifying distribution, decreasing variability, and lessening dependence on specialized treatment infrastructure.

Most importantly, they could expand patient access. Rather than limiting treatment to a small number of specialized centers, future therapies may be delivered through a much broader network of healthcare providers. For Anderson, that potential to simplify supply and expand access is a major driver behind the industry's growing interest in these emerging platforms.

Whether that vision becomes reality remains to be seen. But the motivations behind the shift are difficult to miss. As operational constraints become more influential in therapeutic innovation, approaches that simplify manufacturing, distribution, and patient access will continue to attract attention.

Clinical Supply Is Entering the Design Phase

Today, however, manufacturing remains one of the most significant challenges facing advanced therapies. "The manufacturing platforms for cell and gene therapy are still an area of growth," Anderson notes. "Scaling in a cost-effective manner with consistency has some headwinds."

As a result, manufacturing considerations are appearing earlier in development. "Biotechs are realizing that they need to put more thoughtful consideration into their process development at the earliest stages, so they can reap the benefits once they reach the market."

For clinical supply professionals, the implication is clear: the manufacturing, logistics, and access constraints they manage today are helping inform the therapies that will enter development tomorrow.

Advanced therapies may not succeed simply because they produce better clinical outcomes. They may succeed because they are easier to manufacture, easier to distribute, easier to administer, and easier for patients to access – and that's how clinical supply is shaping what comes next.

About Johnathon Anderson

Johnathon Anderson, Ph.D., is Program Officer and Associate Professor at the UC Davis School of Medicine. He specializes in the translational development of advanced cell and gene therapies, navigating complex regulatory frameworks and CMC logistics to bridge the gap between bench science and clinical reality.