What Will It Take To Turn CGT Innovation Into Market Success?
By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On LinkedIn
Cell and gene therapies continue to move through clinical development and into commercialization, but regulatory approval is only one milestone on the path to patients. Demonstrating durable benefit, establishing a sustainable cost structure, securing reimbursement, and building the infrastructure to deliver these therapies all present significant challenges. At Advanced Therapies USA 2026 in Boston, a panel of industry leaders discussed what it will take to turn promising science into therapies that can reach patients and succeed in the market.
Moderated by Gwendolyn Binder, Ph.D., President of Science and Technology at Cabaletta Bio, the session, “Navigating CGT Landscape, Opportunities and Challenges for Driving Market Success,” brought together Tom Leitch, CTO of KSQ Therapeutics; Sulagna Bhattacharya, CEO of Nanoscape Therapeutics; Anne Brooks, SVP of Global Access, Operations and Proleukin at Iovance Biotherapeutics; Joanna Rossell, SVP and GM of Regenerative Medicine at Sumitomo Pharma America; and Chris Mason, Ph.D., Professor of CGT at University College London and Founder of Ori Biotech.
Their discussion made clear that our industry cannot evaluate success through clinical data alone. Development, manufacturing, reimbursement, diagnosis, and delivery need to be considered as connected parts of the same strategy.
Build the Value Case around Durable Patient Benefit
For developers working on one-time therapies, the reimbursement conversation starts well before approval. Payers need evidence that a treatment can deliver lasting benefit and change the course of a disease. That can be difficult when a therapy enters a new category without established precedents.
Bhattacharya pointed to four years of follow-up data from Nanoscape’s randomized trial as an important part of its value proposition. For patients with degenerative retinal disease, preserving or restoring vision can also mean regaining independence. Many currently rely on a cane, a guide dog, or caregivers. She said the company has been engaging payers, regulators, and patient advocacy groups to communicate both the clinical evidence and the impact on patients’ lives.
Rossell described a different example in ultra-rare congenital immune deficiency. Sumitomo Pharma’s tissue-based therapy has a long history of clinical experience, supported by 25 years of study at Duke University. That experience has helped demonstrate durability and build a reimbursement case. But Rossell emphasized that most developers will not have decades of data when they approach payers.
Her advice was to make a long-term evidence commitment early. Companies need to follow patients, continue demonstrating outcomes, and invest in health economics research. Payers want to see that a therapy changes the disease trajectory, not simply that it produces an initial response. Extrapolating from limited data is unlikely to replace a plan for demonstrating sustained benefit.
Mason reinforced the importance of following the first patients treated. “The best patient we ever treat is our first patient because that sets the durability,” he said. But retaining contact with patients over time can be difficult. He described follow-up as a major challenge, particularly when patients move, change providers, or become difficult to track.
Make Manufacturing and Cost Part of the Product Strategy
The panel also challenged developers to think about cost and manufacturing much earlier. Manufacturing is a major expense, but Mason cautioned against treating it as the only lever available to improve affordability.
He argued for a holistic approach that begins when a company first defines its target product profile. Developers should consider how they will make the product, how they will design the clinical trial, and how they will move through each phase of development. Taking advantage of regulatory and development efficiencies can shorten timelines and reduce spending. Lower development costs can, in turn, reduce the financial burden that a therapy must recover commercially.
Mason’s point was that every step in the chain deserves scrutiny, from early discovery through delivery to patients. Manufacturing improvements matter, but they cannot compensate for inefficient decisions elsewhere in development.
Binder added another important consideration. Manufacturing changes between early and late development are common, but companies need to invest in demonstrating comparability. That work can help preserve the value of earlier clinical data, particularly when those data contribute to long-term durability assessments.
Diagnose Earlier and Change the Treatment Conversation
Early diagnosis emerged as another essential component of market access. For some therapies, the opportunity to preserve function or prevent disease progression depends on identifying patients before irreversible damage occurs.
Brooks described the challenge of introducing tumor-infiltrating lymphocyte therapy into melanoma care. Oncologists are accustomed to treating cancer based on disease progression and established treatment pathways. Cell therapy requires a different mindset. Patients may have better outcomes when they receive treatment before their disease has advanced too far.
“The stronger way to treat a patient with a cell therapy is when the cancer is weak,” she said. She compared the approach with transplantation, where patient condition and timing can influence outcomes. Education is therefore central to helping treating physicians understand when to refer patients for cell therapy.
Rossell highlighted the additional complexity of identifying patients with ultra-rare diseases. Companies cannot rely solely on specialist physicians to recognize cases. Patient advocacy organizations, professional societies, healthcare providers, and government agencies can all help improve awareness and diagnosis.
She described working with broader disease organizations to build education around congenital immune deficiency, even when those groups were not originally focused on the condition. The company has also engaged with healthcare systems and governments around newborn screening.
Mason raised the potential for broader genomic screening as sequencing costs continue to decline. He also challenged the industry to think further upstream about gene therapy, including whether treatment before symptoms appear could prevent disease pathology from developing. That possibility raises important ethical and clinical questions, but it also points to how early diagnosis could reshape the value proposition for some therapies.
Expand Treatment Access Beyond the Approval Milestone
Brooks offered a practical look at what scaling access requires after approval. Since launching its TIL therapy in the United States in February 2024, Iovance has authorized more than 100 treatment centers. The company has focused on reducing the distance patients must travel while expanding beyond major academic hospitals.
She said approximately 95% of the addressable U.S. patient population lives within 200 miles of an authorized treatment center. More than 80% now live within 100 miles. More than one-third of the onboarded centers are community-based, reflecting an effort to make treatment available in settings closer to where patients receive care.
That expansion has required flexibility. Not every center has the infrastructure of a major academic institution. Iovance has adapted its authorization program and developed solutions for product storage, delivery, and other operational requirements. International expansion adds considerations such as tissue licenses, customs clearance, and country-specific regulations. The company has also adjusted its ordering platform to account for additional delivery time when scheduling manufacturing slots.
Access is not achieved simply by securing approval or establishing a treatment center network. Companies must build the operational systems that make treatment feasible for patients and providers.
Treat Commercial Readiness as a Development Priority
Across the discussion, the panel returned to a common theme. Market success depends on decisions made long before commercialization. Developers need to establish how they will demonstrate durable benefit, control costs, identify patients, educate providers, and deliver treatment across different healthcare settings.
Indeed, these challenges cannot be addressed in isolation or left until a program approaches approval. A strong clinical result is an essential starting point, but it does not guarantee reimbursement or patient access. The companies best positioned for success will be those that build the evidence, manufacturing strategy, and delivery model alongside the science.