Current CGT landscape and future outlook
September 16, 2025 | 2024 EDIT UNPUBLISH, Creative and Design, Digital Communications, Equitable access, Health policy and government affairs, Market Access, Medical Communications, Patient support and adherence, Sales October 22, cell and gene therapy, clinical research, medical education, pharmacy, regulatory affairs
What makes CGT unique
Cell and gene therapy (CGT) is a unique treatment that uses cells and genetic material to treat and cure diseases. Personalized to the patient, CGT’s offer longer-lasting effects than traditional medicines and can address complex diseases and rare disorders for which there are no other effective treatments.
These therapies are often classified based on how they are manufactured, specifically, whether they follow an in vivo or ex vivo process. In vivo manufacturing involves delivering genetic material directly into the patient’s body, where it modifies target cells internally. In contrast, ex vivo manufacturing entails removing cells from the patient or a donor, modifying them in a controlled laboratory setting, and then reinfusing them into the patient. This distinction relates to the method of production rather than the mode of therapeutic delivery.
Cell therapy involves the use of live cells that are modified or restored to treat disease. A key example is CAR-T cell therapy, where T-cells are engineered to recognize and attack cancer cells. While many current CAR-T therapies are autologous – using a patient’s own cells – there is a growing pipeline of allogeneic CAR-T therapies that use donor cells. Gene therapy uses genetic material to treat disease by replacing, inactivating, or introducing genes into cells, for example, genetic material is transferred to cells using a carrier like a modified virus, and the faulty DNA is repaired. The CGT landscape has seen significant growth in recent years. As of early 2024, the FDA had approved 36 gene therapies, with an additional 500 in the pipeline and expectations of 10–20 approvals annually.
Looking ahead, the CGT pipeline is robust, with projections indicating up to 66 product-indication approvals by 2032. This growth is accompanied by a diversification in therapeutic areas, with 51% of newly initiated gene therapy trials focusing on non-oncology, up from 39% the previous year. As the field continues to evolve, CGTs hold the promise of revolutionizing treatment models across a spectrum of diseases.i
CGTs demand complex logistical arrangements – they have short shelf lives, are sensitive to temperature, and rely on intricate supply chains, making timely delivery a challenge. This is especially critical for fresh (non-cryopreserved) products, which must be administered within a narrow window post-manufacture and often require same-day or next-day delivery. In contrast, cryopreserved products offer more flexibility in handling and storage but necessitate controlled freezing and thawing.
With dozens more CGTs anticipated, concerns about how healthcare systems worldwide will evaluate, finance, and deliver these therapies are growing. In high income countries (HIC), the main challenges are financial and systemic integration – how to pay for and operationalize CGTs within current systems. In low-and middle-income countries (LMIC), there are additional barriers that are more foundational, involving infrastructure, technology, and capacity. So, while both settings face “hard” challenges, HICs face major obstacles, and the complexity of their systems makes the rollout slower than expected despite having the resources.
Regulatory disparities
Regulatory and financial disparities between the U.S. and Europe significantly impact access to CGTs. The U.S. FDA has expedited pathways, allowing faster access to promising treatments, while the European Medicines Agency (EMA) follows a more centralized approval process. Additionally, Europe’s stringent health technology assessments (HTAs) require each country to independently evaluate cost-effectiveness, which can further limit access. These differences mean that in 2024 only about 50% of FDA-approved CGTsii reach the European market and the Middle East is becoming the second choice after the US for access/launch, highlighting a gap in availability that could be narrowed through aligning global regulations and more flexible pricing policies.
Expanded access
To date, Uniphar has developed go-to-market and clinical development strategies and facilitated access to innovative cell and gene treatments in over 50 countries, benefiting more than 600 patients. Uniphar’s expanded access programs are crucial in connecting patients with critical CGTs that are not yet commercially available. These programs enable patients with serious conditions to receive innovative treatments that are otherwise inaccessible.
Expanded access allows patients with serious or life-threatening conditions to obtain investigational treatments outside of clinical trials when no alternatives exist. Within this framework, Named Patient Supply (NPS) enables physicians to request unapproved or investigational medicines on a case-by-case basis for individual patients, typically before regulatory approval. Free of Charge (FOC) access provides treatments, either approved or in late-stage development, at no cost to eligible patients, often sponsored by the manufacturer, particularly in the pre-launch or early post-trial period.
Expanded Access Programs offer a more structured approach, allowing access to treatments under defined eligibility and oversight criteria, often incorporating real-world data collection to support safe and ethical use prior to full market availability. Uniphar’s access and medical teams work together to handle regulatory, medical and logistical complexities, ensuring smooth and compliant delivery. With expertise in managing high-value cross-border therapies, Uniphar understands the challenges of regulatory compliance, supply chain management, and logistics on a global scale. They ensure timely, safe delivery of CGTs – providing patients with a lifeline to advanced therapies they otherwise couldn’t access.
Scalability
As CGTs are specialist treatments treating small numbers of patients, these medicines need to be delivered globally by companies who have an established global footprint and understand the nuances across the world. Uniphar’s scalable operations in training and distribution allow it to respond effectively and its global infrastructure supports medical training, ensuring healthcare providers are well-prepared to administer CGT safely and successfully.
Uniphar’s extensive distribution network also adapts to meet demand and the expanded access program, linked to their commercialization platform, is critical in supporting successful delivery of these programs by streamlining regulatory navigation, ensuring compliant distribution across diverse geographies, and enabling early stakeholder engagement that facilitates smoother transition to full market launch. This ability to scale enables Uniphar to support the expanding CGT market, ensuring that advanced therapies reach more patients and healthcare systems as demand grows.
Uniphar is now working on a 10th cell and gene project, and as the global leading expert in cell and gene therapy access, Uniphar’s pharma division draws together multifunctional teams and infrastructure to support commercialization. By expanding access, Uniphar supports the widespread adoption of CGT, meeting global healthcare needs without compromising quality.
Key trends
- Regulatory
As CGTs evolve beyond rare diseases into treatments for broader populations, regulatory systems will face increasing pressure to adapt. Current frameworks, designed around niche therapies, may struggle to keep pace with the demands of large-scale deployment. Regulatory coordination across regions will help to accelerate access and reduce global disparities, but it must be balanced with rigorous oversight to ensure patient safety and therapeutic efficacy at scale.
- Expanded Access Programs
The anticipated expansion of CGTs to more common conditions highlights a growing need for robust, scalable EAPs. These will need to evolve from bespoke solutions to more coordinated, multi-regional efforts that support earlier patient access while clinical and regulatory pathways are still maturing. As demand grows, EAPs will become a strategic tool not only for compassionate use but also for real-world evidence generation and market readiness.
- Supply chain
To meet rising demand, the CGT supply chain must shift from boutique-scale manufacturing to global infrastructure. This includes investing in cryopreservation, cold-chain logistics, and decentralized manufacturing models that ensure timely and consistent product delivery across global markets. Scalable, flexible supply networks will be essential to reduce bottlenecks, control costs, and maintain the high standards required for these complex biologics.
Looking ahead: scaling CGT for broader populations
Investment in CGTs continues to grow, with total investments reaching $15.2 billion in 2024, marking a 30% increase on the previous year. Investors are increasingly favoring CGT offers that demonstrate potential for global expansion. Companies with scalable platforms, robust regulatory strategies, and the capability to navigate diverse healthcare systems are particularly attractive, with AstraZeneca’s acquisition of EsoBiotec for up to $1 billion demonstrating the value placed on innovative therapies with global applicability.
As cell and gene therapies evolve beyond rare disease treatments, the next article in this series will look at the challenges of scaling access, aligning global regulatory pathways, and building resilient supply chains. Future opportunities lie in expanding early access programs, strengthening cross-border logistics, and enabling regulatory harmonization to support timely delivery of these complex therapies to wider patient populations. Uniphar’s integrated capabilities across access, regulatory strategy, and commercialization uniquely position it to support this next phase of CGT growth.
Conclusion
CGTs represent a transformative approach to treating complex diseases, but widespread adoption is hindered by significant logistical, regulatory, and financial challenges. With continued innovation and global regulatory alignment, CGTs could expand beyond rare diseases to address more common conditions, bringing life-changing treatments to larger populations. Uniphar is already leading in the delivery of CGTs and is well-positioned to address future challenges, using its expertise in expanded access, scalable infrastructure, and precise delivery to make CGTs more accessible. As the demand for CGTs grows, Uniphar’s role in bridging gaps and enhancing access will be crucial for the future of personalized healthcare worldwide.
This content was provided by Uniphar
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