Our global advanced therapy medical product charging stations and capabilities — 2024 Q1 update

The healthcare landscape is witnessing transformative growth with the advancement of cell and gene therapies (CGTs). With an expanding development pipeline and an increasing number of approvals, these breakthrough therapies are heralding new hope for patients across the globe. However, this rapid growth also presents unique challenges, particularly in logistics and delivery. The burgeoning CGT field underscores the importance of efficient cryogenic shipping solutions in supporting this vital industry.

In this article we will explore:

• The key stats of the gradually expanding CGT development pipeline
• Recent market approvals
• The necessity of a global network of liquid nitrogen recharging stations to support industry growth

What cell and gene therapies were approved in 2024?

The first quarter of 2024 has seen a continuation of the remarkable growth in the cell and gene therapy sector, with several significant approvals marking this period as a time of vibrant progress. Building on the achievements of the previous year, three new therapies have been given the green light across different regions, demonstrating a global commitment to advancing these innovative treatments.

In China, a new CAR-T therapy known as CT-053 (CARsgen) has been approved for myeloma, offering hope to patients battling this challenging condition. Meanwhile, Canada has embraced Beqvez (Pfizer), an AAV gene therapy for hemophilia B, expanding treatment options for those affected by this bleeding disorder. Additionally, the United States has welcomed Amtagvi, an autologous cell therapy that promises to advance the treatment landscape for melanoma patients.

With these recent approvals, we now count 32 gene therapies—including genetically modified cell therapies—globally available for clinical use. RNA therapies have also reached an impressive milestone with 28 approvals to date. The non-genetically modified cell therapy space is not far behind; with Amtagvi's approval in Q1 2024, there are now 68 such treatments accessible to patients worldwide. This surge in therapeutic options is complemented by a robust pipeline: over 4,000 gene, cell, and RNA therapies are currently under development.

Clinical trials too are witnessing substantial growth with an 11 percent increase observed in Phase I programs alone. Despite some challenges faced by start-up financing in early-stage funding rounds during this quarter—evidenced by dips in both number and total value—the overarching narrative remains one of dynamic advancement and unwavering potential within the field of cell and gene therapy.

How many cell and gene therapies are in development?

During 2023 Q4, the development pipeline continued to expand. Total CGTs in development grew from 3,951 at the end of Q4 to 4,002. This can be broken down into the following:

• 2,093 gene therapies
• 885 non-genetically modified cell therapies
• 1,024 RNA therapies

World Courier’s role in advancing therapy medicinal products

To support the growth of the advanced therapy medicinal product (ATMP) industry and enable more patients than ever before to be treated with these life-changing therapies, World Courier utilizes its global network of liquid nitrogen (LN₂) charging centers and stations.

This network is built on World Courier’s existing infrastructure, leveraging our company-owned LN₂ charging centers (office or depot locations) or vetted vendors’ charging stations. This means that ATMP developers can immediately benefit from an established and qualified operation with access to cryogenic shipping solutions close to patient and manufacturing locations.

Our network capabilities enable access to a range of cryogenic shippers with shorter lead times and reduced positioning costs

One of the challenges within the ATMP industry is the delivery of critical shipments, such as aphaeresis donations and final therapies. The short shelf life of fresh shipments and the resulting logistics complexity may encourage therapy developers to qualify cryopreserved transport. To enable this solution, dry shippers need to be charged with LN₂ by fully trained staff for both the LN₂ discharge and the processes for filling the dewar(s) This is a specialized process, in which dry shippers must be:

• Inspected and cleaned prior to use
• Fully charged, with supporting documentation
• Positioned when and where required in readiness for these highly critical therapies

Only after those stringent steps have been taken can the critical shipment take place. These preparations take time, especially when a charging center or station may be located in another country from the clinic or manufacturing site.

Supporting the growing pipeline

Data shows there are over 1,350 therapy developers globally. To support this growth, World Courier has a fleet of cryogenic dry shippers, as well as the ability to charge and ship customers’ units throughout its global network of strategically located owned charging stations and vetted vendors’ charging stations to offer near-total global coverage.

The strategic intention is to leverage a network of owned and qualified vendor charging stations as well as unit suppliers to develop unmatched worldwide coverage as globalization of cryogenic services continues. By utilizing its global network, World Courier helps advanced therapy companies build robust logistics platforms that support the clinical development and commercialization of therapies to meet patient treatment needs.

Source: Gene, Cell, + RNA Therapy Landscape Report, Q4 2023 Quarterly Data Report, American Society of Gene & Cell Therapy