Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine.
For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases.
Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space. There is a lot of excitement that we could be at the start of a new era for the biopharmaceutical industry, with the hope of delivering revolutionary healthcare for previously unaddressed diseases finally becoming a reality.
Although a high number of cell and gene therapies are in early-stage development, a new wave of approvals could be on its way. Gene therapies, for example, are showing a lot of promise for treating rare diseases such as spinal muscular atrophy, macular degeneration, and cystic fibrosis. While just two gene therapy products have been approved by the FDA, a number of companies are nearing the market with therapies targeting rare diseases, including 14 candidates in Phase III plus three in the pre-registration phase, according to GlobalData.
A new frontier in cancer research
Cell and gene therapies are currently at the forefront of immunotherapy cancer research, and a few approved therapies are already making a difference to disease outcomes for patients. Often referred to as a type of gene-modified cell therapy, CAR-T cell therapies involve genetically modifying a patient’s own T cells to produce a protein that enables them to identify and kill cancer cells.
A total of seven CAR-T therapies have been approved around the world (five by the FDA), all for haematological diseases such as myeloma and lymphoma. Another seven CAR-T therapies are in the pre-registration stage, meanwhile there are currently 266 CAR-T cell therapies in Phase II-III development, according to GlobalData. Using a similar treatment to target solid tumours could be the industry’s next major milestone.
Securing the supply chain
The industry is moving at an unprecedented speed. But there are many challenges it will face along the way, such as how to manufacture these drugs in a reproducible, scalable, cost-effective, and safe way for patients. New challenges have also been introduced in the supply chain, especially for cell therapies where logistics timelines must be shorter to ensure cell viability.
Cold chain logistics, meanwhile, have never been so important. “Premium cold chain logistics are vital for cell and gene-based therapies, as there is zero room for error with cell and gene shipments,” explains Richard Rossi, Global Cold Chain & Strategic Projects Director at CRYOPDP. “The cold chain needs to be secure from end to end, both in the chain of possession and the chain of identity of materials being cryogenically transferred to or from manufacturing centres.”
CRYOPDP provides temperature-controlled supply chain & logistics services to the biopharmaceutical industry, with a fast-growing customer base in the cell and gene therapy communities. The company is often a first choice for these innovators due to its specialised services for cryogenic shipments, including a variety of dry shippers and accessories. GPS tracking and environmental monitoring in terms of temperature, shock, vibration and other available parameters are vital components of this service, giving pharma and biopharma companies the peace of mind that their high-value cell and gene goods arrive at their intended destination – whether that’s a laboratory, clinical trial site, manufacturing centre, pharmacy or patient’s home – in the correct condition.
“CRYOPDP’s global teams are acutely aware of the seismic shift in focusing on cell and gene therapy drug development, and we are dedicated to supporting this emerging wave of new cell and gene therapies,” says Rossi.
“By understanding the requirements of cell and gene, CRYOPDP works with partners to present enhanced security options and focus on regulatory requirements. We pride ourselves on developing global transport solutions that mitigate risks and deliver real-time supply chain tracking and visibility.
“When choosing CRYOPDP, our customers are choosing the best-in-class partner who’s able to deliver tailor-made temperature-controlled supply chain & logistics solutions and support the clinical development of life-saving therapies that match the patient’s needs.”
It is certainly a pivotal moment for the cell and gene industry, with the next decade of R&D and clinical trials expected to transform the future of medicine. But getting these drugs to patients requires risky transportation, and safety and success hinges on reliable, temperature-controlled logistics. With its cold chain technologies, customised solutions, and dedicated teams, CRYOPDP is here to help the industry mitigate these risks and properly deliver the promise of cell and gene therapies to patients.