Advanced therapies, including cell and gene therapies, refer to novel pharmaceutical products which leverage cellular and genetic-based approaches for the potential treatment of a vast range of diseases. Advanced therapies have huge potential for the future of medical research and enticingly offer solutions particularly in areas with unmet medical needs.
Being an innovative paradigm in healthcare, advanced therapy development aim to give the answer to patients with previously untreatable illnesses. Despite the promise for lifelong management and even cure of disease, Advanced Therapy Medicinal Products (ATMPs) face many challenges in terms of design and manufacturing, and, to date, they have met with very limited commercial success. Although their medical value is paramount, ATMPs are expensive to administer and produce as they require a high level of proficiency.
From a Regulatory perspective, ATMPs present a number of challenges because of the difficulty to integrate their biological diversity into preceding regulations. The risks associated with ATMPs are numerous considering the variety of products and may have an impact on the regulatory expectations for them. The ATMP regulation was designed to ensure patient safety and quality throughout development as well as to facilitate their manufacturing.
Advancing the advanced therapies by supporting their development with an improved regulatory framework will potentially provide new medical solutions for patients in the future.
Building an Advanced Therapy Medicinal Product industry may have a significant social as well as economic impact, as proven by the growing number of clinical trials in cell and gene therapy and increased investment in the field. Clinical trials are currently working on treatments for a wide variety of conditions that may eliminate the continuous need for medication by harnessing the power of the patient’s own cells, genes and tissue.
TCTC has a rapidly growing portfolio of conducting clinical trials in ATMPs following the successful execution of clinical trials for the first Gene therapy to be approved in Europe. This approval trailblazes the way for other gene therapies in development and strengthens TCTC’s reputation as a leader in the field of Advanced Therapy Clinical Trials.