Emerging trends and technologies in gene and cell therapy

Описание: Gene and cell therapy (GCT) is one of the fastest growing segments in the biopharmaceutical industry. Starting first with the approval and use for autologous CD54+ antigen presenting cells by the Food and Drug Administration (FDA), followed by several other allogeneic hematopoietic cell therapies for a limited group of patients with disorders affecting the hematopoietic system to the first approval for Chimeric Antigen Receptor T-cell (CAR-T) therapy, Kymriah, in 2017, there has been an increase interest and efforts in developing such CAR-T for cancers. CAR-T therapies involve the removal of T cells from individual patients, engineer them to be able to recognize and kill cancer cells before re-administering them to the same patient. It is widely considered as revolutionary treatment for blood cancers, with emerging indications for it as a potential for solid cancers as well. Just in the span of four years, there were 5 CAR-T therapies approved by the FDA. For gene therapies, it is gaining momentum too. As of 2017, an estimated 2,600 gene therapy clinical trials were ongoing or completed, with currently two gene therapies approved by the FDA.

Despite the increase rates of approvals, there are still many limitations faced in GCT, such as the accurate delivery of the genes to appropriate cells and the specificity of the CAR towards the antigen. In addition, many of these treatment require demanding GMP production. With the advancement of scientific technologies, some of these are addressed and been explored.

In this presentation, you will learn:
1. The current trends in providing more accurate delivery of genes and cell engineering
2. New technological solutions to reduce toxicity
3. How the proof of concept, clinical development and GMP production could be streamlined