In the history of medicine, there are some major events that have changed the paradigm of disease treatment, such as the invention of surgery and the discovery of antibiotics. And the next may just be gene therapy. With the improvement of gene vectors, the rise of chimeric antigen receptor T-cell (CAR-T) immunotherapy and the breakthrough of genome editing technology, gene therapy has rapidly absorbed almost all the advances of biotechnology in the past half century. It has contributed a lot to the treatment of tumors and genetic diseases.
· What is gene therapy?
“Gene therapy is a technique that modifies a person’s genes to treat or cure disease,” FDA’s definition of gene therapy is simple, but sometimes the simpler concept contains more. So far, gene therapy mainly works by 3 mechanisms: replace or inactivate a disease-causing gene, and introduce a new or modified gene.
· Explosive growth of global gene therapy market
With the development of gene therapy, the safety and effectiveness of vectors used in gene therapy products have gradually improved, and a number of gene therapy products have been approved for the market.
Driven by technology, capital and policies, the global gene therapy industry is rapidly heating up, and a large number of gene therapy drug research and development have entered the clinical stage, and have shown explosive growth since 2015. Especially after 2017, with the launch of the adeno-associated virus drug Luxturna and 2 CAR-T drugs, Kymriah and Yescarta, the gene therapy industry has become one of the most promising global frontier medical fields.
According to American Society of Gene & Cell Therapy (ASGCT) data, as of the end of 2020, there have been more than 1,300 clinical trials under research of gene therapy worldwide; about 25% of them are in clinical phase II-III, and 6% are in clinical phase III.
· Future of Gene Therapy
“We are definitely still in the early stages of gene therapy,” introduced by a scientist from Creative Biolabs, a gene therapy development service provider, “there are still decades to go, but as far as the current situation is concerned, gene therapy will have a huge impact on the future of medicine.”
The potential of gene therapy is huge, but compared with its promise, the results so far are still quite limited. However, the benefits of gene therapy are considered close-at-hand, especially as some major innovations have appeared. For example, the Human Genome Project (HGP) has opened new doors for exploring gene therapy. And with the advent of new technologies such as biochips, there is no doubt that gene therapy will play an increasingly important and prominent role in the coming decades.
2017 is the beginning that gene therapy officially moved towards the clinic, and from then on, gene therapy ushers in more commercial development and clinical trials. This treatment method may be the most complicated drug developed by humans in modern times, and it is hopeful to cure some diseases that have no effective treatment so far.
https://www.creative-biolabs.com/gene-therapy/approaches-to-genome-editing.htm