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Cell and Gene Therapy Overview

Overview of Cell and Gene Therapy 

The Development of Cell and Gene Therapy

Cell and Gene Therapy (CGT) are overlapping fields of biomedical research and treatments for severe diseases, such as cancer and hereditary genetic disorders (1). Even a few years ago, the concept of altering a gene and editing the genome to cure or treat disease was considered fictional. Today with the advent of the genomic revolution and innovative technologies like CRISPR, it has become reality. As their names indicate, CGTs are designed to repair or replace an abnormal gene or cell within the body. Characteristically, Cell Therapy is introducing new cells into a patient’s body to replace, repair, or grow damaged tissue. Whereby Gene Therapy uses genetic material as a drug, introducing genes into cells— either in vivo or ex vivo. It has the effect of replacing or correcting the abnormal Gene function, causing conditions triggered by hereditary mutation or diseases (2)

Gene Therapy was first conceptualised in 1972. After several commercially unsuccessful attempts, the first FDA-approved Chimeric Antigen Receptor T-cell therapy, Kymriah®, is a successful Gene Therapy medicine for treating relapsed or refractory acute lymphoblastic leukaemia (ALL) (3). Currently, CGTs are being developed primarily for rare disorders like Muscular Dystrophy that have no other adequate treatment options (4). Over the last decade, there are now eleven EMA, and twenty-two FDA approved advanced therapy medicinal products (ATMPs) – that is, CGTs are established in the market (5). Clinical trials and regulatory approvals for CGTs are expected to increase rapidly over the next few years. The FDA estimates that by 2025, it will approve 10–20 CGTs per year.

Consequently, 2BScientific Ltd offers cutting-edge products and bespoke services to support CGT research evolving in the global marketplace. Contact us today to learn more about what we can do for you.   

To get a quote for the products or to place an order, please email Sales@2BScientific.com.

To get a quote for the products or to place an order, please email Sales@2BScientific.com.