Current Challenges in the Globalization of Gene Therapy Products

Today, the ability of gene therapy to cure human diseases is no longer a dream!

.................In contrary, many diseases and pathophysiological conditions that could be cured using gene therapy are still away from the reach of current gene therapy products and techniques. The success of gene therapy process relies on the successful modification of targeted genes to get therapeutic benefits, and the process of tweaking this step is not only tricky but also one of the significant hurdles that the field is facing. The success of gene replacement/repair process entirely relies on robust pre-clinical research data that eventually ends up in running successful human trials and product launch. So the entire journey from setting up of pre-clinical models to clinical trials to FDA approval to product launch is just like a roller coaster and involves a lot of risks. Possible odds against vector based unpredictable and unwanted host interactions, immune responses, and toxicity issues must be evaluated and addressed carefully for the overall success of gene therapy products. Well, here I am referring to both viral and non-viral vectors. I reiterate that we should not hesitate to undertake alternative routes to prevent unwanted host immune response against the gene-edited cells. The use of engineered vectors/nanoparticles to escape the host immune response may be a valuable alternative strategy (out of many of course) against body’s immune response directed towards the gene replacement/repair.

With the increase in the development of gene therapy techniques and its recent success by Spark Therapeutics, the investment in gene therapy has rapidly been increasing. However, this industry still faces a much sit back from the pharmaceutical manufacturers, as the disease targets of gene therapy products are very complex and work in a network (a network of genes). Here, the famous and classic “one gene-one protein” theory doesn’t work. Therefore, companies and manufacturers take bold and careful steps while manufacturing and commercializing such expensive products on the market. Moreover, the failure of any gene therapy product also costs a leg and an arm to the manufactures, a significant limiting factor in the investment of gene therapy products.

To overcome this issue, in the recent years, focus on cryopreservation of gene-modified cells for their preservation and efficient transport has been increased. This technique could really be helpful to better address economic and global application issues of gene therapy products.