Rohan A T | Friday, 23 September 2022
Cell and gene therapies are going to be catalysts of paradigm shifts in the global healthcare arena. Both cell and gene therapies are overlapping fields having similar therapeutic objectives. And it targets DNA or RNA outside the body and both these therapies aims at modifying the genetic materials in order to treat diseases.
The fact that the global cell and gene therapy market is expected to reach USD 25,002 million by 2027 which is nearly more than five times its current market value highlights the unprecedented growth and soaring popularity of cell and gene therapies. There are multiple facets that are accelerating the growth of the market including the increasing cases of cancer and other chronic diseases, more clinical trials, increasing flow of funding into the market, and favorable regulatory environment. Still, there are challenges such as lack of reliable vector production, complex manufacturing and distribution systems, and the hassles relating the commercialization of therapeutics that can hinder the growth of the cell and gene therapy market.
That said, there are plethora of innovations happening in the cell and gene therapy segment which can eclipse these challenges to a certain extent and give hope to millions of patients and healthcare professionals worldwide. Let’s analyze some of these innovations and how it can impact patient treatments.
Restoration of Sight by Fixing Faulty Genes More than 100 genes can be directly related to vision disorders in individuals and recently, new game changing gene therapies have emerged that can help treat this issue. This is done by adding new and healthy copies in place of faulty genes. And this approach is called CRISPR-Cas-9 gene editing which has the potential to treat genetic forms of vision loss that are not suited for other medical conditions. As of now, a clinical trial is happening with this approach on severe form of childhood blindness which at present doesn’t have any treatment. Apart from this, there are also similar gene editing therapies going on for correcting genes within the blood cells.
Using RNA for the Treatment of Brain Cancer Up till recently, RNA was predominantly known only for its helper functions but scientists now have started to identify the bigger role it plays in human biology. By tapping into this unrealized potential of RNA, they are aiming to develop new therapies for a myriad of diseases including glioblastoma which is a serious form of brain cancer. And owing to its highly adaptable nature, this cancer is challenging to treat effectively. A team has recently identified a trio of microRNAs. These are a class of RNA molecules and the important role it plays in healthy neurons but this is lost as the cancer develops.
To tackle this, microRNAs can be stitched together into a single unit which can be delivered into the brain using a virus. And the current studies show that this makes tumors more vulnerable to existing treatments. Another group is studying microRNA called miR-10b and blocking its activity is causing the tumor cells to die.
Gene Edition Solution and Xenotransplantation The number of patients who is waiting for an organ transplant is rising but the inadequate supply of donor organs is increasing the waiting times and making health conditions more complex for these patients. One of the most effective solutions for this issue is xenotransplantation. This is the process of harvesting organs from animals and using that in human patients. And the recent advancements made in gene editing technology are making it possible to remove, insert or even replace genes accurately. This method can be leveraged to sidestep the human immune system which is capable of identifying foreign tissues and creating a reaction. That said, more clinical trials are needed before xenotransplantation become a common treatment methodology when it comes to organ transplants.
Utilizing Gene Therapy for Brain Disorders Gene therapy holds immense potential when it comes to finding an effective treatment for diseases that are currently untreatable. This includes diseases pertaining to brain as well as spinal cord. That said, at present, the commonly used vehicle for gene therapy, AAV which is a virus cannot penetrate the blood-brain barrier. And to solve this issue, scientists are developing new versions of this virus that has the capability to penetrate this barrier. By leveraging different molecular strategies, they have been able to modify protein shells that can surround the virus. The good news is that, these modified viral vectors are currently under development and will be ready for clinical testing in the near foreseeable future. The success of these new viruses during clinical trials can be a game changer as it will help in treating a myriad of neurological conditions including neurodegenerative diseases.
Developing Cancer Killing Cells Even though, scientists, researchers and other healthcare professionals have been able to make impressive leaps in treating cancers, solid tumors remain one of the hardest to treat. This is in part owing to the hostile environments in which they are seen. And to create an effective treatment for solid tumors, scientists are devising innovative cell therapies. One of the latest approaches in this field involves the creation of CAR-T cells that are like computers and it relies on identifying which cells is cancer. And by building myriad of logic gates and merging them, scientists hope to find ways to target new tumor types.
Another innovation happening in this segment is using cancer cells in cancer-killing cell therapy. With the help of gene editing and other molecular engineering technologies, researchers are aiming to turn tumor cells into potent cancer killers. Innovations happening in cell and gene therapies can reshape the global healthcare domain. Not only can it help find new ways for treating diseases that at present doesn’t have effective treatment protocols, but as it grows and becomes more popular, the cost of treatment for various diseases can also become more affordable making those treatments more accessible. And newer findings and developments in this space can go a long way in improving patient care.
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