AMR Future Breif | How Has Gene Therapy Transformed the Medical Treatment Landscape?

Advancements in medical science have improved the outcomes at each level including diagnosis, treatment, and rehabilitation. The overall efficiency and quality of healthcare solutions have become better over time due to these advancements. A major focus of most of the advancements in the medical sector is on the development of drugs or medications that help in treating rare diseases and alleviate the pain associated with them. Gene therapy is one such example.?

Gene therapy is an advanced medical technology that relies on manipulating genetic expression to produce a therapeutic effect. Thus, it involves treating the disease by addressing the problem at the genetic level. As opposed to administering drugs or medications, gene therapy, thus, offers a novel approach to treating the disease by altering the genetic code. As part of gene therapy, doctors can turn on a gene to develop resistance against the disease. In some cases, genes are turned off to stop their improper functioning; while in other cases, a part of genetic material is added or removed to make the gene free from any disorders. Thus, gene addition, gene silencing, and gene editing come under the umbrella term of gene therapy.?

Gene therapy: A ray of hope for rare diseases?

Despite advances in drug development, certain rare genetic diseases and lifestyle diseases like cancer aren’t fully curable yet. Medical research has shown that normal therapies have proven to be insufficient in fighting the disease. In such cases, gene therapy, using gene editing techniques, can offer a perfect solution to tackle these diseases.??

A major advantage of gene editing therapy is that it is offered to the patient as a one-time dose as opposed to other conventional techniques which require multiple dosages. Also, since the changes made through gene editing are at the genetic level, they offer a lifetime cure for the disease. Rare genetic diseases are feared because the defects in genes can be passed on to the next generations. By addressing the defects in these genes, gene editing completely nullifies the chances of these defective genes being carried onto the next generations.?

Medical researchers across the globe, through their independent studies, have been able to prove the efficacy of gene editing therapies in treating medical conditions such as Severe Combined Immune Deficiency (SCID), hemophilia, cystic fibrosis, Adenosine Deaminase (ADA) deficiency, sickle cell disease, etc.?

Which are the gene editing therapies that have received approval from the FDA??

The United States Food and Drug Administration (US-FDA) is the primary regulatory authority in the US that provides approvals to new drugs and medication after conducting rigorous medical trials. Due to its highest quality standards, the FDA has become one of the most reputed and reliable drug approval bodies in the world. The safety and efficacy of a drug, medication, or therapy is hence decided upon whether it has received FDA approval.?

In 2017, Luxturna?, a genetic editing therapy for treating Leber congenital amaurosis, received FDA approval. Leber congenital amaurosis is a rare inherited retinal (eye) disease that causes progressive blindness starting from the first few months of life. With this approval, Luxturna? became the first gene therapy to be ever approved by the FDA.?

Similarly in 2019, the FDA approved Zolgensma? (Onasemnogene abeparvovec), a gene editing therapy used to treat spinal muscular atrophy in children. Spinal muscular atrophy, a rare neuromuscular disease, results in progressive muscle wasting in children owing to loss of motor neurons. The drug Onasemnogene abeparvovec has been developed by Novartis Gene Therapies, a leading biotechnology company, and has received approvals in multiple countries following the FDA approval.?

How are gene editing techniques helping in finding a full-proof cure for cancer??

The rising incidence of cancer and the increasing popularity of gene editing therapies have led to medical experts engaging in research to formulate a comprehensive cancer treatment regime. In the past few years, certain gene editing therapies have been developed such as CAR T-cell therapy, CAR NK-cell therapy, oncolytic viruses, and therapeutic cancer vaccines. Among these, CAR T-cell therapy is currently used to treat different kinds of blood cancers including B-cell acute lymphoblastic leukemia, Follicular lymphoma, Multiple myeloma, etc. In this therapy, the CAR receptor binds itself to the T-cells and directs them to the tumor cells in the body. On the other hand, oncolytic viruses therapy involves the introduction of engineered viruses into the body to directly kill the cancer cells. This gene editing therapy is widely used for treating advanced melanoma.?

To sum it up, gene editing techniques are now being increasingly used to treat rare genetic diseases due to their advantages over traditional treatment regimes. The FDA has started approving these therapies which has resulted in an increased trust of patients concerning these techniques. Medical researchers have now even started to develop gene editing techniques that completely cure different types of cancer.?

For more tips and suggestions on how to capitalize on the opportunities offered by the gene therapy industry, feel free to contact us.?

Short Description: Gene therapy refers to the addition, removal, or alteration of specific genes to offer a therapeutic treatment for a disease. Gene editing techniques have become a popular treatment mechanism for rare genetic diseases owing to the advantages offered by these advanced medical therapies. Furthermore, researchers are also trying to develop gene therapies for curing different types of cancer.?

? **?????????????? ????????????: Akhilesh Prabhugaonkar?

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