Clinical Trial Innovations in Gene Therapy

Carsten Thiel
May 14 · 5 min read

Once considered a futuristic venture, cell and gene therapies are moving rapidly into the areas of the clinical trial industry. With advancements in gene therapy technology, the industry has reached a record 2,900 registered clinical trials in gene therapy mostly in the United States, Europe and China. Other countries are beginning to increase their research as the impact of this clinical research is confirmed on both the human and industrial level. These trials testify to the swiftly emerging research in the biopharmaceutical industry.

Oncology remains the largest area of study in gene and cell therapy with monogenic diseases, diseases with just one missing or nonworking gene, not far behind. Gene therapy includes a number of innovative treatments that are offering exciting potential. It encompasses a wide variety of treatment options using genetic materials to modify cells. With advancements in DNA technology, research and commercialization, gene therapy has become much more applicable. It takes time for these new therapies to be trialed and possibly approved, but because of these advancements, the FDA will be adding additional reviewers for gene and cell therapy as the new drug applications are rapidly increasing and new FDA approvals are expected each year. It is estimated that there will be 40 approved gene therapies by 2022.

Genetic engineering has led to the growth and expansion of the pharmaceutical industry. It is the process of altering the DNA in an organism’s genome. This can mean changing, deleting or introducing an additional copy of a gene. It is used by scientists to enhance or modify the characteristics of an organism. For example, DNA technology has provided the ability to make human insulin on a commercial level from yeast and bacteria very similar to our own after years of gathering discarded pancreases from cows and pigs in slaughterhouses to remove the insulin. By producing insulin from the human insulin gene, it can be developed cheaper and cleaner and be mass produced on an industrial level.

Developing designer proteins for gene editing is another well-known means for creating engineered treatments. Usually a person’s own genes produce specific proteins. The goal with these treatments is to give the body a new copy of the missing or compromised gene to prevent or treat genetic diseases. These proteins can be used to remove mutations in DNA and replace them with healthy DNA. Scientists have discovered that viruses, because of their ability to enter the body, can actually be utilized to deliver the genetic material to the cell. Some of these viruses can be programmed and used to kill cancer cells leaving the healthy cells intact. Gene replacement therapy has been studied in humans since 1989 and it has taken nearly 30 years for the first gene replacement therapy to be approved by the FDA for clinical studies. The first gene therapy approved by the FDA was injected into melanoma to kill the cancer cell while not harming the surrounding healthy cells and to prompt the immune system to kill other melanoma cells located in other places on the body. Cell based vaccines can modify cancer cells causing the immune system to become responsive against the cancer cells. Viral cancer vaccines can be used by inserting a specific antigen in the virus and delivered, with the desired result being using the immune system to attack the gene that was coded by the virus.

Gene editing is a gene based therapy that inserts, changes or replaces specific parts of a person’s DNA. The most common form of gene editing is called clustered regularly interspaced short palindromic repeats, or CRISP-R. CRISP-R is used to change DNA sequences to precisely-targeted areas. It is often a controversial tool mainly because its reliability, safety and ethical use are a major concern. Some of those concerns are that is can be used to make genetic changes that can be passed down for generations if used in cells like eggs and sperm. A new trial by University of Pennsylvania scientists is a landmark moment in the United States. It can allow scientists to remove cancer-causing abnormalities from DNA and replacing it with an immune boosting DNA sequence. Their goal is to delete one gene using CRISP-R and add another, giving it the ability to fight the cancer. This isn’t the first time that CRISP-R has been used on humans. The international genetics community was stunned to learn in 2018 He Jiankui from China used CRISP-R to edit human embryos. His goal was to make the embryos of the twin girls resistant to HIV. Most clinical trials using CRISP-R have been conducted in China. The clinical trial in Pennsylvania is intended to take a very different approach by conducting it carefully and ethically to pave the way for more trials in the United States.

Cell and gene therapies are becoming an integral part of the clinical trial industry. Research in the biopharmaceutical field in gene therapy is demonstrating the impact that it is having on the human level and the industry. Genetic engineering has led to growth in the pharmaceutical industry, products such as insulin can be made on an industrial level, making it cheaper and cleaner for patients. Gene editing can be used to remove mutations in DNA and replace them with healthy DNA using viruses to deliver the genetic material to the cell. CRISP-R is being utilized for the first time as part of a clinical trial in the United States after years of concern for its safety and reliability. It carries enormous possibilities to further advance human health when used ethically and safely. These gene therapies and others are offering innovative treatments with exciting potential demonstrating the rapidly emerging research that the biopharmaceutical field is providing.

Follow Carsten on Twitter

Carsten Thiel

Written by

Inspired by improving health through innovation, believes in servant leadership, dedicates time to child education