A Brief Discussion of How Cell and Gene Therapies are Being Improved for Future Generations


While the last few years have seen major advances in all areas of the pharmaceutical industry, some of the newest and brightest talents are now focusing on cell and gene therapies.

A recent article in CheckOrphan reports that the FDA expects to receive about two hundred investigational new drug applications (IND). That translates to over two hundred requests annually from pharmaceutical companies for approval to begin clinical trials on their new therapies.

The new systems put in place at the FDA have been successful in expediting their approval system to the extent that it anticipates approving ten to twenty new cell and gene therapy products annually.

About Cell and Gene Therapy

A type of gene replacement therapy that treats a rare form of vision loss was approved by the FDA in 2017. Gene replacement therapy has been in “study mode” for the past thirty years. This was its first approval by the FDA allowing it to be used in humans outside of clinical trials.

At this point, while it uses genes for the treatment or prevention of some diseases, gene therapy is still mostly in the experimental stage. At some time in the future though, it is expected that the technique of inserting genes into a patient’s cells will take the place of drugs and surgery. The healthy copy of the gene will replace the mutation that causes the disease.

Cell therapy of the future will treat diseases that cannot be addressed by current pharmaceuticals. It is a procedure that introduces healthy cells that grow and replace damaged tissues in the body. Currently, there are a variety of cells in many stages of clinical development, especially in the treatment of blood cancers.

Cell therapy includes organ and bone marrow transplants, platelet transfusions, and blood transfusions. It has the potential to treat neurological conditions as well as autoimmune diseases. The list is long and includes multiple sclerosis, amyotrophic lateral sclerosis, muscular dystrophy, Parkinson’s disease, lupus, and Crohn’s disease.

 And Now The Other Side of the Coin

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study for safety and efficacy. Gene therapy is currently being tested only for diseases that have no other cures.

About Viral Vectors

Viruses use specialized molecular mechanisms to transport their genomes efficiently into the cells they infect.

Now scientists are able to modify viruses and render them harmless. Since neurological and autoimmune diseases are the result of mutations in the DNA sequence for certain genes, these modified viruses are used in clinical trials for delivery of genes to cells in the patient’s body.

Nucleic acids (RNA and DNA) are small biomolecules that are vital to every form of life. However, unmodified nucleic acids will create an immune response. They become degraded by enzymes in the bloodstream and gut. This requires RNA medicine to be sent to target cells by way of a vector (transport vessel).

Viral vectors, on the other hand, have the following side effects:

  • Can cause liver damage
  • Create adverse immune reactions
  • Lead to accidental mutations causing cancer

These problems are being addressed by Arcturus Therapeutics through its LUNAR system that delivers RNA molecules safely into targeted cells in the patient’s body. According to the company, its system is focusing on the problems affecting the viral delivery systems.

The next Annual Cell and Gene Therapy Innovation Summit will be held in February 2020. The Summit is committed to increasing innovation for the exciting area of Cell and Gene Therapy.​

Have you or anyone you know been advised to have any type of cell therapy?


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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