What Is Gene Therapy? How Does It Save and Improve the Quality of Life?

What Is Gene Therapy?

Gene therapy is a new and exciting technique, defined as the use of genetic material to cure or alleviate disease. It is considered revolutionary, yet still in its infancy, with many new therapies currently undergoing clinical trials.

Gene therapy has the potential to transform the treatment for diseases, significantly changing how doctors manage and treat patients.

Two Types of Gene Therapy

There are two main types of gene therapy.

The first corrects a specific disease causing genetic mutation. These are targeted towards inherited genetic disorders such as hemophilia or Duchenne muscular dystrophy. The second gives new functions to cells allowing them to fight disease.

A good example of these therapies are chimeric antigen receptor T cell (CAR-T) therapies. Both Novartis’ Kymriah and Gilead’s Yescarta are examples of CAR-T therapies that have demonstrated exceptional cancer remission rates where other forms of treatment have failed.

Cancer is the by far the largest category of disease with 65% of gene therapy clinical trials being investigated, followed by 11.1% for inherited monogenetic disease, 7% for infectious disease, and 6.9% for cardiovascular disease1.

How Does Genetic Material Get Delivered to Host Cell(s)?

Genetic material gets delivered to a host cell via a delivery system known as a vector. Vectors deliver genetic material via one of the two methods. By directly injecting genetic material into the patient (in vivo), and where selected cells collected from the patient, undergo modification outside (ex vivo) before introducing them back into the patient.

The most commonly used type of vector is a virus. While there are other methods of delivering genetic material into a cell, viruses have now been developed that demonstrate a good balance between efficacy and safety.

Commercially Successful Gene Therapies

Developing a commercially successful gene therapy is challenging. It requires balancing several different considerations. Having a clinical effective therapy is essential, but this alone is not sufficient to ensure product success. In addition to this, reimbursement, quality and regulatory considerations, and manufacturing also must be considered.

To date, a total 11 gene therapies have received marketing approval. However, behind this there is a strong clinical pipeline with >1000 clinical trials underway, and 92 drugs in Phase 3.

Furthermore, there has been significant investment with >$50B being invested in the area in the past 3 years.

This investment, coupled with the accelerating understanding of disease at the genetic level, holds immense potential. Academic, commercial manufacturers, and industry suppliers are actively seeking new approaches that deliver these therapies as quick as possible to a waiting population.

Author Details:

Clive Glover

Director, Cell & Gene Therapy

Pall Corporation


About Pall Corporation:

Pall Corporation is a filtration, separation and purification leader providing solutions to meet the critical fluid management needs of customers across the broad spectrum of life sciences and industry. Pall works with customers to advance health, safety and environmentally responsible technologies. The Company’s engineered products enable process and product innovation and minimize emissions and waste.