This blog gives a brief overview of what gene therapy is and describes the difference between the two types. It also discusses commercially successful gene therapies.
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.
It has the potential to transform the treatment for diseases, significantly changing how doctors manage and treat patients.
Two types of gene therapy
The first corrects a specific disease-causing genetic mutation. These are targeted toward 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 Kite Pharma’s Yescarta are examples of CAR-T therapies, which 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 disease (1).
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 direct injection into the patient (in vivo), or by modification of selected cells that are collected from the patient, modified outside the body (ex vivo), and reintroduced 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 clinically 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 of 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 (2).
Furthermore, there has been significant investment with > $50B being invested in the area in the past 3 years (3).
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.
References
- Key Considerations for Gene Therapy Commercialization, Cell Culture Dish. Updated September 12, 2018.
- 2019 Cell & Gene Therapies State of the Industry Briefing, Alliance for Regenerative Medicine. Updated January 7, 2019.
- Annual Regenerative Medicine Data Report, Alliance for Regenerative Medicine, 2016-2018. Updated 2018.
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Kymriah is a trademark of Novartis AG. Yescarta is a trademark of Kite Pharma, Inc. Any other third-party trademarks are property of their respective owners.