Gene Therapy: Transforming Medicine Through The Years

The need to treat rare genetic diseases, enhance quality of life, and unlock the next wave of treatment innovations is projected to promote the growth of the gene therapy market.  Various universities and institutes are home to a broad-ranging portfolio of products in the pipeline. In addition, several researchers are trying to make gene therapy available at clinics. However, before discussing the therapy advancements and driving factors of the market, let us understand gene therapy.

Gene therapy is an advanced medical technology that can alter genes inside a body’s cells to treat or prevent genetic disorders. It functions by manipulating gene expression by replacing or inactivating a muted gene and adding a new gene to produce a therapeutic effect. It holds a promise of treating several diseases, especially genetically passed diseases, including cancer and other viral infections. Gene therapies can be performed using two major methods- in vivo and ex vivo. While in vivo gene therapy is delivered using an injection directly into the receiver’s body, ex vivo removes cells and delivers genes to the same cells outside the receiver’s body.

Gene therapies are regulated by CBER, a part of the FDA, as biologics, and the products are categorized as advanced therapies by U.S. regulatory authorities. The United States, Japan, and Europe have also built detailed regulatory frameworks for gene therapy products, with few bends that project regional differences across developing and commercialized gene therapies. In Latin America, the regulation differs by country. While few countries, such as Brazil and Mexico have established regulatory policies, other countries, such as Peru and Bolivia, are still developing their regulatory systems. Therefore, it is essential to analyze and understand the imposed regulations on gene therapy products.

Types of Gene Therapy

• Gene Editing

It is a set of technologies that allows precise alterations of DNA. It can fix gene mutations for the genes to function properly, and help the gene to fight diseases. It can further turn off a gene that fails to function correctly and remove a disease-causing DNA piece. CRISPR-Cas9 is one of the well-known gene-editing technologies that enables researchers to target and edit specific DNA stretches with high accuracy.

• Gene Replacement/Addition

These are techniques for modifying an individual’s genome. Gene replacement involves substituting a defective gene with a healthy version, using homologous recombination for precision. It is mostly used for correcting gene mutations. Gene addition, on the other hand, introduces a new gene into the genome without removing any existing genes. Both these techniques are crucial in genetic research, therapeutic interventions, and biotechnological applications.

• Cell Therapy

Cell therapy is a medical treatment in which living cells are injected, grafted, or implemented into a patient’s body to achieve a therapeutic effect. Common types of cell therapy include stem cell therapy, which uses stem cells’ ability to differentiate cell types, and immunotherapy, which involves modifying immune cells to fight diseases such as cancer.

• RNA Therapy

RNA therapy/treatment focuses on RNA over DNA. It uses RNA molecules to treat disease by altering the way genes are expressed in cells. This involves giving cells the instructions to make beneficial proteins by turning off harmful genes. This therapy is being majorly explored for several diseases, including genetic disorders and cancers.

Factors of Growth

The growth of the gene therapy market can be attributed to expansion in advanced therapies and gene delivery technologies. Rising competition among prominent market players focused on commercializing their therapies also positively impacts growth. Biotechnology companies invest in mergers and acquisitions and expansions as key strategies to increase in-house expertise and strengthen product pipelines.

Traditional genome editing tools do not possess enough capacity to keep up with the speeding progress of genome modification technologies. The development of CRISPR/Cas9 nuclease allows more precise genome editing. As a result, in recent times, the gene-editing department has been witnessing a significant amount of research activities. Funding and investments in the gene therapy market have also increased in recent years. For instance, in January 2022, 64x Bio which is a U.S.-based gene therapy biotech company, raised over USD 50 million in funding for the advancement of its gene therapy manufacturing platform.

Challenges in the Gene Therapy Market

The involvement of alterations in genes in gene therapy has raised several ethical concerns. Gene therapy holds the capacity to safeguard a family’s future generations from particular genetic disorders. However, gene therapies can have an impact on the development of a fetus in various unexpected ways. In addition, regulatory agencies such as the FDA have established several documents demonstrating the preclinical and clinical activities as a key to regulating and reviewing gene therapy products. Stringent regulatory guidelines on gene therapy product approval have slowed down the development of the market. Moreover, drug companies have made these narrowly targeted treatments extremely expensive, which has resulted in lesser patient involvement. These are some of the drawbacks limiting the speedy growth of the gene therapy market.