Gene Therapy Market

Global Industry Analysis (2018 – 2021) – Growth Trends and Market Forecast (2022 – 2026)


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Gene Therapy in Demand with Rising Cancer Prevalence

Gene therapy, a technique that modifies a person’s gene(s) to treat, or cure a certain disease, is more specifically the genetic modification of cells to obtain a therapeutic effect. It can potentially help in restoring, reconstructing, or repairing the defective genetic material and lead to a possible treatment for a certain disease condition. The capacity of gene therapy to bring about desired gene improvement involves the correction of altered (mutated) genes, or site-specific modifications that have therapeutic treatment as the target. The approach is broad, with potential treatment of diseases caused by recessive gene disorders (cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anaemia), acquired genetic diseases such as cancers, Parkinson's, and certain viral chronic ailments such as acquired immune deficiency syndrome (AIDS). The demand for gene therapy is surging worldwide as the prevalence of cancers has been consistently on the rise. Increasing funds coming to the gene therapy research area further strengthens the scope of growth for the global gene therapy market.

Gene Therapy Mechanisms

Gene therapies can work by several mechanisms: Gene augmentation (introducing a new gene into the body to help fight a disease), gene inhibition (replacing a mutated gene that causes disease with a healthy copy of the gene), killing of specific cells (inactivating, or knocking out, a mutated gene that is functioning improperly).

There are two different types of gene therapy depending on which types of cells are treated-

  • Somatic gene therapy: transfer of a section of DNA to any cell of the body that doesn’t produce sperm or eggs. Effects of gene therapy will not be passed onto the patient’s children
  • Germline gene therapy: transfer of a section of DNA to cells that produce eggs or sperm. Effects of gene therapy will be passed onto the patient’s children and subsequent generations

One of the most often used techniques consists of recombinant DNA (rDNA) technology, in which the gene of interest or healthy gene is inserted into a vector, which can be plasmidial, nanostructured, or viral; the latter is the most often used due to its efficiency in invading cells and introducing its genetic material.

There is a variety of gene therapy products, including-

  • Plasmid DNA: Circular DNA molecules can be genetically engineered to carry therapeutic genes into human cells
  • Viral vectors: Viruses have a natural ability to deliver genetic material into cells, and therefore some gene therapy products are derived from viruses. Once viruses have been modified to remove their ability to cause infectious diseases, they viruses can be used as vectors (vehicles) to carry therapeutic genes to human cells
  • Bacterial vectors: Bacteria can be modified to prevent them from causing an infectious disease, and then used as vectors (vehicles) to carry therapeutic genes to human tissues
  • Human gene editing technology: The goals of gene editing are to disrupt harmful genes or repair mutated genes
  • Patient-derived cellular gene therapy products: Cells are removed from the patient, genetically modified (often using a viral vector), and then returned to the patient

Target cells such as the patient's liver, or lung cells are infected with the vector. The vector then unloads its genetic material containing the therapeutic human gene into the target cell. The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state.

Gene Therapy Costs Continue to Restrict Rapid Market Expansion

The viruses used in gene therapy are altered to enhance their safety. However, concerns continue to exist around the viruses being safe. Once the gene therapy administration begins, it is difficult to discontinue the treatment, and it is too early to know how long the effects of a gene therapy would last. Moreover, a negligible number of patients have been given gene therapy for any length of time to know whether it poses any safety risks in long term. Another stumbling block is the incredibly high price of gene therapy. Gene therapies are currently some of the most expensive treatments on the market, which also indicates the fact that most of them are patient-customised.

Kymriah indicated for paediatric leukemia, and advanced lymphoma costs US$ ~375,000-475,000 in the US, whereas the same treatment is priced at ~US$ 306,000 in Japan, and ~US$ 413,000 in Australia. In the US, Yescarta, a treatment for adults with large B-cell lymphoma, is priced at ~US$ 373,000. Luxturna, prescribed for a specific type of inherited retinal dystrophy, costs ~US$ 850,000, while Zolgensma, a lifesaving gene therapy for paediatric spinal muscular atrophy (SMA), is priced at a whopping ~US$ 1.5 Mn per patient.

Gene Therapy Becomes a Lucrative, and Promising Business

Gene therapy is arguably the most remarkable, and exciting area of biotechnology at this moment as it has produced treatment results for the diseases that were beyond any treatment around a decade ago. The recent success stories of genetic medicine have paved the path for broader therapies, laying a strong foundation for next-generation technologies. According to Global Genes, 7,000 distinct rare diseases exist today and nearly 80% of the rare diseases have an identified genetic origin. One in 10 Americans, 30 million in the US, and 400 million globally, are affected by a rare disease. Rare diseases typically impact more people than cancer, and AIDS combined.

Approximately 3-10% of all hospitalisations (regardless of the patient age) are related to a rare disease, whereas 65% of rare diseases are associated with reduced lifespan. The average time to get an accurate rare disease diagnosis is between six and eight years. Nearly 50% of the people affected by rare diseases are children, and 3-10 children with a rare disease would not even live to see their 5th birthday, as per the reported estimations. By unlocking the promise of gene therapy for millions worldwide, established, as well as new companies are coming out with pioneering breakthroughs expecting a change in patients’ lives. When the first gene therapy was approved in the US, there were 854 companies developing such therapies. According to Alliance for Regenerative Medicine, there were 1085 companies in that space by the end of 2020, and more than 400 gene therapy trials are still underway.

Gene Therapy is at Inflection Point – a Highly Regulated Area

As many investigational gene therapies continue to demonstrate their clinical and commercial potential, companies including small, and large biotech players are positioning themselves for the opportunities to acquire new gene therapy technologies. The notable deals of the last year include the acquisition of AskBio Pharmaceuticals by Bayer for $4 Bn, and of Prevail Therapeutics by Eli Lilly for $1.5 billion. Gene therapy is currently stringently regulated by the US Food and Drug Administration’s (FDA) Centre for Biologics Evaluation and Research (CBER), and the National Institute of Health (NIH). However, around 95% of rare diseases still do not have a single FDA-approved drug treatment. The European Union has EudraLex, and European Medicines Agency (EMA).

Major Gene Therapy Companies in Global Market

Asklepios BioPharmaceutical, CRISPR Therapeutics, Editas Medicine, Homology Medicines, Intellia Therapeutics, Passage Bio, Pfizer, Poseida Therapeutics, Prevail Therapeutics, Roche, Sangamo Therapeutics, Bluebird Bio, Kura Oncology, Translate Bio, Krystal Biotech, Replimune, StrideBio, Cardior, Generation Bio, Evox Therapeutics, and Mediphage Bioceuticals are some of the most prominent players in the global gene therapy market.

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