Table of Contents
Several disorders that arise inside the body are a result of either a direct genetic aberration or a dysfunctional/non-functional protein. The attempt to use nucleic acids to correct or delete the genes causing a particular disease is known as gene therapy. Although gene therapy has not contributed significantly to the global pharmaceutical market yet, it is anticipated to grow at a fast pace over the next decade.
Gendicine, developed by SiBionoGeneTech, was the foremost gene therapy that entered market in 2004 in China. Since then four more therapies have received approval in China, Philippines, Russia and the EU. This number for approved / marketed therapies seems weak at present; however, the strong and highly populated pipeline holds tremendous potential. There are 12 gene therapies in late stage of clinical development for the treatment of cancer, ocular and cardiovascular disorders.
There are several concerns that remain to be answered; examples include insertional mutagenesis, treatment of multigene disorders, curbing the risk of immune reactions, eugenics, high cost of therapy and ethical concerns related to making alterations at the genetic level. Despite this, gene therapy does offer a ray of hope for patients who either have no treatment options or show no benefits with drugs that are currently available. Such a benefit far outweighs any disadvantages that may be associated with this upcoming therapeutic field.
There is a lot research underway for the first generation of controlled gene therapy, gene expression cassettes which deliver optimised gene expression and vectors which minimise the risk of immunogenicity. These research efforts have received strong support from various venture capital firms and institutional investors. With the expected launch of several new gene therapies in the US and EU, we expect the market to witness significant growth in the coming decade.
SCOPE OF THE REPORT
The ‘Gene Therapy Market, 2015-2025’ report provides an extensive study on the marketed and pipeline gene therapies. A lot of research has been carried out in this field for over a decade but there are only five approved therapies (four available in Asian markets; one approved in the EU). There are many promising therapies which are currently being developed worldwide; the approach is likely to result in several commercial success stories in the foreseen future. The report covers various aspects, such as key players, marketed gene therapy products, products in clinical / pre-clinical research, associated ethical issues, likely future developments and upcoming opportunities for a variety of stakeholders.
As pharma companies continue to expand their research programs in this area, one of the key objectives outlined for this report is to understand the future potential of the market. This is done by analysing:
- The gene therapy pipeline in terms of phase of development, therapeutic area, target indications, types of vectors, gene types, etc.
- Interest of venture capital firms and recent funding rounds that have taken place in the gene therapy space.
- Development and sales potential based on target consumer segments, likely adoption rate and expected pricing.
- The constraints of gene therapy and future developments planned to overcome these.
The study provides an estimate of the short-midterm and long term market forecast for the period 2015 - 2025. The research, analysis and insights presented in this report include potential sales of the five approved gene therapies and 12gene therapy products in late phases of development.
Owing to niche nature of the market, with most products in the pipeline, we have provided three market forecast scenarios to add robustness to our model. The conservative, base and optimistic scenarios represent three different tracks of industry evolution. All actual figures have been sourced and analysed from publicly available information and discussions with industry experts. The figures mentioned in this report are in USD, unless otherwise specified.
1. Gendicinebecame the first approved gene therapy in China in 2004. So far, there have been three more marketed gene therapies:Rexin-G, Oncorine and Neovasculgen. All of these have been made commercially available in Asian countries. Glybera was the first gene therapy to be approved in the EU in 2012.
2. We have identified a total of 483 gene therapy molecules in the marketed and the clinical pipeline which are designed for different therapeutic areas. Except a few which are in advanced stages of development, majority of the gene therapies are in early development stages.
3. There are several challenges and ethical issues associated with this field; germline gene therapy is one of the important issues. The existing gene therapies use somatic cells. However, research is in progress to introduce germline gene therapy in the market since some of the serious inherited genetic diseases, such as Lecsh-Nyhan Syndrome, can only be treated by germline gene therapy.
4. There has been a growing interest from Venture Capital firms in the gene therapy space. In fact, in 2013 / 2014, several companies were involved in investment in gene therapies; during January 2013 to April 2014, a total USD 600 million was raised by the US companies alone. This activity is likely to increase further and will help companies, especially start-ups, drive their respective products to commercialisation.
5. We expect the genetherapymarket to be worth USD 11 billion by 2025, representing a healthy annual growth rate of 48.9%; specific therapies such as Prostavac, ProstAtak and TroVax are likely to achieve blockbuster status.
Most of the data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.
The secondary sources of information include
- Annual reports
- Investor presentations
- SEC filings
- Industry databases
- News releases from company websites
- Government policy documents
- Industry analysts’ views
While the focus has been on forecasting the market over the coming ten years, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.
Chapter 2 presents an executive summary of the report. The summary offers key highlights of the gene therapy market and a high level view on where the market is headed in the mid-long term.
Chapter 3 provides a detailed introduction to the market. In this section, we have talked about theclassification and routes of administration of gene therapy. It also includes the advantages, challenges and ethical issues associated with gene therapy.
Chapter 4 provides a comprehensive view of the gene delivery systems. It includes details about the different viral and non-viral vectors associated with the process of gene therapy.
Chapter 5 includes information on over 483 molecules which are currently approved orin different stages of development (both clinical and pre-clinical / discovery). The detailed analysis of this pipeline covers information on drug developers, phases of development, therapeutic areas, target indications, vectors used, gene types and the genes involved.
Chapter 6 provides details on the marketed therapies. The chapter includes detailed profiles of marketed gene therapy products. We have presented information on several parameters such as history of development, clinical trial results, manufacturing, costs, collaborations and the likely sales forecast over the next ten years.
Chapter 7 includes detailed profiles of gene therapy products in late stage clinical trials. We have presented information on several parameters such as history of development, technology used, clinical trial results, manufacturing, costs, collaborations and the likely sales forecast over the next ten years.
Chapter 8 highlights the promising new therapeutic areas for gene therapies. It elaborates on the number of products being developed under each therapeutic area.
Chapter 9 includes details about the venture capital investments that have taken place in this field. It also highlights the contract manufacturers participating in development of gene therapy.
Chapter 10 summarises the overall report. In this chapter, we provide a recap of the current gaps in the industry and highlight the future scope of gene therapy.
Chapter 11 is a collection of interview transcripts of the discussions which were held during the course of this study.
Chapter 12 and 13 are appendices, which provide the list of companies and tabulated data for all the figures presented in the report
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