Gene Therapy Market, (2nd Edition), 2018-2030

LONDON, June 12, 2018 /PRNewswire/ -- INTRODUCTION
The concept of gene transfer into mammalian cells can be traced back to the 1920s. However, the first gene therapy, Gendicine®, was only approved in 2003 in China; since then, the domain has evolved significantly. The year 2017 was particularly eventful; despite the withdrawal of Glybera® from the European market in early 2017, the latter half of the year witnessed the approval of two gene therapies, namely Invossa™ and Luxturna™. In fact, Luxturna™ became the first gene therapy to gain approval in the US. Further, promising clinical results were reported for several gene therapies that are currently in late phases of development. The growing popularity and potential of gene therapies can be correlated with an exponential increase in the number of patents that have been filed; the cumulative number has increased from 7,300 patents in 2013 to 42,300 patents till the third quarter of 2017. Further, over the past five years, capital worth more than USD 9.8 billion has been invested by venture capital (VC) firms and government bodies to fund research activities in this domain.

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The overall market is expected to witness significant growth in opportunities for a variety of stakeholders in the coming decade. It is important to highlight that several technology providers, aiming to develop and / or support the development of gene therapies, with improved efficacy and safety, have designed and already introduced advanced platforms for the engineering of vectors. Innovation in this domain has also led to the discovery of novel molecular targets and strengthened the research pipelines of companies focused in this space. The capability to target diverse therapeutic areas is considered to be amongst the most prominent growth drivers of this market.

SCOPE OF THE REPORT
The 'Gene Therapy Market (2nd Edition), 2018-2030' report provides an extensive study on the current market landscape of gene therapies, with a prime focus on gene augmentation based therapies and oncolytic viral therapies, featuring an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features:
• A detailed assessment of the current market landscape of gene therapies, providing information on various drug / therapy developers, phase of development (clinical, preclinical or discovery stage) of product candidates, key therapeutic areas and indication(s), information on gene type, vector type, type of therapy (ex vivo, in vivo) and type of modification (gene augmentation, oncolytic viral therapy and others).
• A discussion on the various types of viral and non-viral vectors, highlighting information on design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vectors.
• A world map representation, depicting the most active geographies in terms of the presence of companies developing gene therapies, and a bull's eye analysis highlighting the distribution of clinical pipeline candidates in terms of phase of development, type of vector and type of therapy (ex vivo / in vivo).
• Comprehensive profiles of marketed drugs, highlighting their history of development, current developmental status, mechanism of action, affiliated technology, patent portfolio, dosage and manufacturing, and information on the developer.
• Comprehensive profiles of clinical stage (phase II/III and above) drug candidates, highlighting their current status of development, mechanism of action, affiliated technology, patent portfolio, clinical trial information and recent developments.
• A section on emerging technologies and platforms that are aiding the development of gene therapies, featuring detailed profiles of technologies that are presently being used in the development of four or more products / product candidates.
• An overview of the most commonly targeted therapeutic indications and details on the gene therapies that are being developed against them.
• An analysis of the investments made at various stages of development in companies that are focused in this area, including seed financing, venture capital financing, debt financing, grants, capital raised from IPOs and subsequent offerings.
• A detailed analysis of the recently filed patents (since 2013); the study highlights the emerging trends in innovation and identifies the key players involved. In addition, it presents a high-level view on the valuation of these patents as well.
• A case study on the prevalent and emerging trends in vector manufacturing, with information on companies offering contract services for manufacturing vectors. The study includes a detailed discussion on the manufacturing processes of various types of vectors as well.
• An analysis on the various factors that may form the basis for the pricing of gene therapies, featuring different models / approaches that may be adopted in order to decide the price of a product that is likely to be marketed in the coming years.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the market. Based on parameters, such as target consumer segments, likely adoption rates and expected pricing, we have provided an informed estimate of the likely evolution of the market in the short to mid-term and mid to long term, for the period 2018-2030. To account for the uncertainties associated with the development of gene therapies and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market's evolution.

The opinions and insights presented in the report were also influenced by discussions held with senior stakeholders in the industry. The study includes detailed transcripts of discussions held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Christopher Reinhard (CEO and Chairman, Cardium Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory), Michael Triplett (CEO, Myonexus Therapeutics) and Robert Jan Lamers (CEO, Arthrogen). All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY
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 12 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 OUTLINES
Chapter 2 provides an executive summary of the insights captured during our research. It offers a high-level view on the likely evolution of the gene therapies in the short to mid-term and mid to long term.

Chapter 3 provides a general overview of gene therapies covering their historical background, popular types (somatic versus germline; in vivo versus ex vivo), application areas and route of administration. It also includes information on the steps required to transfer gene(s) into the body, along with a discussion on the advantages and disadvantages of the process. Further, the chapter features a discussion on the ethical and social concerns related to gene therapies, and highlights certain future constraints and challenges in terms of manufacturing, reimbursements and commercial viability. It also presents details on the regulatory guidelines for these therapies in the US, Europe and China.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available vectors.

Chapter 5 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. It also features the challenges that exist in this domain, and highlights some of the recent collaborations and developments related to manufacturing processes related to gene therapies. In addition, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 6 includes information on over 300 gene therapies that are currently approved or are in different stages of development. It features a comprehensive analysis of pipeline molecules, highlighting the drug developers, target therapeutic areas and indications, phases of development, vectors, target gene segment, type of therapy (gene augmentation / oncolytic viral therapy / others) and type of somatic cell therapy (in vivo / ex vivo). In addition, it features a schematic world map representation, highlighting the key regional hubs where gene therapies are being developed for the treatment of various disorders. Further, we have provided a logo landscape of product developers in North America, Europe and the Asia Pacific region on the basis of company size.

Chapter 7 provides detailed profiles of marketed gene therapies. Each profile includes an overview of the developer and information on various other parameters, such as history of development, indication, mechanism of action, patent portfolio, current developmental status, target, clinical trial results and manufacturing information.

Chapter 8 contains detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each profile provides information on the mechanism of action, current status of development, route of administration, affiliated technology platform (if applicable), dosage form, clinical studies and key clinical trial results.

Chapter 9 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. It features brief profiles of some of the key technologies as well. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships established related to the same. In addition, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 10 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include cardiovascular disorders, hematological disorders, metabolic disorders, muscular disorders, neurological disorders, ocular disorders and oncology.

Chapter 11 presents details on various investments and grants received by companies that are engaged in this domain. It includes an analysis of the funding instances that have taken place in the period between 2013-2017, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 12 highlights our view point on the various factors that may be taken into consideration while pricing gene therapies. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt to decide the price at which their gene therapy product is likely to be marketed.

Chapter 13 provides a detailed analysis of the patents that have been filed in this domain. The study highlights emerging trends in recently filed patents, and identifies the key players that are driving the innovation in this space. Moreover, it presents a high-level view on the valuation of these patents as well.

Chapter 14 presents a comprehensive forecast analysis, highlighting the future potential of the market till the year 2030. It includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of key therapeutic areas (cardiovascular disorders, hematological disorders, muscular disorders, ocular disorders, oncology and others), type of vector (AAV, adenovirus, lentivirus, plasmid DNA, retrovirus and others), type of somatic cell therapy (ex vivo and in vivo) and geography (the US, EU, RoW (Australia, China, Israel Japan and South Korea)).

Chapter 15 is a summary of the overall report. In this chapter, we have provided a list of key takeaways from the report, and expressed our independent opinion related to the research and analysis described in the previous chapters.

Chapter 16 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Christopher Reinhard (CEO and Chairman, Cardium Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) Michael Triplett (CEO, Myonexus Therapeutics) and Robert Jan Lamers (CEO, Arthrogen). In addition, a brief profile of each company has been provided.

Chapter 17 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 18 is an appendix, which provides the list of companies and organizations mentioned in the report.

EXAMPLE HIGHLIGHTS
1. Around 300 product candidates are currently under various stages of development for a diverse range of indications. Eight gene therapies are commercially available; of these, Imlygic® (Amgen), Strimvelis® (GSK), Invossa™ (TissueGene / Kolon Life Science / Mitsubishi Tanabe Pharma) and Luxturna™ (Spark Therapeutics) were approved after 2015. Nearly 48% of the pipeline molecules are under clinical development; of these, 21 molecules are being investigated in phase II/III and above, 8 molecules in phase III (planned), 21 molecules in phase II, 48 molecules in phase I/II and 49 molecules in phase I clinical trials. However, majority (49%) of the product candidates in the pipeline are still in the preclinical and discovery stages.
2. A significant proportion (59%) of product candidates in the pipeline follow the gene augmentation modification strategy, by delivering functional copies of missing genes into diseased hosts. Other types of modifications include immunotherapy (20%; delivering genes coding for antigens in order to generate an immune response) and oncolytic therapy (10%; using replication competent viruses to infect and kill cancer cells). Late stage (phase II/III and above) oncolytic therapies that are under development include ProsAtak® (prostate cancer), Pexa-Vec (HCC) and Toca-511 (GBM). In addition, a significant share of the pipeline comprises of in vivo gene therapies against various indications.
3. The market landscape is characterized by the presence of large-sized (17), mid-sized (34) and small-sized companies (63). Some of the prominent large-sized companies engaged in this domain include (in alphabetical order) Amgen, Biogen, BioMarin Pharmaceuticals, BMS, GSK, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi. Similarly, mid-sized companies that are actively contributing to the development of gene therapies include (based on year of approval) Spark Therapeutics, Kolon Life Science and Human Stem Cell Institute. In addition, small companies, such as (in alphabetical order) Agilis Biotherapeutics, Angionetics (subsidiary of Taxus Cardium Therapeutics), AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics and XyloCor, are also actively involved in this domain.
4. Close to 25% of the products in the development pipeline are designed to treat various oncological indications, including (ordered by decreasing number of gene therapies in the pipeline) prostate cancer, bladder cancer, head and neck cancer, breast cancer, lung cancer, ovarian cancer and pancreatic cancer. Nearly 15% of therapy candidates are being developed for the treatment of metabolic disorders (ordered by decreasing number of gene therapies under development), including mucopolysaccharidosis (MPS) IIIA, MPS IIIB, ornithine transcarbamylase (OTC) deficiency and diabetes. We also observed that close to 14% of the pipeline molecules are being developed for treating ocular disorders, such as (ordered by decreasing number of gene therapies under development) retinitis pigmentosa, wet age associated macular degeneration, achromatopsia and Leber's hereditary optic neuropathy.
5. AAV (38%) is presently the preferred type of vector used by drug developers to design gene therapies. This can be attributed to the advantages offered by these vectors, such as high viral yields, mild immunogenic response, ability to infect a wide spectrum of cells and a better safety profile. Other prominent delivery vehicles include adenovirus (16.5%), lentivirus (14.5%) and non-viral vectors (12%), such as plasmid DNA.
6. Over 34,000 patents, related to gene therapies, have been filed in last five years alone. Research efforts in this domain are led by both the industry and academia. Leading academic players that are actively engaged in developing gene therapies include (in decreasing order of number of patents) University of California, University of Texas, Stanford University, Harvard University, Massachusetts Institute of Technology and Johns Hopkins University.
7. Around 240 funding instances, amounting to USD 9.5 billion worth of capital, have taken place in order to support R&D in gene therapies in the last five years. California Institute for Regenerative Medicine (CIRM), OrbiMed Advisors, Deerfield Management, Versant Ventures, Novartis Venture Fund, National Institute of Health (NIH) and 5AM Ventures have emerged as some of the prominent investors (in terms of number of funding instances).
8. With many candidates in late stages of development, the market is likely to witness several approvals and grow steadily over the coming decade. Despite the fact that the first gene therapies were approved in Asian countries, North America and the EU together are likely to capture the over 75% of the market share by 2030. Within Asia Pacific region, South Korea is expected to dominate the market by 2030.

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