Gene Therapy Market (3rd Edition), 2019-2030

NEW YORK, Sept. 30, 2019 /PRNewswire/ --

INTRODUCTION

Since the approval of the first therapy, Genedicine® in 2003, the gene therapy domain has evolved significantly. Specifically, in 2019, three gene therapies, namely Zolgensma® (US), Zynteglo™ (Europe) and Beperminogene Perplasmid (Japan), have received approval / conditional approval, leading to a marked upward surge in the interest in this field. In fact, the growing popularity can be correlated to the substantial increase (more than 90%) in the number of patents that have been filed / granted in the last three years. Moreover, in the same time period, more than USD 12.5 billion in capital has been invested by various private and public investors to fund research activities. Presently, there are more than 10 approved gene therapies in the market, while many others are being investigated across various phases of clinical research.

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Over time, the efforts of industry stakeholders and clinical researchers have led to the discovery of novel molecular targets, thereby, strengthening the research pipelines of companies involved in the development of gene therapies. Further, several technology developers have designed innovative ways to improve the efficacy and safety of gene therapies and introduced advanced therapy development and vector engineering platforms. It is also worth mentioning that, in the last 4-5 years, there has been a marked rise in the M&A activity in this domain, including the involvement of several big pharma players as well. The capability of such therapies to target diverse disease indications is considered to be amongst the most prominent growth drivers of this market. Backed by promising clinical results and several therapy candidates in late phases of development, we believe that the overall market is expected to witness tremendous growth in the coming decade.



SCOPE OF THE REPORT



The "Gene Therapy Market (3rd Edition), 2019-2030" report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies and genome editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.



Amongst other elements, the report features:

• A detailed review of the overall landscape of gene therapies and genome editing therapies, including information on various drug / therapy developer companies, phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others) and target disease indication(s), information on gene type, type of vector used, type of therapy (ex vivo and in vivo), mechanism of action, type of gene modification (gene augmentation, oncolytic viral therapy and others) and special drug designation (if any).

• A discussion on the various types of viral and non-viral vectors, along with 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 engaged in developing gene therapies, and a bull's eye analysis, highlighting the distribution of clinical-stage pipeline candidates by phase of development, type of vector and type of therapy (ex vivo and in vivo).

• A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.

• Detailed profiles of marketed and phase II/III and gene therapies, including a brief history of development, information on current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, along with information on the developer company.

• An elaborate discussion on the various commercialization strategies that can be adopted by drug developers for use across different stages of therapy development, namely prior to drug launch, at / during drug launch and post-marketing.

• A review of various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.

• An in-depth analysis of the various patents that have been filed / granted related to gene therapies and genome editing therapies, since 2016. The analysis also highlights the key parameters associated with the patents, including information on patent type (granted patents, patent applications and others), publication year, regional applicability, CPC classification, emerging focus areas, leading industry / non-industry players (in terms of the number of patents filed / granted), and patent valuation.

• A analysis of the various mergers and acquisitions that have taken place in this domain, highlighting the trend in the number of companies acquired between 2014-2019. The analysis also provides information on the key value drivers and deal multiples related to the mergers and acquisitions that we came across.

• An analysis of the investments made at various stages of development in companies that are focused in this area, between 2014-2019, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other offerings.

• An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio.

• A case study on the prevalent and emerging trends related to vector manufacturing, with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.

• A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

• An analysis of the various factors that are likely to influence the pricing of gene-based therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.



One of the key objectives of the report was to estimate the existing market size and the future opportunity for gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2019-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others), [B] various types of vectors used for therapy development (adeno associated virus, adenovirus, lentivirus, plasmid DNA, retrovirus and others), [C] type of therapy (ex vivo and in vivo), [D] type of gene modification (gene augmentation, oncolytic viral therapy and others) and [E] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry's growth.



The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals (in alphabetical order):

• 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)

• Robert Jan Lamers (CEO, Arthrogen)

• Ryo Kubota (Chairman, President and Chief Executive Officer, Acucela)

• Tom Wilton (Chief Business Officer, LogicBio Therapeutics)



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 METHODOLOGYThe 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 till 2030, the report also provides our independent view on various emerging trends 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 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.



Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas and the route of administration of such therapeutic interventions. In addition, it includes information on the various steps involved in the transfer of therapeutic gene(s) into the body, along with a discussion on the advantages and disadvantages of this treatment paradigm. Further, the chapter features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.



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 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, China, Japan, South Korea and Australia. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.



Chapter 6 includes information on over 450 gene therapies and genome editing therapies that are currently approved or are in different stages of development. It features a comprehensive analysis of pipeline molecules, highlighting the drug developers, key therapeutic areas and target disease indication(s), phases of development, type of vector used, target gene type, type of therapy (gene augmentation / oncolytic viral therapy / others), type of somatic cell therapy (in vivo / ex vivo), mechanism of action and drug designation (if any). 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 presented 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 information related to manufacturing.



Chapter 8 features an elaborate discussion on the various strategies that can be adopted by the therapy developers across key commercialization stages, namely prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis on the key strategies adopted by drug developers for the commercialization of their gene therapies that were approved post 2014.



Chapter 9 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 10 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. In addition, it features brief profiles of some of the key technologies. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.



Chapter 11 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 12 provides insights from a detailed patent analysis, presenting an overview on the filed / granted patents related to gene therapies and genome editing therapies since 2016. For this analysis, we looked at the patents that have been published by various players till May 2019. It also highlights the important information and trends associated with these patents, including patent type (granted patents, patent applications and others), patent publication year, regional distribution, CPC classification, emerging focus areas and the leading industry / academic players (in terms of the number of patents filed / granted). The chapter also includes a patent benchmarking analysis and a detailed valuation analysis.



Chapter 13 features a detailed analysis of the various mergers and acquisitions that have taken place in this domain, highlighting the trend in the number of companies acquired between 2014-2019, based on parameters such as key value drivers, year of acquisition, type of acquisition, geographical location of the acquirer and the acquired company, and financial details of the deal (if available). In addition, the chapter presents a schematic world map representation of the geographical distribution of this activity, highlighting intracontinental and intercontinental deals.



Chapter 14 presents details on various funding instances, investments and grants that have been made within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2014 and 2019, highlighting the growing interest of the venture capital community and other strategic investors in this domain.



Chapter 15 highlights our views 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 of its proprietary products.



Chapter 16 highlights top ten big biopharma players in the field of gene therapy. It features tabulated profiles of the companies and each profile includes a brief overview of the company, its financial information (if available), information on its product portfolio and recent developments.



Chapter 17 presents a comprehensive forecast analysis, highlighting the future potential of the market till the year 2030. It also 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, muscular disorders, neurological disorders, ocular disorders, oncology and others), type of vector (adeno associated virus, adenovirus, lentivirus, plasmid DNA, retrovirus and others), type of somatic cell therapy (ex vivo and in vivo), type of gene modification (gene augmentation, oncolytic viral therapy and others) and geography (the US, EU5, RoW (Australia, China, Israel Japan and South Korea)).



Chapter 18 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain, and highlights some of the recent collaborations and developments related to manufacturing processes of gene therapies. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.



Chapter 19 provides a general overview on the supply chain of gene therapies. It features the process and steps followed in the supply chain to deliver gene therapies to target patients for the treatment of various rare disorders.



Chapter 20 summarizes the entire report. It presents a list of key takeaways and offers our independent opinion on the current market scenario. Further, it captures the evolutionary trends that are likely to determine the future of this segment of the gene therapies industry.



Chapter 21 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), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (Chairman, President and Chief Executive Officer, Acucela) and Tom Wilton (Chief Business Officer, LogicBio Therapeutics). In addition, a brief profile of each company has been provided.



Chapter 22 is an appendix, which provides tabulated data and numbers for all the figures included in the report.



Chapter 23 is an appendix, which contains a list of companies and organizations mentioned in this report.



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