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.
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 the 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.
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.
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)
- [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.
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 a 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.
- An 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 the number of gene therapies under development, funding information, partnership activity and strength of the 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 of 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.
Key Topics Covered:
2 Executive Summary
4 Gene Delivery Vectors
5 Regulatory Landscape And Reimbursement Scenario
6 Competitive Landscape
7 Marketed Gene Therapies
8 Key Commercialization Strategies
9 Late Stage (Phase Ii/Iii And Above) Gene Therapies
10 Emerging Technologies
11 Promising Therapeutics Areas
12 Patent Analysis
13 Mergers And Acquisitions
14 Funding And Investment Analysis
15 Cost Price Analysis
16 Big Pharma Players: Analysis Of Gene Therapy Related Initiatives
17 Market Forecast And Opportunity Analysis
18 Vector Manufacturing
19 Case Study: Gene Therapy Supply Chain
21 Interview Transcripts
22 Appendix 1: Tabulated Data
23 Appendix 2: List Of Companies And Organizations
For more information about this report visit https://www.researchandmarkets.com/r/pbrmpf