INTRODUCTION Clustered regularly interspaced short palindromic repeats (CRISPR) are a family of DNA sequences, which constitute a primitive immune system that is responsible for protecting prokaryotic cells from phage infections.

New York, Aug. 24, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global Forecasts, 2021-2030" - https://www.reportlinker.com/p06130494/?utm_source=GNW It was first described in 1987, however, its potential as a gene editing tool was not realized until 2012. Since then, the CRISPR revolution has not shown any signs of slowing down and has been responsible for significant advances in molecular biology and therapy development. Fundamentally, the CRISPR/Cas system involves specific palindromic DNA sequences which work in tandem with a family of caspase enzymes (Cas9, Cas12), in order to excise gene fragments with high precision. Compared to the other targeted nuclease-based systems, CRISPR is relatively faster, and cost-efficient; as a result, the demand for this gene editing tool is very high. The relatively recent discovery / development of novel accompanying nucleases, namely Cas12a, Cas13, Cas14 and dCas9, has significantly improved the precision of this technology. Presently, there are several companies using different variants of the CRISPR/Cas technology for basic research, and the development of gene editing solutions. However, the therapeutic use of this versatile genetic manipulation tool is only being investigated by a select few stakeholders in the pharmaceutical industry. The aforementioned scenario is attributed to the surrogate licensing model, which has granted exclusive control of the associated intellectual property (IP) to three leading players, namely Editas Medicine, CRISPR Therapeutics and Intellia Therapeutics, in the contemporary market.

Clinical trials of CRISPR based therapeutics are currently focused mainly on oncological and hematological disorders; however, several product candidates against certain neurological disorders and infectious diseases, specifically targeting recurrent conditions, are under investigation. Post 2014, the overall interest in this technology has grown exponentially, with several start-ups entering the market and 6 of the top 10 pharmaceutical companies restructuring their efforts in this direction. Over time, a substantial body of evidence has also been generated validating the therapeutic applications of this technology, which has, in turn, prompted the establishment of numerous strategic partnerships (focused on therapy development and clinical research) and has caused investors to put in significant capital into innovator companies involved in this domain, over the last two years alone. In fact, the three leading companies in this industry segment together have combined market capitalization of more than USD 10 billion, and have raised more than USD 2.8 billion in various funding rounds. Despite the possibly limitless potential of the CRISPR/Cas technology, further investigation, probing its safety and therapeutic efficacy in large diverse populations, is required. Key impediments to approval and other existing challenges that are being addressed by stakeholders, include off-target toxicity-related concerns and complexities related to the delivery of CRISPR components into target cells. Concerning delivery, innovators in this field have reported notable success using different types of platforms for facilitating the intracellular administration of CRISPR components; examples of successful delivery methods include electroporation, AAV vectors and lipid nanoparticles (LNPs). A few companies are also evaluating bacteriophages as a potential delivery system for such products. Promising clinical results, and ongoing technical developments, coupled to the growing interest of biopharmaceutical developers, are anticipated to push pipeline products to higher phases and on to commercialization. We believe that the market is likely to evolve at a commendable pace over the next decade.

SCOPE OF THE REPORT The CRISPR Based Therapeutics Market, 2021-2030 report features an extensive study of the current market landscape and future opportunity for the players involved in the development of CRISPR based therapeutics for the treatment of a variety of disease conditions. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographies. Amongst other elements, the report includes: A review of the CRISPR based therapeutics that are currently in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as target therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others), phase of development (discovery, preclinical and clinical), approach of therapy (ex vivo and in vivo), cell source (autologous and allogeneic), type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), and the type of technology used. It also includes information on the completed, ongoing and planned clinical trials for CRISPR based therapeutics, sponsored by various industry players. Elaborate profiles of key players in this domain. Each company profile features a brief overview of the company, its financial information (if available), a brief description of its therapeutic candidates, recent developments, and an informed future outlook. An in-depth analysis of around 2,000 patents related to CRISPR technology that have been filed / granted, since 2015, highlighting the key trends associated with these patents, across type of patent, publication year and application year, regional applicability, IPCR symbols, emerging focus areas, inventor information, leading patent assignees (in terms of number of patents filed / granted), patent benchmarking and valuation. An analysis of the partnerships that have been inked by various stakeholders engaged in the development of CRISPR based therapeutics, during the period 2014-2020, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. An analysis of the investments made at various stages of development of the companies engaged in this field, covering instances of seed financing, venture capital financing, grants / awards, capital raised from IPOs and subsequent offerings. An analysis of the start-ups (established in the time period between 2013-2020 and have less than 200 employees) engaged in the development of CRISPR based therapeutics, based on several parameters, such as number of candidates in discovery, preclinical and clinical phase of development, therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

One of the key objectives of the report was to estimate the future growth potential of CRISPR based therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2021-2030. For this purpose, we have segmented the future opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL), [D] key geographical regions (North America, Europe and Asia-Pacific). To account for uncertainties and to add robustness to our model, we have provided three market forecast scenarios, portraying the conservative, base and optimistic tracks of the anticipated industrys growth.

KEY QUESTIONS ANSWERED Who are the leading players engaged in the development of CRISPR based therapeutics? Which key clinical conditions can be treated by CRISPR based drugs? What are the investment trends in this industry? Which partnership models are commonly adopted by stakeholders engaged in this domain? How has the intellectual property landscape in this market evolved over the years? Which factors are likely to influence the evolution of this market? How is the current and future market opportunity likely to be distributed across key market segments?

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 and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been validated 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 views on various non-commercial trends emerging in this industry. This opinion is solely based on our knowledge, research and understanding of the relevant market trends 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 CRISPR therapeutics market and its likely evolution in the short-mid to long term.

Chapter 3 provides a general introduction to CRISPR/Cas system. In addition, we have briefly described the components of CRISPR/Cas system, its mechanism of action and vehicles to deliver CRISPR/Cas components in to the target cells. The chapter lays emphasis on the applications of CRISPR technology. It also includes a discussion on the challenges associated with the use of CRISPR based therapeutics.

Chapter 4 presents a detailed assessment of the current market landscape of CRISPR based therapeutics, along with information on type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), approach of therapy (in vivo and ex vivo), cell source (autologous and allogeneic), phase of development (discovery, preclinical and clinical), type of delivery vehicle used (viral vector, electroporation, LNPs, bacteriophage and others), target disease indication and therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others). In addition, it provides an overview of the CRISPR based therapeutics developer landscape, highlighting the players that are active in this domain. It includes information on their year of establishment, company size (in terms of number of employees) and location of headquarters of the drug developers. We have presented a logo landscape, highlighting the distribution of the drug developers based on company size and location of headquarters. Further, it presents an analysis on the initiatives of the big pharma companies in this domain.

Chapter 5 includes profiles of the key players engaged in the development of CRISPR based therapeutics (shortlisted based on strength of product portfolio). Each profile features a brief overview of the company, its financial information (if available), brief details of gene editing technology, therapeutic pipeline, recent developments and an informed future outlook.

Chapter 6 provides an in-depth analysis of the patents filed / granted for CRISPR technology since 2015. The analysis also highlights the key trends associated with these patents, including type of patent (granted patent, patent application and others), publication year, application year, geographical location / patent jurisdiction (North America, Europe, Asia-Pacific and Rest of the World), IPCR symbols, key inventors and leading industry / non-industry players. In addition, it includes a detailed patent benchmarking analysis of leading players and patent valuation analysis, which evaluates the qualitative and quantitative aspects of these patents.

Chapter 7 features a detailed analysis of the partnerships and collaborations that have been inked in this domain since 2014, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. The chapter includes analysis based on year of partnership, type of partnership model, purpose of licensing deal, and most active player(s) (in terms of number of partnerships inked). In addition, the chapter features a discussion on the surrogate licensing practice in the CRISPR based therapeutics market.

Chapter 8 provides an analysis of the investments made since 2014 at various stages of development of companies engaged in this domain, based on the year of investment, number of funding instances, amount invested and type of funding, highlighting most active players (in terms of number of funding instances and amount raised) and most active investors (in terms of number of funding instances).

Chapter 9 presents an analysis of the start-ups (established after 2012 and having less than 200 employees) engaged in the development of CRISPR based therapeutics based on the parameters, such as number of candidates in discovery, preclinical and clinical phase of development, target therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

Chapter 10 features an elaborate discussion on the future market potential of various CRISPR based therapeutics. The chapter provides insights on the likely distribution of the current and forecasted opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL) and [D] key geographical regions (North America, Europe and Asia-Pacific).

Chapter 11 is a summary of the overall report, highlighting the key facts and figures related to the research and analysis presented in the previous chapters.

Chapter 12 is a collection of interview transcripts of discussions held with representatives of renowned organizations engaged in the CRISPR technology domain. In this chapter, we have presented the insights on our conversation with Harrison Wong (Public Relations, Burns McClellan, for eGenesis).

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix, which consists the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06130494/?utm_source=GNW

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