Gene Editing beyond CRISPR Market: Focus on Zinc Finger Nucleases, Transcription Activator-Like Effector Nucleases and Meganucleases Edited Therapies: Distribution by Type of Payment and Distribution by Geography: Industry Trends and Global Forecasts, 2022-2035

Isolation of the first site-specific restriction enzyme, Hind II, in the 1970s, became one of the breakthrough advances in biotechnology. This led to the discovery of various methods for manipulating living creatures at the genomic level, thereby, opening up a slew of new possibilities in basic and applied life sciences domain. Soon after, in the 1980s, the US FDA authorized human insulin, marketed under the brand name HUMULIN®, as the world's first genetically modified medication. With the development of various DNA modulation technologies, such as zinc-finger nucleases (ZFNs), TAL effector nucleases (TALENs), engineered endonucleases / meganucleases (EMNs) and clustered regularly interspaced short palindromic repeats (CRISPR), genetic engineering and genome editing concepts have gained significant attention over the last two decades. In fact, there have been several advancements in the field of genome editing, which provide investigators the ability to introduce sequence-specific modifications into the genomes of a broad spectrum of cell types and organisms.

Several medical researchers and industry stakeholders are presently engaged in exploring the potential of different gene editing technologies for basic research and development of gene editing solutions. However, the therapeutic use of these versatile genetic manipulation tools is only being investigated by selective stakeholders in the pharmaceutical and biotechnology sector. This can be attributed to the implementation of surrogate licensing model, which has granted exclusive control of the associated intellectual property (IP) to the drug developers. Clinical trials of ZFNs, TALENs and meganuclease based therapeutics are primarily focused on infectious diseases and oncological disorders; however, several product candidates against certain hematological disorders, genetic disorders and neurological disorders are being evaluated in discovery and preclinical stages of development. Over time, a number of industry and non-industry players have also been validating the therapeutic applications of these technologies, which has, in turn, prompted the establishment of strategic partnerships. In fact, the growing popularity of such technologies has attracted an investment worth USD 2 billion into companies engaged in this field of research. Promising clinical results, and ongoing technical developments, coupled with the growing interest of biopharmaceutical developers, are anticipated to push the pipeline products to higher phases of development. We believe that the market is likely to evolve at a sustained pace over the next decade.

Scope of the Report

The “Gene Editing beyond CRISPR Market: Focus on Zinc Finger Nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs) and Meganucleases Edited Therapies: Distribution by Type of Payment (Upfront and Milestone Payment) and Distribution by Geography (North America, Europe, Asia-Pacific and Rest of the World): Industry Trends and Global Forecasts, 2022-2035” report features an extensive study on the current market landscape, offering an informed opinion on the likely evolution of the ZFNs, TALENs and meganucleases based therapies market, in the mid to long term. The study underlines an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain.

Amongst other elements, the report includes:

• A detailed assessment of the current market landscape of ZFNs based therapies, based on several relevant parameters, such as phase of development (clinical and preclinical), biological target (BCL 11A, C9ORF72, CD 19, HLA-A2, HTT gene, MAPT, SNCA gene, VEGF-A, HPV16/E7 and HPV18/E7), delivery vehicle (non-viral and naked plasmid), target indication, therapeutic area (blood disorders, infectious diseases, neurological disorders, oncological disorders and others) and type of therapy (in-vivo and ex-vivo). In addition, it provides details on the companies engaged in the development of ZFNs based therapies, along with information on their year of establishment, location of headquarters and company size.
  
• A detailed assessment of the current market landscape of TALENs based therapies, based on several relevant parameters, such as phase of development (clinical and preclinical), target indication, type of cells (T Cells, NK Cells, HPV positive and B Cells), biological target (iNK, CD19, BCMA, TCR-α/CD52, CD22, CD38, CS1, GPCR, HPV18/E7, HPV16/E6, HPV16/E7 and HPV18/E6), cell / gene therapy (NK-Cell Therapy, T-Cell Therapy and CAR-T), type of therapy (in-vivo and ex-vivo), gene editing approach (insertion and deletion) and route of administration (intravenous, topical and intravaginal). In addition, it provides details on the companies engaged in the development of TALEN based therapies, along with information on their year of establishment, company size and location of headquarters.
  
• A detailed assessment of the current market landscape of Meganucleases based therapies, based on several relevant parameters, such as phase of development (clinical and preclinical), gene editing approach, delivery vehicle (AAV9 and lentiviral), therapeutic area(s) (genetic disorders, liver disorders, kidney disorders, rare disorders, ocular disorders and oncological disorders), route of administration, type of cells, type of therapy and biological target(s) (HA01, TTR, PCSK9 and ApoC3). In addition, it provides details on the companies engaged in the development of meganucleases based therapies, along with information on their year of establishment, company size and location of headquarters.
  
• Detailed profiles of prominent players engaged in the development of ZFNs, TALENs and meganucleases based therapies. Each profile features a brief overview of the company, details on its drug candidates, its financial information (if available), recent developments and an informed future outlook.
  
• A detailed analysis of completed and ongoing clinical trials of various ZFNs, TALENs and meganucleases based therapies, based on different parameters, such as trial status, trial registration year, target disease indication, trial phase, study design, number of patients enrolled and type of sponsor / collaborator. In addition, the chapter highlights the active industry players and location of the trials.
  
• A detailed review of around 1,250 peer-reviewed, scientific articles related to research on ZFNs, TALENs and meganucleases based therapies on the basis of several parameters, such as year of publication, therapeutic area, emerging focus area. The chapter also highlights the top journals and top authors (in terms of number of articles published).
  
• An in-depth analysis of academic grants that have been awarded to various research institutes for projects related to ZFNs, TALENs and meganucleases based therapies, during the period, 2017-2021, based on parameters, such as year of grant award, amount awarded, type of funding institute center, activity code, support period, prominent program officers, emerging focus areas and type of grant application. Further, the chapter also highlights the popular recipient organizations, (in terms of number of grants and amount awarded), study section, amount awarded and purpose of grant, and location of recipients.
  
• An analysis of the partnerships that have been established in this domain till 2021, covering instances of R&D agreements, clinical trial agreements, mergers / acquisitions, product development and commercialization agreements, licensing agreements, asset acquisitions and product development and manufacturing agreements. 
  
• A detailed analysis of the various investments made till 2021, including grants, seed financing, venture capital financing, IPOs, secondary offering, other equity, post IPO equity and equity crowdfunding in the companies focused on the development of therapies based on ZFNs, TALENs and meganucleases.
  
• An insightful analysis of the patents filed / granted for ZFNs, TALENs and meganucleases till 2021, taking into consideration various parameters, such as type of patent, issuing authority / patent offices involved, Cooperative Patent Classification (CPC) symbols, type of company, leading industry and non-industry players (on the basis of number of patents) and individual patent assignees (in terms of size of intellectual property portfolio).
  
• A list of key opinion leaders (KOLs) within this domain, and their assessment (based on the strength and activeness) represented in the form of 2×2 matrices. The chapter also includes an analysis evaluating the (relative) level of expertise of different KOLs, based on number of publications, number of citations, participation in clinical trials, number of affiliations and strength of professional network.
  
• A case study on the CRISPR / Cas based therapies that are currently in different stages of development. The chapter features a detailed analysis of pipeline molecules, based on various 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. 
  

One of the key objectives of the report was to estimate the existing market size and identify potential future growth opportunities of novel technologies for the development of ZFNs, TALENs and meganucleases based therapies in the mid to long term. Based on the likely licensing deal structures and agreements that are expected to be signed in the foreseen future, we have provided informed estimates on the evolution of the market over the period 2021-2035. The future opportunity within the gene editing market has been segmented across [A] type of payment (upfront payment and milestone payment) and [B] geography (North America, Europe, and Asia Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.

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.

Key Questions Answered

• Who are the players engaged in the development of gene editing therapies beyond CRISPR?
  
• Which are the key drugs being developed across early and late stages of development?
  
• Which companies are actively involved in conducting clinical trials for ZFNs, TALENs and meganucleases based therapies?
  
• What is the focus of various publications related to ZFNs, TALENs and meganucleases based therapies?
  
• Which are the leading administering institute centers supporting the research related to gene editing market beyond CRISPR?
  
• What kind of partnership models are commonly adopted by industry stakeholders?
  
• What is the trend of capital investments in the gene editing beyond CRISPR market?
  
• How has the intellectual property landscape in this market evolved over the years?
  
• How is the current and future opportunity, related to ZFNs, TALENs and meganucleases based therapies, likely to be distributed across key market segments?
  

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