Cell and Gene Therapy Fundamental Analysis

Cell and Gene Therapy Overview

Contemporary medical science has traced thousands of clinical conditions to a genetic cause. Cancer, a life-threatening disease, also has genetic origins, and is considered among the leading causes of death across the globe. In fact, the World Health Organization (WHO) has reported that close to 10 million cancer related deaths annually, across the world. Moreover, experts believe that there are over 7,000 different types of rare diseases (including some rare forms of cancer), most of which originate as a consequence of genetic anomalies. The majority of the aforementioned conditions are still considered incurable. As a result, these disease areas are characterized by a significant unmet need for curative interventions; and therefore, considered among the most lucrative opportunity areas for biopharmaceutical developers. For example, ZOLGENSMA®, a blockbuster product developed by Novartis, and indicated for the treatment of spinal muscular atrophy, generated net revenues of approximately USD 1.35 billion in 2021 alone. The first gene therapy trial was conducted in 1990, and it took almost three decades for the first of such interventions to enter the market. Given recent developments in genetic manipulation, cell biology and molecular targeting, a number of highly specific interventions have been developed against prominent types of cancers and certain rare genetic conditions. Currently, there are over 20 cell and gene therapies approved for use in the United States alone. 

During the COVID-19 pandemic, the pace of R&D in this field slowed down - a consequence of complex manufacturing protocols, extensive logistical considerations and supply chain-related concerns. However, the field still witnessed a considerable inflow of capital, with over USD 21 billion invested into various companies since the start of the pandemic. With over 1,200 product candidates in various stages of development, experts suggest that, by 2025, the US FDA may start approving around 10 to 20 cell and gene therapy products, on an annual basis. It is likely that, over the next two decades, gene therapies facilitate the evolution of medical practice from a treatment-based paradigm to a prevention-focused approach. Despite the fact that niche startups are spearheading the innovation in this domain, several big pharma players are also actively acquiring capabilities related to upcoming advanced therapy medical products (ATMPs). Prominent players in the field, such as Juno Therapeutics, AveXis, and Kite Pharma, have been acquired as a consequence of the rapid expertise building efforts of more established pharma companies. Moreover, gene therapy-focused businesses that have gone public, have experienced considerable growth in share value as their respective products / product candidate progressed through the various stages of development. Taking into consideration both the historical and contemporary scenario, the cell and gene therapies market continues to present lucrative investment opportunities for both short- and long-term investors.

Scope of the Report

The “Investor Series: Opportunities in the Cell and Gene Therapy Market” report provides detailed information on the cell and gene therapy industry, covering both core and peripheral products, and affiliated services. It offers a technical and financial perspective on how the opportunity in this domain is likely to evolve, in terms of future business success, over the coming decade. The information in this report has been presented across multiple deliverables, featuring MS Excel sheets (some of which include interactive elements) and an MS PowerPoint deck, which summarizes the key takeaways from the project, and insights drawn from the curated data.

The report features the following details:

• A qualitative and quantitative (wherever information was available) perspective on the current need for cell and gene therapies. It also provides information on some of the important applications of cell and gene therapies, the benefits of using such therapies over conventional treatment options, the various challenges faced in the development / production of these ATMPs, and the opinions of  representatives from key stakeholder companies involved in this domain. It also includes details of some exemplary ventures that have either succeeded or failed in this market, and highlights the various events, related to this field of research, which have already taken place, or yet to take place, since 2020. 
• A detailed analysis of cell and gene therapy focused companies that were established on or after 2005, featuring inputs on observed trends related to basic input parameters, such as year of establishment, headquarters, company size, and type of venture. 
• A quantitative perspective on the relative health (based on basic company details, product details, financing activity, and estimated revenues and profits) of different innovator companies that have been described in detail in this report. This analysis is based on a proprietary scoring criterion, which was informed via secondary research. 
• An assessment of the various products and affiliated services, offered by the companies mentioned above, featuring analysis based on number and type of product, and an informed perspective on the value of the aforementioned offerings based on multiple relevant aspects, namely, therapy-related value, value to patients, developer value, and others.  
• A company competitiveness analysis, which offers a quantitative basis for comparing the strengths / contributions of various industry stakeholders that are involved in the development of cell and gene therapies / product candidates, captured in this report. It is worth mentioning that this analysis is based on the insights generated from the abovementioned relative health indexing and value proposition analyses.
• A detailed analysis of the funding and investment activity that has taken place in this domain, since 2011. It also includes financing category-wise trends, describing the relative maturity (in terms of number of funding instances and total capital raised) of the key innovator companies discussed in the report. Further, it features a list of the leading investors in cell and gene therapy market, based on their participation in financing activity in this industry segment. 
• A proprietary analysis defining a basis for estimating the relative valuation of the private companies among the key innovators discussed in this report. The value statements generated under this analytical framework, were based on information gathered via secondary research, for a sample set of companies. Key inferences drawn from the sample set were then extrapolated using the appropriate mathematical models to generate the likely valuation estimates for all the companies in the dataset.
• An elaborate review of the overall cell and gene therapy market from a financial perspective, including detailed fundamental (insights from the balance sheet, and key financial ratios) and technical analyses (insights from historical and recent stock price variations, and analysis using popular stock performance indicators) of financial data of the publicly listed companies within the key innovators dataset. 
• A business risk analysis, focused on some of the major categories of risk that are usually discussed in the industry – namely operations-related risks, overall business-related risks, financial risks, product / technology associated risks, and social, economic, environmental and political risks. 
• Case studies of instances where investors have exited various cell and gene therapy-related ventures, offering insights on returns on investment made (based on availability of data). Leveraging the abovementioned details, the report offers an informed opinion on the future outlook for investors in the cell and gene therapy market. 
• A key acquisition targets analysis, based on the insights generated during the course of this study, highlighting some of the promising early-to-mid stage business ventures around which there is likely to be interest for future acquisitions / mergers.

One of the key objectives of the report was to evaluate the current opportunity and the future potential of cell and gene therapies over the coming decades. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2021-2035. The opportunity has been segregated on the basis of [A] key market segments (namely stem cell therapies, gene therapies and T-cell therapies) [B] therapeutic areas (namely oncological disorders, genetic disorders, metabolic disorders and others) [C] routes of administration (intravenous and others) and [D] geography (North America, Europe, Asia Pacific and the rest of the world). To account for future uncertainties in the market 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.