Gene Therapies & Associated Vectors: Intellectual Property Landscape

Gene Therapies & Associated Vectors: Intellectual Property Landscape

Although speculations related to using tailored DNA / RNA sequences to cure genetic disorders were made as early as the 1960s, the first reported use of a gene therapy was in 1990, to treat adenosine deaminase (ADA) deficiency related severe combined immunodeficiency (SCID). Briefly, a replication defective retrovirus was used to transfect autologous T-cells with a functional copy of the ADA gene, which restored normal immune function. The success of the aforementioned regimen spurred several similar attempts over the following years. However, it was the human genome project (1990-2003) that evidently ushered in the era of personalized medicine, turning the tide of treatment for several rare genetic conditions, which were previously considered undruggable. Since then, several gene-targeted therapies have been developed and launched; popular examples include ABECMA™ (Celgene, approved by the US FDA in 2021), BREYANZI (Juno Therapeutics, approved by the US FDA in 2021), SKYSONA™ (bluebird bio, approved by the EC in 2021), TECARTUS™ (Kite Pharma, approved by the US FDA in 2020), and ZOLGENSMA® (Novartis Gene Therapies, approved by the US FDA in 2019). In addition, there are over 1,700 product candidates under development, and close to 40 in the mid to late stages of development (phase II and above), which are anticipated to soon (two to three years) enter the market. Considering the target specificity of such products, many experts believe that this particular segment of modern advanced therapy medicinal products (ATMPs) truly offers the promise of lasting disease remission.

Although the COVID-19 pandemic limited certain aspects of the wider cell and gene therapies segment of the biopharmaceutical industry, it created significant future opportunities. From the financial perspective, the domain reported strong positive growth, primarily driven by drugs such as ZOLGENSMA®, which reported close to 50% year-on-year growth in 2021. In addition, some innovators, leveraging proprietary technologies, launched COVID-19 directed R&D programs, while others took the opportunity to reposition existing assets in the same direction; either way, industry stakeholders were able to tap into the vast infectious diseases market. Taking into consideration both historical and recent developments, and modern technological advances, it is evident that this upcoming industry segment is at an inflection point. With the rapidly growing success of genetic medicine prompting more innovators to enter the market, the focus is now on building better and more efficacious assets, based on next generation biotechnology platforms. This is likely to pave the way for a much broader wave of new biological interventions. It is, therefore, important to identify key pockets of innovation and areas of improvement, in order to truly innovate whilst maintaining a competitive edge. This report summarizes some of the key R&D trends related to gene therapies and affiliated vectors. It also provides a perspective on the pace and focus of innovation in this field, briefly describing the future of gene therapies as mainstream healthcare solutions.

SCOPE OF THE REPORT

The “Gene Therapies & Associated Vectors: Intellectual Property Landscape” report features an extensive study of some of the key historical and contemporary intellectual property (IP) documents (featuring granted patents, patent applications and other documents) describing gene therapies and affiliated vector constructs. The insights generated in this report have been presented across two deliverables, namely a MS Excel sheet and a MS PowerPoint deck, summarizing the ongoing activity in this domain. Key inclusions are briefly described below:

Overall Intellectual Property Landscape

An in-depth review of the various patents and affiliated IP documents that have been published related to the diverse technologies, methods, and compositions associated with the use of genomic data and synthetic DNA / RNA constructs, for therapeutic purposes. It features insights on both historical and recent trends in R&D within this niche.

Popular / Relevant Prior Art Search Expressions

An examination of IP literature, shortlisting key words and phrases that have been used to describe gene therapies and associated vector constructs. The analysis includes information on the historical use of the aforementioned terms across different types of IP filings, key affiliated terms (which can be used to further identify similar innovations), and other related trends.

Patent Valuation Analysis

A competitive benchmarking and valuation analysis of the IP documents published in this field of innovation, taking into consideration important parameters, such as type of IP document, year of application, time to expiry, number of citations and jurisdiction (factoring in regional GDP).

Patentability and Freedom to Operate

A systematic approach to identify relevant areas of innovation by analyzing published IP documents, defining the uniqueness of patented / patent pending innovations, understanding the scope of patentability in this domain, and pinpointing jurisdictions where new and / or modified claims may be filed without infringing on existing IP.

Analysis of Patent Applications

A detailed summary of the patent applications that were filed across different jurisdictions and their relative value in the IP ecosystem. The analysis segregates the intellectual capital in terms of area of innovation and intended applications, thereby, offering the means to understand key areas of research and identify innovation-specific IP filing trends.

Analysis of Granted Patents

An elaborate summary of the granted patents across different jurisdictions and their relative value in the IP ecosystem. The analysis also features a meaningful classification system, segregating granted patents into relevant categories to help develop a more detailed perspective on the diverse types of innovations in this domain and their intended applications, and the feasibility for innovators to enter into promising product markets.

Pockets of Innovation and White Spaces

An insightful analysis of the various CPC codes used in published IP literature and their affiliated families, in order to identify historical and existing pockets of innovation (based on the functional area / industry described by the elaborate and systematic system of classifying IP); the analysis also features a discussion on prevalent white spaces (based on CPC symbols) in this arena of research.

Claims Analysis

One of the objectives of the report was to analyze and summarize key inferences from the independent claims mentioned in granted, active patents in the dataset. Using a systematic segregation approach, we have analyzed trends associated with [A] the preamble, [B] type of patent (technology patent or method patent), [C] type of claim (open ended claim or closed ended claim) and [D] key elements of a claim (individual aspects of an innovation that are covered in a singular claim).

RESEARCH METHODOLOGY

The data presented in this report has been gathered via secondary research and analyzed via proprietary methods / tools to develop a detailed perspective on the current status of the innovative advances in this domain and affiliated developer landscape that is spread across different global regions. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include

Company websites

Annual reports

Patent information aggregator portals

Industry databases

Press releases

Industry analysts’ views

The insights presented are solely based on our knowledge, research and understanding of the relevant market, as gathered from various secondary sources of information.

DELIVERABLE OUTLINES

Excel Deliverable

Sheet 1 features details regarding how the input data for this project was collated, including the search strings used to query a popular patent database (lens.org), and data segregation notes.

Sheet 2 is a summary MS Excel dashboard, offering a detailed graphical perspective of the intellectual property landscape of gene therapies. It includes pictorial representations of the [A] overall patent landscape, [B] key prior art search expressions, [C] trends related to patent applications (including insights on patentability and freedom to operate), [D] trends related to granted patents (including insights on patentability and freedom to operate), [E] key inferences from a proprietary claims analysis, [F] list of popular CPC symbols (featuring key pockets of innovation), [G] list of popular applicants (shortlisted based on number of published IP documents).

Sheet 3 is an elaborate tabular representation of the overall IP landscape, featuring information on the various patent application- and granted patent-related documents, related to gene therapies, which have been published since 1985.

Sheet 4 includes a tabular representation of key words and phrases that are used to describe gene therapies and associated vectors.

Sheet 5 is a subset of sheet 3, featuring all the patent applications, covering innovations related to gene therapies and associated vectors.

Sheet 6 is a subset of sheet 3, featuring all the granted patents, covering innovations related to gene therapies and associated vectors.

Sheet 7 is an insightful summary of key inferences from the independent claims mentioned in the granted, active patents in the dataset. We have used a systematic segregation approach to analyze trends associated with the preamble, type of patent (technology patent or method patent), type of claim (open ended claim or closed ended claim) and key elements of a claim (individual aspects of an innovation that are covered in a singular claim).

Sheet 8 provides insights related to some of the key applicants in this field of research, featuring company-specific details (such as year of establishment, and location of headquarters), and inputs on their respective IP publication trends.

Sheet 9 features an analysis of the most popular CPC symbols and CPC families(in terms of frequency of appearance in the dataset), related to the diverse applications of gene therapies and associated vectors in the treatment of different clinical conditions with a genetic cause.

Sheet 10 is an appendix which includes pivot tables that drive the charts and interactive elements for the complete IP landscape, in sheet 2.

Sheet 11 is an appendix, featuring details related to the categorization done in the report, and important abbreviations used in reference to the data categories mentioned in the document.

PowerPoint Deliverable

Chapter 1 briefly describes of the need for targeted and more efficacious therapeutic interventions, and the key advantages of gene therapies. Further, it provides an overview of the intellectual property landscape related to gene therapies (including certain types of cell therapies and associated vector constructs).

Chapter 2 and 3 feature brief (pictorial) summaries of the approach used for data collection and the key objectives of the study.

Chapter 4 features an executive summary of the key insights generated from analyzing the intellectual property landscape of gene therapies and associated vectors.

Chapter 5 features a brief description of gene therapies and offers an informed perspective on the contemporary need for targeted and efficacious therapeutic solutions, in the treatment of clinical conditions that are characterized by a genetic cause; this includes oncological disorders, several metabolomic and degenerative diseases, and close to 40% of rare diseases. It provides an impactful summary of the important milestones in this field of research and highlights some of the distinguishing features of both early generation and modern variants of these personalized therapies. It also includes a list of important focus areas (in terms of innovation) and key areas of application. Finally, the chapter describes the various advantages of gene therapies and highlights existing limitations.

Chapter 6 includes a review of the various patents and IP documents that have been published related to technologies and methods associated with gene therapies and associated vectors, featuring insights related to both historical and recent trends.

Chapter 7 features an insightful examination of IP literature, highlighting key words and phrases that are used to describe gene therapies and associated vectors, including information on historical usage in IP filings, key affiliated terms (which can be used to further identify similar innovations), and other related trends.

Chapter 8 offers insights from a competitive benchmarking and valuation analysis of the IP documents published related to this field of research, taking into account important parameters, such as type of IP document, year of application, time to expiry, number of citations and jurisdiction (factoring in regional GDP).

Chapter 9 provides a detailed summary of the patent applications that were filed across different jurisdictions and their relative value in the IP ecosystem. The analysis segregates the intellectual capital in terms of area of innovation and intended applications, thereby, offering the means to understand key areas of research and identify innovation-specific IP filing trends. Based on a systematic approach, the chapter highlights relevant areas of innovation from a deeper analysis of published IP documents, defining the uniqueness of patent pending innovations, understanding the scope of patentability in this domain, and pinpointing jurisdictions where new and / or modified claims may be filed without infringing on existing IP.

Chapter 10 is an elaborate summary of the granted patents across different jurisdictions and their relative value in the IP ecosystem. The analysis uses a proprietary segregation criterion, based on type of product / solution and intended applications; this offers the means to identify unique innovations that presently have marketing exclusivity and explore future opportunities to enter into promising product markets, once their patents expire. Based on a systematic approach, the chapter highlights relevant areas of innovation from a deeper analysis of published IP documents, defining the uniqueness of patented innovations, understanding the scope of patentability in this domain, and pinpointing jurisdictions where new and / or modified claims may be filed without infringing on existing IP.

Chapter 11 features profiles of some of the most popular applicant companies, which were shortlisted based on patent filing activity. Each profiles includes, a brief overview of the company, information on annual revenues (wherever available), details of its initiatives focused on the healthcare sector, names of key management team members and recent developments.

Chapter 12 includes an insightful analysis of the various CPC symbols mentioned in the published IP literature and their affiliated families, in order to identify historical and existing pockets of innovation (based on the functional area / industry described by the elaborate and systematic system of classifying IP); the analysis also features a discussion on the prevalent white spaces (based on CPC symbols) in this area of research.

Chapter 13 concludes the report by providing insights on anticipated developments in this domain (from the perspective of eminent industry representatives of stakeholders in this domain), and trends that are likely to shape the future of the gene therapies market.

Chapter 14 is a set of appendices.