Gene therapies have been the subject of tremendous scientific research and investment.  With hundreds of clinical trials in progress and many target applications, the biotechnology, agricultural, and pharmaceutical fields are pushing full speed ahead into this promising field.  Helping spur innovations in this field are important issues to be addressed including preventing off-target effects, engineering on- and off-switches to control the genetic modifications, finding safe and efficacious modes of delivering the gene therapy to intended targets, avoiding undesired immune responses, creating manufacturing and processing techniques and practices that are accepted by regulatory authorities, and establishing and validating analytical techniques to demonstrate safety, efficacy and reproducibility. 

Over the past 20 years, scientists have developed increasingly precise and efficient gene editing techniques using CRISPR and other gene editing technologies with applications directed towards treatment of a broad spectrum of human diseases and conditions, including ocular conditions, liver diseases, blood diseases, brain diseases, cancer, cardiovascular disease, metabolic disease, neurodegenerative disease, viral diseases, and hematological diseases.[1] Scientists have also developed gene editing technology based research tools with applications toward cell imaging, gene expression regulation, epigenetic modification, therapeutic drug development, functional gene screening, and gene diagnosis.[2]

GENE THERAPY REGULATORY LANDSCAPE[3]

These investments and tireless research endeavors have produced an increasingly crowded landscape of companies of various sizes vying for regulatory approval for therapies for numerous different types of conditions:

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FILED GENE THERAPY PATENTS AND PATENT APPLICATIONS OF ASSIGNEES WITH MARKETED OR CLOSE TO MARKET GENE THERAPY PRODUCTS[4]

Among the companies obtaining or nearing regulatory approval, a race to secure and defend intellectual property rights has resulted in a landscape visualized as follows:   

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As of the fourth quarter of 2022, there are a total of 3726 gene, cell and RNA therapies in development, with more than half being gene therapies.[5] Out of those therapies, 24 gene therapies are approved for clinical use globally as of Q4 of 2022.

APPROVED GENE THERAPIES AS OF Q4 2022[6]

Innovators in the gene therapy field have been increasing their patent filings.  As the field of published prior art has grown, the issuance rate of patents in this space has decreased as shown in the following figure:  

APPLICATION AND ISSUED PATENT TRENDS[7]

Interestingly, the number of applications filed in 2022 decreased precipitously compared to prior years.  It remains to be seen if this drop is the beginning of a trend or merely an anomaly.

ASSIGNEES WITH THE LARGEST PATENT PORTFOLIOS IN THE CRISPR TECHNOLOGY SPACE[8]  

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ASSIGNEE RELATIONSHIPS BETWEEN ASSIGNEES WITH THE LARGEST PATENT PORTFOLIOS IN THE CRISPR TECHNOLOGY SPACE[9]

Collaborations in the CRISPR field have resulted in commonly owned intellectual property as shown in the following table: 

Innovators in this space have also sought to protect the critical components of the technologies by claiming the guide RNAs and delivery systems used to deliver their CRISPR technologies in vivo or in vitro.  With the emergence of mRNA vaccines and approved lipid nanoparticle (LNP) delivery systems, gene editing companies have increasingly focused on lipid-based delivery vehicles, although viral vector delivery systems also remain in use as a favorite delivery platform.

ASSIGNEES WITH THE LARGEST PATENT PORTFOLIOS COVERING GUIDE RNAs[10]  

ASSIGNEES WITH THE LARGEST PATENT PORTFOLIOS COVERING LIPID DELIVERY SYSTEMS[11]

Over the last two decades, gene therapy has transformed from a promising discovery to one of the most significant biological breakthroughs of our time. The investments that research institutions, governments, biotechnology companies, and pharmaceutical companies have made into gene therapy technologies show that the field believes that such technologies will be an important tool in developing the next generation of therapeutics and research tools. Strong investment in this field also means that the patent landscape surrounding innovations in this area is becoming increasingly crowded.  Both existing players and new entrants in this field should investigate, understand, and continuously monitor the patent landscape to identify white space, landmines, and potential partnership or licensing opportunities. Existing market players should continually assess their existing patents and pending patent applications to identify opportunities to maximize and grow their patent estates. We will continue to monitor new developments in gene therapy technologies and provide updates.

[1] See, e.g. Hongyi Li, et al. Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects, 5 Sig. Transduct. Target Ther. 1 (2020).

[2] See, id at 17.

[3] Source: Wells Fargo Legacy Programs, 2021.

[4] Source: Search of US and WIPO databases Feb. 2023 (patent and patent application results generated using the keyword query: ALL_AN:(roche OR gensight OR novartis OR bluebirdbio OR “orchard therapeutics” OR biomarin OR abeona OR sangamo OR lysogene OR “ptc therapeutics” OR biogen OR regenxbio OR pfizer OR renova OR “krystal biotech” OR “csl behring”) AND TACD:(“gene therapy” or CRISPR or “gene edit*”).

[5] ITELINE, Gene, Cell, & RNA Therapy Landscape (2022) (Q4 2022 Quarterly Data Report, American Society of Gene + Cell Therapy).

[6] Source: Pharmaprojects, Citeline, Jan. 2023. See also ITELINE, Gene, Cell, & RNA Therapy Landscape (2022) (Q4 2022 Quarterly Data Report, American Society of Gene + Cell Therapy).

[7] Source: Search of US and WIPO databases Feb. 2023 (patent and patent application results generated using the keyword query: TAC:(“CRISPR” OR “Gene Editing” OR “guide RNA” OR “Cas*”) AND IPC:(C12N2310/20 OR C12N15/07 OR C12N15/09 OR  C12N15/113 OR C12N15/907 OR C12N15/63 OR  C12N15/902) within the U.S. and WIPO).

[8] Source: Search of US and WIPO databases Feb. 2023 (patent and patent application results generated using the keyword query: TAC:(“CRISPR” OR “Gene Editing” OR “guide RNA” OR “Cas*”) AND IPC:(C12N2310/20 OR C12N15/07 OR C12N15/09 OR  C12N15/113 OR C12N15/907 OR C12N15/63 OR  C12N15/902)).

[9] Source: Search of US and WIPO databases Feb. 2023 (patent and patent application results generated using the keyword query: TAC:(“CRISPR” OR “Gene Editing” OR “guide RNA” OR “Cas*”) AND IPC:(C12N2310/20 OR C12N15/07 OR C12N15/09 OR  C12N15/113 OR C12N15/907 OR C12N15/63 OR  C12N15/902)).

[10] Source: Search of US and WIPO databases Feb. 2023 (patent and patent application results generated using the keyword query: TAC:(“CRISPR” OR “Gene Editing” OR “Cas*”) AND ICLMS:(“guide RNA”) AND IPC:(C12N2310/20 OR C12N15/07 OR C12N15/09 OR  C12N15/113 OR C12N15/907 OR C12N15/63 OR C12N15/902)).

[11] Source: Search of US and WIPO databases Feb. 2023 (patent and patent application results generated using the keyword query: TAC:(“CRISPR” OR “Gene Editing” OR “Cas*” OR “guide RNA”) AND ICLMS:(“lipid*” OR “LNP”)).