Who are the leading innovators in gene splicing using nucleases for the pharmaceutical industry?

MEEFRO February 16, 2023

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The pharmaceutical industry continues to be a hotbed of innovation, with activity driven by the evolution of new treatment paradigms, and the gravity of unmet needs, as well as the growing importance of technologies such as pharmacogenomics, digital therapeutics, and artificial intelligence. In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases.

However, not all innovations are equal and nor do they follow a constant upward trend. Instead, their evolution takes the form of an S-shaped curve that reflects their typical lifecycle from early emergence to accelerating adoption, before finally stabilising and reaching maturity.

Identifying where a particular innovation is on this journey, especially those that are in the emerging and accelerating stages, is essential for understanding their current level of adoption and the likely future trajectory and impact they will have.

110 innovations will shape the pharmaceutical industry

According to GlobalData’s Technology Foresights, which plots the S-curve for the pharmaceutical industry using innovation intensity models built on over 756,000 patents, there are 110 innovation areas that will shape the future of the industry.

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. Adeno-associated virus vectors, alcohol dehydrogenase compositions, and antibody serum stabilisers are some of the accelerating innovation areas, where adoption has been steadily increasing. Among maturing innovation areas are anti-influenza antibody compositions and anti-interleukin-1, which are now well established in the industry.

Innovation S-curve for the pharmaceutical industry

Gene splicing using nucleases is a key innovation area in the pharmaceutical industry

Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. Nucleases are enzymes that hydrolytically cleave the phosphodiester backbone of DNA. As part of the DNA repair process, nucleases play an important role in DNA replication, nucleotide excision repair, mismatch repair, and double strand break repair. They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins. These include Zinc Finger Nucleases (ZFN), Transcription Activator-like Effector Nucleases (TALEN) and Clustered Regularly Interspaced Short Palindromic Repeat nucleases (CRISPR). Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.

GlobalData’s analysis also uncovers the companies at the forefront of each innovation area and assesses the potential reach and impact of their patenting activity across different applications and geographies. According to GlobalData, there are 190+ companies, spanning technology vendors, established pharmaceutical companies, and up-and-coming start-ups engaged in the development and application of gene-splicing using nucleases.

Key players in gene splicing using nucleases – a disruptive innovation in the pharmaceutical industry

‘Application diversity’ measures the number of different applications identified for each relevant patent and broadly splits companies into either ‘niche’ or ‘diversified’ innovators.

‘Geographic reach’ refers to the number of different countries each relevant patent is registered in and reflects the breadth of geographic application intended, ranging from ‘global’ to ‘local’.

Sangamo Therapeutics is the leading patent filer in gene splicing using nucleases. Sangamo Therapeutics has a market cap of $560m and is primarily focused on developing genomic medicines in rare disease, neurology, oncology and autoimmune. The company’s proprietary zinc finger nuclease (ZFN) in vivo genome editing approach is being evaluated in multiple clinical trials to treat haemophilia B and lysosomal storage disorders MPS I and MPS II. S E. Regeneron Pharmaceuticals and CRISPR Therapeutics are the other key patent filers in gene splicing using nucleases.

In terms of application diversity, Takeda Pharmaceutical is the top company, followed by Factor Bioscience and Puretech Health. By means of geographic reach, Reflection Biotechnologies holds the top position, while Curevac and Ipsen stand in second and third positions, respectively.

To further understand the key themes and technologies disrupting the pharmaceutical industry, access GlobalData’s latest thematic research report on Pharmaceutical.