Human Gene Editing

Reinventing gene therapies with next-generation technologies

Overview

For decades, medical science has been foraging for permanent cures for a large array of diseases. Great strides have been made in the field of genetic technologies within the last decade, which hold the potential to fundamentally disrupt the human evolutionary process and bring forth unprecedented benefits to the human race. 

Gene editing is an emerging technology that can alter and modify a species' DNA for a specific purpose. In simple words, gene editing is similar to how an author edits or corrects a book or an artist amends an art piece. The technology is not necessarily new; it is already used in agriculture to create genetically modified animals and plants. For humans, gene editing can potentially deliver permanent cures—in potentially one-time treatments—for a myriad of diseases across various therapeutic areas, such as neurology and oncology. 

Human gene editing achieved a key milestone in December 2023. The first gene-editing therapy (“Casgevy” developed by CRISPR Therapeutics) was approved by the FDA to treat sickle cell disease in patients aged 12 and above. The same drug was later approved for patients with beta thalassemia in January 2024. More gene-editing treatments are underway in Phase I and II clinical trials and can be expected to enter the market in the next few years.

Industry Updates

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Market Sizing

The US Human Gene Editing market could reach USD 24.3 billion–44.4 billion by 2030

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Market Mapping


CRISPR technology dominates the human gene-editing space

Most companies in gene editing are focused on the research and development of novel CRISPR/Cas9-based treatments due to the technology’s simplicity, versatility, and cost-effectiveness. Although low in number, a number of drug-developing companies, including several early-stage disruptors, are leveraging other older yet evolving technologies, such as TALEN and ZFN, owing to fewer observed off-target effects and greater efficiency (in certain gene applications such as heterochromatin, the gene responsible for Fragile X syndrome and sickle cell anemia) compared to CRISPR.

On the other hand, gene-editing SaaS platforms remain the smallest segment, with a relatively low number of startups in the space due to most companies choosing to use internally developed gene-editing platforms and technologies at present. However, demand for these platforms could increase as gene editing becomes commercialized, and more pharma and drug developers plan to enter the space quickly, using pre-built gene-editing platforms.

Most companies across the space fall under the seed stage, with only a few startups in the early stage. This reflects the industry’s current nascent stage, with no FDA-approved, commercially available treatments; most companies are either in the preclinical or early clinical trial stages. 

The incumbent involvement in the industry is predominantly focused on the CRISPR/Cas9 segment, which includes prominent Big Pharma companies such as Merck, AstraZeneca, and Bayer. 

The Disruptors


CRISPR-focused startups shine in the gene-editing space with high value and progress

Leading gene-editing startups have raised funds via public listings, with market capitalizations going as high as USD 5 billion. Some companies that have taken this route include CRISPR Therapeutics, Intellia Therapeutics, and Editas Medicine.

Over 60% of human gene-editing disruptors were founded after 2015, following the introduction of CRISPR/Cas9 technology in 2012. Notably, most companies incorporated before 2010 (less than 10%) were observed to be involved in the research and development of gene-editing treatments based on technologies introduced prior to CRISPR, such as TALEN, ZFN, and meganucleases.

A large number of industry disruptors developing gene-editing treatments are involved in multi-year collaborative programs with Big Pharma incumbents; for instance, gene-editing startup CRISPR Therapeutics and pharma company Vertex Pharmaceuticals are developing a CRISPR-based treatment for sickle cell disease and beta thalassemia. 

Funding History

Competitive Analysis


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Incumbents


Incumbents enter gene editing mostly via partnerships

Major Big Pharma companies such as AstraZeneca, Pfizer, Bayer, Eli Lilly, and Bristol-Myers Squibb are involved in the CRISPR/Cas9 drug developers segment owing to the high potential and increasing popularity of the technology.

The most common method incumbents have used to set foot into the gene-editing space has been through partnerships and research collaborations with startup companies. Although the number of incumbents with in-house gene-editing capabilities is low, a few prominent Big Pharma incumbents, such as Novartis, Merck, and AstraZeneca, have initiated in-house R&D initiatives over the last few years to stay at the forefront of gene editing. 

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Notable Investors


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