CRISPR Cas9 technology is arguably one of the greatest breakthroughs in modern medicine. It became popular in the scientific community for being quicker, cheaper, more accurate, and efficient than other genetic engineering methods. As of June 2020, more than USD 3.2 Bn has been invested in CRISPR startups, of which many have gone public over the last five years.
What is CRISPR?
CRISPR Cas9, shorthand for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9, is a gene-editing technology that allows scientists to make precise changes to DNA in a lab. It can work on any organism, including humans, to bring about desired outcomes through targeted gene alteration. This revolutionary technology is finding applications in biological research, agriculture, and developing treatments for human genetic diseases.
Here’s a deep-dive into the state of the CRISPR startup ecosystem, including the key application areas, investments by pharma companies, and top-funded startups.
CRISPR startups by application
The CRISPR startup ecosystem can be broadly classified into four distinct groups: biomedical R&D, agricultural biotech, industrial biotech, and biological R&D.
This field uses CRISPR to focus on targeted gene therapies for human diseases. It includes researching cures for diseases like cancer, genetic blindness, AIDS, cystic fibrosis, muscular dystrophy, and Huntington’s disease.
Examples of CRISPR startups in this space include Mammoth Biosciences, which has created the first CRISPR-mediated platform for human disease detection. Caseiba Therapeutics is another startup in this category that’s focused on finding treatments for inherited diseases like hemophilia.
Startups in this category use CRISPR to make gene-edited crops which have characteristics superior to natural ones. This helps decrease pesticide use and pollution and expedites food production. Agro-biotech startup Plantedit is a pioneer in this field, with a focus on producing cost-effective and modified crop species that can withstand infection, climate change, and other types of stress.
Some startups in this category are also using CRISPR for breeding genetically-altered and disease-resistant livestock, promising better and more organic animal products. For example, Caribou Biosciences is collaborating with animal genetics company Genus in developing Porcine Reproductive and Respiratory Syndrome virus (PRRSv) resistant pigs, using this technology.
These startups use CRISPR for sustainable processing and production of chemical products, raw materials, and fuels. Innovation in this field has led to the development of changeable gene-editing applications, which allows its users to customize the tool for their specific needs, with Inscripta being a key leader.
In the industrial sector, CRISPR is also being explored for producing chemicals from natural enzymes — a cheaper, more energy-efficient, and environment-friendly alternative. Biotech startup Solugen has used this method to make hydrogen peroxide, which was used in their new line of biodegradable cleaning wipes.
Startups in this field are testing CRISPR for developing techniques like animal-to-human organ transplants, building synthetic DNA molecules, and developing antibiotic compounds for modern medicine. Oxford Genetics’ brand OxGene is using CRISPR for cell engineering in mammals and developing antibody-based therapies.
CRISPR startup funding trends
With the potential to cure major diseases and create designer babies, the disruptive power of CRISPR technology is second to none. So it’s little surprise that several organizations want to be a part of this revolutionary technology. Large corporations, including pharma behemoths such as Novartis and Bayer, are driving investments in CRISPR-based therapeutics, cell therapies, disease diagnostics, and more.
Biomedical R&D-based CRISPR startups, particularly the ones focused on therapeutics, have received the maximum of funding over the years.
According to a study by Netscribes, the funding for CRISPR startups has grown by 69% CAGR over the last four years from 2016-2019.
2019 was a great year for CRISPR startups in terms of fundings received, with Poseida Therapeutics being at the forefront of that list (through series C funding of USD 142 mn). Beam Therapeutics was a close second with its series B funding of USD 135 mn, and Inscripta came third with its series D funding of USD 125 mn.
Some of the largest investors in this field include Arch Venture Partners, Fidelity Management & Research Company, GV (formerly Google Ventures), and Alexandria Venture Investments.
In the last five years, some of the most prominent start-ups have become public, including the following:
Partnerships and M&A
Many pharma giants have jumped on the CRISPR bandwagon in their race in recent years. Bayer and CRISPR Therapeutics joined hands in 2016 and founded their new gene-editing venture Caseiba Therapeutics. Vertex Pharmaceuticals acquired Exonics Therapeutics in 2019 for USD 245 mn. In the same year, the parent company of Juno Therapeutics, Celgene, was acquired by Bristol-Myers Squibb.
Other leading pharma investors in this field include AstraZeneca (AZ), Novartis, GlaxoSmithKline (GSK), AbbVie, Eli Lilly, Pfizer, Roche, and Johnson & Johnson.
Top-funded CRISPR startups
Specialization: CRISPR-associated natural and synthetic nucleases
Main projects: They have an enzyme engineering program that allows their customers to create their own customized gene-editing applications. Inscripta has also created a family of CRISPR enzymes (MADzymes) which have innovative features, said to be able to increase the speed and efficiency of precision genome editing.
2. Inari Agriculture
Main projects: They’re focusing on using CRISPR to manage specific gene expressions in plants. They are also developing customized seeds that significantly reduce the land, water, and other natural resources required to produce food and feed.
Specialization: Genetic engineering solutions that bring precision and automation to genome editing
Main projects: They have come up with a CRISPR Design Tool which uses several built-in algorithms to instantly identify guide sequences targeting a gene, and simplifies gRNA design. They’re also developing a Gene knockout kit v2 that’s designed to guarantee a gene knockout, saving scientists from trial and error cycles in their CRISPR experiments.
4. KSQ Therapeutics
Specialization: Transformative medicines
Main projects: They have developed vitro cell-autonomous screens and more complex genome-scale screens of immune cells in vivo. They have developed an industrialized discovery engine called CRISPRomics which can generate disease-specific insights for every human gene, and also identify and genetically validate optimal novel targets for drug discovery.
Specialization: CRISPR-based Xenotransplantation
Main projects: They use rapid and automated DNA sequencing to perform microbial fingerprinting. They have been awarded the first US Patent for their rapid DNA sequencing platform to control hospital-acquired infections. They have partnered with Quihan Bio (China) in using CRISPR to create the most extensively genetically engineered pigs, whose tissues have all the features necessary for being transplanted into humans.
6. Verve Therapeutics
Specialization: Cardiovascular and coronary artery diseases
Main projects: They are targeting the PCSK9 gene whose disruption through variants can cause lower levels of cholesterol and resistance to heart attacks. They aim at developing a one-time gene therapy to confer lifelong resistance to coronary artery disease.
7. Tango Therapeutics
Specialization: Novel medicines targeting cancer vulnerabilities
Main projects: Using the principle of synthetic lethality, the company is developing medicines that target specific tumors. They have built a target discovery platform that uses CRISPR to find vulnerabilities in specific cancers. Tango entered a collaboration with Gilead Sciences in 2018, to discover, develop and commercialize a pipeline of innovative targeted immuno-oncology therapies.
The way forward
CRISPR-Cas9 gene editing is continuing to improve, which makes for a very promising future ahead for investment opportunities. Researchers are currently looking into how it can be used to help fight COVID-19, and similar new viral strains that have the potential to reach the level of a pandemic. It may someday lead to standardized genetic cures and even help eradicate certain diseases completely.
In July 2020, CRISPR pioneer Jennifer Doudna’s research team at UC Berkeley identified a new CRISPR enzyme which can lead to further developments in this field. Some geneticists, like George Church from Harvard, are also currently using CRISPR to create hybrid embryos of extinct animal species such as the Woolly Mammoth.
CRISPR is beginning to create a major impact on various fields of biotechnology, which is sure to give rise to numerous opportunities across industries. For a deeper analysis of the CRISPR market landscape and potential growth opportunities, contact us.