Top 5 CRISPR Companies To Invest In (May 2024)

The CRISPR Revolution

Hundreds and even thousands of diseases have a root cause in genetics. Many of these are, to this day, untreatable or poorly treated. And most are either heavily incapacitating or deadly.

While some gene modification technology has been around since the 1990s, they were somewhat crude, causing much damage and unwanted modification to the modified cells. This was good enough for making GMO corn but not enough for human medicine.

So when, in 2012, Jennifer Doudna and Emmanuelle Charpentier discovered CRISPR-Cas9, a lot of hopes grew. This is because the CRISPR system allows us to “edit” genes in a targeted fashion that was impossible until then.

Source: CRISPR Therapeutics

This discovery led both women to launch their own companies separately, looking to investigate and monetize this invention and winning the Chemistry Nobel Prize in 2020.

A dozen of CRISPR therapies are now being tested and running clinical trials for previously uncurable diseases.

The CRISPR Mania

Because of its massive potential, CRISPR became immediately the center of a massive R&D effort in the biotech industry. New CRISPR systems got discovered, like Cas12, CAs12a, but also Cas13, Cas 5, Cas8, Csx10, etc…

For now, most of the research efforts for human medicine are concentrated on CAs9 and CAs12. If you are technically minded and want to learn more about the difference between these two main CRISPR systems, I recommend reading this scientific publication and this article.

This massive investment in CRISPR also triggered new companies dedicated to the topic and new program launches by other biotech and pharmaceutical companies. This creates a bit of mania when combined with the rush for anything biotech-related during the pandemic. Since 2021, this has somewhat cooled off, with valuations stabilizing lower.

Lately, many of these therapies have finished the last stage of clinical trials or are even already approved. So, this is a good time for investors to look back at the sector and see which are the best stocks to ride the incoming wave of commercialization of CRISPR-based therapies.

Top 5 CRISPR Stocks

(The selection has been made according to a subjective evaluation of technical achievements and financial profiles).

1. CRISPR Therapeutics AG

CRISPR Therapeutics was founded by CRISPR Cas9 co-discoverer Emmanuel Charpentier. The company focuses on applying to human medicine the Cas9 system.

In its R&D pipeline, the 2 flagship programs are blood therapies for  Beta-thalassemia and sickle cell diseases (SCD). Both are developed in partnership with Vertex.

Another application of CRISPR Therapeutics' technology is cancer treatment. The idea is to use modified immune system cells to attack cancer cells. Until now, cells from the patient had to be genetically modified, a process taking several weeks, which often can be too late for a patient's quickly degrading health.

Instead, CRISPR Therapeutics is developing a modified immune system cell that can be manufactured in advance and fit all patients. The company currently has 8 candidates in the pipeline, of which 2 already in clinical trials.

The company is also in a partnership on a diabetes treatment with ViaCyte. CRISPR Therapeutics wants to improve the technology by turning it into a lifelong cure and not requiring immunosuppression drugs for life like the initial version of ViaCyte treatment requires.

ViaCyte was acquired by Vertex in the Summer of 2022, deepening the ties between Vertex and CRISPR Therapeutics.

In the long run, CRISPR Therapeutics aims to develop in vivo therapies, where the cells can be modified directly in the patient's body, instead of the current ex-vivo approach, where modified cells are re-injected into the patient.

Source: CRISPR Therapeutics

While CRISPR Therapeutics is still in the pre-revenue stage, it made much progress in its R&D pipeline. It also has $2B in cash to cover expenses until 2024. So, it might still need to raise money if its blood therapies are not approved by 2024. This might also be avoided thanks to revenues from Vertex for hitting R&D milestones in the diabetes program.

2. Vertex Pharmaceuticals Incorporated

Vertex is the leader in Cystic Fibrosis treatment, a deadly genetic disease, with 4 different treatments targeting different patient profiles. Patients who cannot be treated with the current therapy have a drug in phase III of clinical trials, Vanzacaftor. They are also developing gene therapy for cystic fibrosis using mRNA technology.

As a whole, Vertex is very R&D-focused, with 70% of operating expenses dedicated to finding new drugs and therapies.

Source: Vertex

They partner with CRISPR Therapeutics to help develop the blood disease gene therapy mentioned above.  They also have 2 different programs in development for Type-1 Diabetes. The first is the one we described in more detail in the CRISPR Therapeutic program. The second is a device that would isolate the insulin-producing cells from the immune system, removing the need for ViaCyte hypo-immune technology.  You can read more about this diabetes treatment technology in this detailed article.

Thanks to this dual approach, Vertex has a serious chance of turning diabetes into a fully curable disease and maybe even without immunosuppressants.

Lastly, their VX-548 treatment for acute pain is the last stage of development and might be a new alternative to opioids, without the risk of addiction and fewer side effects.

Source: Vertex

Vertex can rely on its stable income stream from its leading position in cystic fibrosis to finance all of its expansion into new therapeutic fields. This makes it a good CRISPR stock for investors wary of pre-revenue companies.

3. Ginkgo Bioworks Holdings, Inc.

The company is producing on-demand organisms for specific applications. It has diversified its applications widely with many research programs and partnerships:

• Cannabinoids
• mRNA vaccine production and nucleic acid medicine
• Food proteins
• Biological fertilizer production in partnership with Bayer
• Programmable microbes for gut diseases
• Microplastics bioremediation
• Biosecurity and pathogens detection
• Recycling waste and contaminants

Many of these modifications rely on CRISPR or similar gene editing technologies, notably its CAR-T cancer cell therapies.

By providing a ready platform for cell engineering, Ginkgo is becoming a key service provider in the biotech industry, going beyond the pharmaceutical industry and into agriculture, biosecurity, and industrial chemical processes. It provides expertise and speed and can help reduce fixed costs and the quantity of capex needed for a research project.

This is demonstrated by the very diverse array of clients and partners the company had over the last few years.

Source: Gingko Bioworks

The company is not profitable yet, as it is investing a little more than its total current revenues ($81M in Q1 2023) in R&D. The company nevertheless had a relatively comfortable $1.2B in cash in Q1 2023. With a decrease in net cash of $110M in the same period, Ginkgo seems capitalized enough to keep operating and grow its business for the foreseeable future.

This makes it an attractive stock for investors looking to bet on gene editing and cell engineering technologies, but not one application in particular. This is also typically more interesting for growth-focused investors.

4. Beam Therapeutics Inc.

The company was founded in 2017, focusing on developing the technology of “base editing.” This promises more precise gene editing than traditional CRISPR-Cas9 technology. It could also edit multiple spots in a gene or multiple genes at once.

“Many existing gene editing approaches are like ‘scissors’ that cut the genome. Base editors are like ‘pencils’ that enable erasing and rewriting one letter of the genome at a time.” – Giuseppe Ciaramelle, President & CSO at Beam Therapeutics

Beam Therapeutics is at an earlier stage than other CRISPR companies, with its manufacturing facilities expected to start only in late 2023. Most of its pipeline is still at the research stage of entering phase 1/2 of clinical trials.

It has pretty much the same focuses as CRISPR therapeutics: hematology (sickle cell disease), oncology, and rarer genetic diseases (impaired glycogen metabolism and Alpha-1 Anti-trypsin Deficiency – AATD).

Source: Beam Therapeutics

Beam Therapeutics is overall a harder-to-predict company, as it is at a rather early stage. Its technology must prove it can achieve better therapeutic outcomes than its competitors, which will reach the market before. Due to its early stage, it is also likely to need to raise more capital before reaching any FDA approval and commercialization.

5. Editas Medicine, Inc.

Editas was founded by CRISPR-Cas9 discoverer Jennifer Doudna. Editas started working with Cas9 but is now focused on a proprietary version of Cas12 that they engineered: AsCas12a.

You can read more about Cas12a's unique properties in our dedicated article “What Is CRISPR-Cas12a2? & Why Does It Matter?”.

To resume it shortly, Cas12as's uniqueness is because:

• Hard-to-solve problems with Cas9 could be workable with Cas12a
• It results in higher chances of gene editing happening than with Cas9.
• More than one gene can be modified at once with CAs12a.

You can also read an overview of all of Jennifer Doudna's companies in the corresponding article “Top Jennifer Doudna Companies to Watch.”

Editas is focused on Sickle Cell Disease (SCD), with 40 patients in a clinical trial at phase 1/2, with the first results expected by the end of 2023.

Editas is behind CRISPR Therapeutics in clinical trials for sickle Cell Disease but ahead of Beam Therapeutics.

As this therapy and innovative treatments for this disease are seemingly becoming a little crowded, investors will want to look in-depth at the clinical trial results and assess if AsCas12a is producing superior results to “classic” CRISPR Therapeutics Cas9 technology, which is likely to be approved by the FDA first.