We mainly focus on the application of gene editing in medicine, so its market space is closely related to the diseases it can use and the number of patients who use it.
From the perspective of the types of diseases, gene editing can theoretically cause any related diseases caused by genetic variations.
According to the official website of the American Organization for Rare Diseases, there are currently more than 7,000 known rare diseases in the world, 80% of which are genetically related, and there are about 300 million patients with rare diseases, of which 50% are children.
More importantly, in terms of medical value, the unmet needs of rare disease patients are significant, with less than 5% of the world's known rare diseases** being met.
In addition, in addition to rare diseases, conventional diseases are also the scope of gene editing.
Therefore, from the perspective of the most advanced disease types, the huge unmet medical needs of patients with rare diseases provide a huge market space for gene editing.
From the point of view, the cost of gene editing is still high, taking the newly approved SCD genes ** Cavevy and Lyfgenia as examples, the former is priced at $2.2 million, and the latter is priced at $3.1 million.
Such a high ** is bound to keep most patients out, and it will be very important to reduce costs, reduce prices, and improve the penetration rate of gene editing ** in the future.
However, judging from the cost and trend of new technologies in the past, with the maturity of technology, it is expected that gene editing will decline in the future, so as to benefit more patients.
Therefore, as an emerging technology, with the decline of ** and the increase of indications, the gene editing market has broad prospects and will surely experience a stage of rapid growth.
According to the statistics of Allied Market Research and Global Market Research, from 2021 to 2022, the market size of the global gene editing industry increased from 48$1.1 billion to $541.2 billion US dollars, with a year-on-year growth rate of 1249%。
It is estimated that by 2030, the global gene editing market will reach 360$6.1 billion, with a CAGR of 223%, the industry growth rate is fast.
In addition, Cathie Wood, the sister of wood, has also carried out **, the annual revenue of gene editing will reach 30 billion US dollars in 2030, and if it can be expanded to **type 1 diabetes, it may expand to 60 billion US dollars, and the upper limit of the market space will be doubled.
From the perspective of market segments, CRISPR technology has dominated with its faster, cheaper, and more accurate characteristics, and this position is expected to continue in the future.
Geographically, the North American market is the world's largest market, accounting for 329%, followed by Europe and Asia-Pacific.
The core competitiveness in the gene editing industry is mainly highlighted in the number of underlying patents, which is essentially the competition for technological development.
Throughout the world, the regions with more developed gene editing technology are mainly North America and Europe, and the world's largest gene editing companies are concentrated in the above regions.
There are no gene-editing listed companies in China, and A-share concept stocks such as Biocytogen and Shanghai Model Biology are mainly engaged in animal model-related business, but there are many companies in the primary market that are actively deploying in this field, such as EdiGene, BRL Medicine, Yaotang Biotechnology, Yijie Like, etc.
We have listed the relevant data of the major gene-editing listed companies in the U.S. stock market as follows:
1. CRISPR Therapeutics, with Carpentier as the co-founder, is headquartered in Basel, Switzerland, and conducts R&D business in Cambridge, Massachusetts, USA.
The company is committed to gene silencing, knock-in and knock-out through CRISPR Cas9 technology to achieve the goal of disease **, and currently focuses on drug development in four major fields: hemoglobin, immuno-oncology, regenerative medicine and in vivo.
The recent FDA-approved product Casgevy came from the company, becoming the first company to have CRISPR gene editing** on the market.
2. Beam Therapeutics, by Feng Zhang, D**id Liu, and JCo-founded by Keith Joung, a pioneer in the field of BE and the first company to develop a new ** using CRISPR single-base editing technology.
BEAM's base editing platform combines the characteristics of CRISPR systems that target specific DNA sequences with base-editing enzymes that chemically modify DNA bases, enabling precise editing of individual bases.
3. Intellia Therapeutics, as the co-founder, Dudna completed the listing within two years after its establishment, mainly committed to the development of CRISPR Cas9 technology in the most advanced fields, such as leukemia, cancer, etc.
The company's main product pipeline is divided into in vivo and in vitro.
4. Verve Therapeutics, founded by renowned cardiologist Sekar Kathiresan and others, is committed to developing a method for cardiovascular disease (CVD) that will transform from chronic management to a single-course gene-editing drug.
The company's first two programs targeted the genes PCSK9 and AngPTL3, which regulate blood lipid levels, respectively, and the lead product candidate, Verve-101, is the world's first to enter the clinic and perform base editing in the human body**.
5. Editas Medicine, founded by Zhang Feng, is a company based on CRISPR Cas9 to develop gene editing for genetic diseases and cancer**, and is also the first listed CRISPR gene editing company.
Regarding Editas, there is also a patent dispute that has to be mentioned since the birth of CRISPR Cas9 in 2012. The controversy is mainly between two parties: Carpentier and Doudna (the CVC team) on one side, and the Broad Institute where Zhang Feng works, on the other.
In February 2022, the U.S. Patent and Trademark Office determined that the Broad Institute team was the first team to invent CRISPR Cas9 technology to edit human cells and use it to make drugs, and therefore has a patent for the use of CRISPR Cas9 gene editing technology in eukaryotic cells.
Since Editas has received an exclusive license from the Broad Research Institute team, this means that companies using CRISPR Cas9 gene editing technology will need to pay royalties to them to obtain a license to use it.
However, the patent battle between the two sides has not really settled, and after the decision of the US Patent and Trademark Office, the CVC team appealed to the US Court of Appeals for the Federal Circuit, which is reported to make a decision after mid-2024.
In addition, Editas is also developing the CRISPR genes for sickle cell disease and thalassemia, Reni-Cel, which differs from the approved Casgevy in two main ways. One is that the gene editing tools used are different, and the other is that the target is different.
6. Caribou Biosciences, co-founded by Doudna and dedicated to using CRISPR technology to develop transformative ** for patients suffering from life-threatening diseases.
The company's genome-editing platform, including its patented Cas12A chrDNA technology, enables the development of cells** with greater precision and enhanced persistence.
In addition, in contrast to other companies, in addition to medicine, CARIBOU is also committed to translational research of gene editing technologies in the fields of agriculture, biology and industrial biology.
7. Metagenomi, co-founded by UC Berkeley metagenomics scientists Brian Thomas and Jillian Banfield, is dedicated to developing the best methods for patients using a proprietary, comprehensive metagenomics-derived genome editing toolbox.
Metagenomi intends to use artificial intelligence to find new gene editing systems in nature to produce the next breakthrough similar to CRISPR that can be used in gene medicine development.
To this end, the company has developed a modular and diverse toolbox to mine microbial genomes through the application of machine learning to find new nucleases that can be used for gene editing.
Using high-throughput screening, artificial intelligence, and proprietary algorithms, it has rapidly mined billions of novel proteins from genome-elluding metagenomic databases to create genome editing tools.
8. Sangamo Therapeutics, formerly known as Sangamo Biosciences, Inc., uses the first generation of gene editing system ZFN instead of CRISPR technology.
As the leader of ZFN, Sangamo achieved a monopoly in the early stage of the development of the industry with its ZFN technology patents, but the closure caused by the monopoly also led to the stagnation of ZFN technology development.
Since then, with the rise of Talen and CRISPR, the company's competitiveness in the field of gene editing has begun to decline, but the company still has high capabilities in the field of cells and genes.
9. Cellectis, headquartered in Paris, France, is currently a leading company in the development of universal CAR-T (UCAR-T) products, focusing on the development of allogeneic CAR-T cell immunity based on Talen gene editing**.
In other words, its core goal is to develop UCAR-T**, and the use of the gene-editing tool Taren is to better achieve this goal.
10. Precision Biosciences is a clinical-stage biological company that uses its Arcus genome editing platform to develop allogeneic CAR-T** and in vivo gene editing**.
In other words, one of its goals is to develop ucar-t**, but it uses a special editing platform, arcus.
Arcus is based on a naturally occurring genome editing enzyme, i-Cre, which evolved in the algae Chlamydomonas and allows for highly specific cleavage and DNA insertion in cellular DNA.
i-CREL is a type of meganuclease (homing endonuclease or large nuclease), which is the earliest gene editing tool.
Among the gene editing companies, Sangamo achieved a monopoly in the early stage of the industry with its ZFN technology, but with the rise of Talen and CRISPR technology, the company's competitiveness has declined.
Secondly, CRISPR Therapeutics, BEAM, Editas, etc., have taken advantage of the rapid development of CRISPR technology and developed rapidly.
The vast majority of the world's leading gene editing companies use CRISPR technology, and the closer they are to the present, the more companies are adopting CRISPR technology.
For example, Sourdive of Cellectis believes that when a large number of genes are cut or modified, CRISPR will lead to the excision of non-target sites, resulting in off-target phenomena.
As a result, CRISPR is less efficient than Talen in large-scale gene editing applications. TALEN's high accuracy is even more advantageous in industrial production.
If CRISPR technology is used in production, each cell needs to be examined to confirm that it is off-target. This is an extremely complex and tedious task. Therefore, CRISPR is currently more suitable for pre-screening than industrial production.
Now, we have a basic understanding of the principles, tools, industries, and companies of gene editing.
We can intuitively feel that it is an exciting technological progress in biotechnology, so how to implement it into investment? Here is my immature opinion for your reference:
1. We need to keep in mind that gene editing is an investment track that can be followed for a long time, with a large market space, fast growth rate, and one of the rigid needs of human health, which is worthy of long-term follow-up research.
2. On the other hand, we must also keep in mind that it is still in the early stage of growth and faces a lot of uncertainties, such as the impact of regulations, ethics, security, technical route selection, etc., which should be reflected in our control.
3. The judgment of the technical route is a very important thing, which is worthy of our attention and tracking, and it will also be reflected in our allocation ratio of different technical route companies.
4. Similarly, we can see that most gene editing companies are still not profitable, which makes it difficult for us to value companies in the industry, and it is difficult to grasp the first point in time.
I think we can use the stock price on December 8, 2023, the day the FDA approved the first gene edit**, as a starting point, after all, this is a sign that the ideal is shining into reality.
If the company's stock price is lower than the stock price on the approval date due to other reasons in the case of little change in fundamentals, it can be focused on, and it can be used as a time to open a position if there is a sufficient margin of safety.
5. When to consider selling, I think you can make a reasonable valuation of the company based on the industry size and the company's market share, and once the stock price is irrational, so that it deviates significantly from the reasonable valuation, you can consider selling.
Reminder: This article is only a record of the author's thoughts, for readers' reference only, and is not intended as any investment advice; Investment is risky, and you should be cautious when entering the market.