Thanks to a breakthrough in biotechnology made by two Nobel Prize-winning researchers in 2020, it will soon be possible to treat genetic diseases and cancer through selective gene sharing. The innovative method CRISPR/Cas9 it has also aroused interest among investors, thanks to which both Nobel laureates were able to create independent companies and list them on the stock exchange, allowing us to assess the development of this technology and its promising potential.
A breakthrough in the treatment of genetic diseases
In 2020, Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize in Chemistry for “developing a genome editing method” – CRISPR/Cas9 – whose mechanism was first described in 2012. The aforementioned genetic engineering technique has allowed scientists to manipulate genes. not only from animals and humans, but also from plants, which significantly expands the potential spectrum of the use of this method in commercial terms as well. Both Nobel laureates founded companies called CRISPR Therapeutics and Intellia Therapeutics. Both companies have already produced drugs approved for commercialization by US FDA regulators. The largest companies in the pharmaceutical industry, such as Regeneron or Bayer, co-create CRISPR and take on the challenge of fighting hitherto incurable and deadly diseases . The implementation of foreign DNA in the selected part of the genome makes it possible to “replace” the mutated gene with the correct one. This method is already used in the treatment of monogenetic diseases such as sickle cell anemia, cystic fibrosis or Duchenne muscular dystrophy. Thanks to the CRISPR/Cas9 method, first described 10 years ago, companies in the food industry can modify their crops in a different way than genetically modified organisms (GMOs).
What is CRISPR/Cas9 and how does genome editing work?
CRISPR-Cas9 it was adapted from a natural genome-editing system that bacteria use as an immune defense. Using the Cas9 enzyme, scientists can “cut” specific DNA fragments, e.g. those that contain an unfavorable genetic variant, and replace them with the healthy one. Cas9 follows the guide RNA to the same location in the DNA sequence and makes a cut on both strands of the DNA. Although the cell is able to put these broken pieces of DNA back together, this process is imperfect and leads to numerous changes in the genome and consequently to the deactivation of the gene present at that location. Thanks to this, CRISPR technology makes it possible to incorporate another piece of DNA in the indicated place. This method makes it possible to treat not only diseases caused by the abnormal version of the gene, which in turn lead to the production of a defective protein, but also those caused by the lack of a protein necessary for the proper functioning of the organism.
CRISPR market size and forecasts for the next few years
The global CRISPR technology market was valued at $2.2 billion in 2021. The projected CAGR (average annual growth rate) over the period from 2021 to 2030 is close to 20%. The global market for CRISPR technology is expected to reach $11.6 billion by 2030.
The largest and fastest growing markets
According to forecasts, Asia and the Pacific will be the markets with the most dynamic development in terms of CRISPR technology. Among all regions, North America emerged as the largest market for CRISPR technology, with a share of approximately 34.67% and $2,251.21 million in revenue in 2021. The CRISPR market in the Americas region is anticipated to del Norte will experience significant growth during the forecast period due to the multiple benefits of using CRISPR technology and numerous technological innovations and product approvals, as well as legislation implemented by the government.
Source: US Census Bureau, Illumina, NHGRI 2019
In the 1990s, the cost of sequencing the human genome was high, costing $2.7 billion and taking nearly 13 years. Today, costs are around $700 and the process takes just a few days. Improving statistics creates opportunities to implement new solutions and investigate data along with developing an optimal business model. In 2020, thanks to CRISPR-Cas9 gene editing, a faulty genetic mutation was eliminated in the human body for the first time.
5 interesting companies that use the CRISPR method
CRISPR Therapeutics (CRSP.US)
The company is the world’s leading gene publisher and was founded in 2012 by Emmanuelle Charpentier, winner of the 2020 Nobel Prize in Chemistry, and Rodger Novak. The company has partnered with Bayer and Vertex. The cooperation with Bayer resulted in the creation of a joint venture: Casebia Therapeutics. Bayer committed to finance the company’s operations with $300 million over a period of 5 years. CRISPR Therapeutics owns 50% of Casebia. The company focuses on the treatment of blindness, heart defects, and blood diseases using the Cas-9 method. The company has already implemented the drug CTX001, approved by the FDA (Federal Drug Administration), which is used for the treatment of beta thalassemia and sickle cell disease. To develop the CTX001 drug, the company entered into a cooperation with Vertex, which acquired a 60% stake in the research program and invested $900 million. The estimated value of the program to combat both diseases reached approximately $9 billion. CRISPR Therapeutics appears to have a well diversified and comprehensive model of applied methods thanks to the use of two gene editing methods known as LVP and AAV and LNP technology licensed from the Massachusetts Institute of Technology. The company, together with StrideBio, is developing solutions to improve the AAV method and increase production.
Intellia Therapeutics (NTLA.US)
Intellia was founded by Nessan Bermingham and 2020 Nobel Prize winner Jennifer Doudna. The company has partnered with Novartis and Regeneron. Shareholders include investment funds BlackRock, Vanguard and ArkInvest. The most promising program is NTLA-2001, whose goal is to reduce the concentration of transthyretin amyloidosis. The program has already brought the company nearly $700 million in benefits. Intellia implements it in cooperation with Regeneron. Intellia is working with Novartis to develop NTLA-2002 to combat hereditary subcutaneous edema. The companies are also working on the OTQ 923 project aimed at finding a cure for sickle cell disease. It is estimated that the program will generate between 5,000 and 6,000 million dollars in revenue. Intellia has also partnered with Cellex and Blackstone in collaboration where it focuses on so-called universal allogeneic therapies. Intellia operates in the US market. As the only company in the industry, it has a program to combat acute leukemia, NTLA-5001. The potential value of revenue in the US market alone is estimated at $6-8 billion. Like CRISPR Therapeutics, Intellia uses LNP and AAV methods. Along with Regeneron, the company is also exploring a “hybrid” LNP-AAV delivery method that could gain potential advantage.
Beam Therapeutics (BEAM.US)
The company says its approach offers several advantages over other CRISPR techniques. These include the ability to precisely target a specific gene sequence and edit genes without unintended consequences, such as genome rearrangements on a larger-than-intended scale. Beam is not yet as advanced as CRISPR-focused companies in the industry. The company is in the process of introducing lead candidate BEAM-101 into Phase 1/2 clinical trials to combat sickle cell disease. He also runs preclinical programs using database editing to treat beta thalassemia, leukemia, and various other genetic diseases. The capitalization of the company is at a similar level compared to other CRISPR companies with much more advanced production lines. However, the long-term potential of Beam technology may justify the current high valuation.
Editas Medicine (EDIT.US)
Editas Medicine is a leader in the development of therapies for rare eye diseases based on CRISPR gene editing technology. In September 2021, the company announced preliminary results from the Phase 1/2 BRILLIANCE trial of EDIT-101, used for the treatment of Leber’s congenital amaurosis 10 (LCA10). Investors were disappointed with these data. However, it is possible that further testing of higher doses of the CRISPR-based therapy could improve outcomes in the future. Editas also has another candidate in the clinical phase, EDIT-301. Like the leading Beam and CRISPR Therapeutics programs, EDIT-301 focuses on sickle cell disease. However, Editas believes that its approach of editing the HBG1 and HBG2 genes could provide an advantage over rivals that edit the BCL11A gene. The company hopes to bring EDIT-301 to clinical trials as a treatment for transfusion-dependent beta-thalassemia. In recent years, Editas has generally been seen as one of the leading producers of CRISPR-focused drugs. However, EDIT-101’s mixed preliminary results left Editas with a much smaller market capitalization than other early winners, including CRISPR Therapeutics and Intellia Therapeutics.
Sangamo Therapeutics (SGMO.US)
Sangamo was founded in 1995 as Sangamo BioSciences, Inc. and focused on researching new genome editing technologies. The company partnered with Pfizer to develop treatments for hemophilia A and ALS. Sangamo Therapeutics is the only company focused solely on the development of Zinc Finger Nucleases (ZFNs) as a genetic engineering tool. The company uses the tool in both ex vivo and in vivo gene editing therapies, as well as a genome regulation tool. The company is currently sponsoring clinical trials in sickle cell disease and beta thalassemia using ZFNs for gene editing. The company has established partnerships with many leading companies, including Pfizer, Biogen, Novartis, Takeda, Sanofi, and Kite Pharma.
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