The Problem Of Genetic Diseases
In the 70 years since the discovery of the DNA double helix, we started t truly understand the cause of many mysterious incurable diseases, like hemophilia, cystic fibrosis, etc.
Most of these come from defective genetic sequences: most of the time, a gene has the wrong sequence and produces a non-functional or dysfunctional protein. This error then goes on a make important organs’ functions fail.
The issue has long been to solve such diseases. As the issue is at the genomics level, it means every single cell of the patient is equally defective. And it is not doable to bring the “right” protein in every single cell that needs it.
This was until gene editing became a reality. With new groundbreaking treatments approved in the last few months, it is time to look at the potential of this market.
Gene Editing
Gene editing has been looked after by researchers for almost 20 years now. Early technologies, like zinc-finger nuclease and TALENS, looked promising but were often not sufficient to produce therapeutic results except for a few narrow cases.
The turning point was the 2012 discovery of CRISPR-Cas9. It allowed for a very targeted approach to gene editing. It also earned its discoverers the Nobel Price of Chemistry in 2020.
A few other different CRISPR-Cas systems have since been discovered. Most notably, the CRISPR-Cas12, which we discussed in the article: “What Is CRISPR-Cas12a2? & Why Does It Matter?” You can also read more about gene editing technology in general in this article.
Now a few 10 years later, we are seeing new therapies coming out of CRISPR technology. And some even more advanced gene editing techniques as well.
The Gene Editing Market
Only in a nascent market, the approval of a hemophilia gene editing therapy in November 2022 has demonstrated that gene editing can be both medically powerful and financially viable.
With a price tag of $3.5M/therapy, it broke new records of approved price, previously held by another gene therapy, Novartis’ Zolgensma ($2M/therapy).
The cost might seem outrageous, but it should be compared to the real-life cost of treating these diseases. For example, hemophilia can cost up to $28M, and still not fully allow for a good quality of life.
Source: ARK Invest
With almost 400 clinical trials for gene therapies ongoing, this is a market ready to grow very quickly. It could multiply by more than x10 in the next 7 years.
Source: ARK Invest
Ark Invest projection is a base case of annual revenues of $30B.
This is in the case gene therapies stay confined to rare diseases and genetic problems for the entirety of this decade.
Source: ARK Invest
A Larger Potential
Another possibility is for gene editing to be applied to a much larger pool of diseases. Rare genetic diseases were the obvious test subject for the technology, as we knew what needed to be fixed, existing treatments were poor to non-existent, and even expensive gene editing is still cheaper than most conventional therapies.
But there is a wider range of possibilities. A lot of ongoing clinical trials are in new therapeutic areas, like neurology, oncology (cancer), or diabetes.
And while rare diseases often affect a few thousands or tens of thousands of people, these new applications concern tens of millions of people.
Source: ARK Invest
If some of these therapies demonstrate the efficacy of gene editing for more than just rare diseases, Ark Invest estimated the potential market could be a lot larger. So it could reach annual revenues of up to $60B by 2030. And might keep up growing after that date as well.
Gene Editing Companies
These companies are all working on gene editing technology, including beyond the scope of rare diseases. They have been organized from the largest to the smallest market capitalization at the time of writing of this article
Vertex (VRTX)
Vertex is a company mostly active in the treatment of cystic fibrosis, at least when it comes to its approved portfolio of drugs. It is investing in the potential of genetic therapies for the most difficult case of cystic fibrosis.
But its main involvement in gene editing is tow-fold.
First with therapies developed with CRISPR Therapeutics for blood disease: beta-thalassemia and sickle cell disease.
Secondly, with the acquisition of ViaCyte. ViaCyte is developing, in partnership with CRISPR Therapeutics, a solution for type-1 diabetes. This treatment would be a mix of stem cell therapy and gene editing therapy. You can read more about this diabetes treatment technology in this detailed article.
CRISPR Therapeutics (CRSP)
One of the founders of CRISPR Therapeutics is Emmanuel Charpentier, the discoverer of CRISPR–Cas9 and the Nobel Prize of Chemistry in 2020 for that discovery.
CRISPR is working on multiple thematic, including blood diseases, oncology, and diabetes (the diabetes treatment is developed in collaboration with Vertex)
You can read more about CRISPR Therapeutic in our article “Gene Editing: CRISPR Therapeutics vs. Beam Therapeutics”.
Beam Therapeutics (BEAM)
Another quickly progressing gene editing startup is Beam Therapeutics. It uses a variant of gene editing called base editing, able to modify only one letter at a time in the genetic code. Its main focus is on hematology (sickle cell disease) and then on oncology and rare diseases.
You can follow the link above for a point-by-point comparison between Beam and CRISPR Therapeutics.
UniQure (QURE)
UniCure is the company of which we mention the $3.5M/dose hemophilia gene therapy that has been recently approved in the USA and the EU.
The company is not using a CRIPR-like technology, preferring to it Adeno-Associated Viral Vectors (AAVV).
The company hopes that the recent success in hemophilia can be expanded to other pathologies, notably Huntington’s disease.
Editas (EDIT)
Editas is dedicated to leveraging the capacity of the CRISPR-Cas12a system. We have covered in detail the unique capacities of Cas-12a in a dedicated article. But to resume it shortly:
- 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
The company is only at the early stages of clinical trials, with a focus on eye and blood diseases.
You can see a more detailed profile and a point-by-point comparison to CRISPR Therapeutics in this article: “CRISPR Therapeutics vs Editas Medicine”.
Sangamo Therapeutics (SGMO)
The company is working on multiple stem cell therapies, as well as an hemophilia A gene therapy, now in phase 3 of clinical trials, with an application for approval expected by the end of 2024.
It is also working on a gene editing therapy for Fabry’s disease, a condition affecting the liver.
You can read more about Sangamo (as well as Vertex) in our article “5 Best Stem Cell Companies to Invest In (April 2023)”.
Precigen (PGEN)
The company is leading the field of utilizing gene editing for cancer treatment.
Its UltraCAR-T platform is modifying the patient’s own white cells’ genetics, making them attack cancer cells in a targeted fashion.
Most of the CAR-T treatments are still in phase 1 of clinical trials, with other non-gene editing-focused products more advanced in the R&D pipeline.
Beyond Human Therapeutics
Gene editing is a technology still in its infancy. It has the potential to revolutionize the way we treat living organisms.
For example, it can be used in agriculture to:
- create new varieties and breeds.
- reduce the need.
- increase yield.
You can read more about it and the companies active in that field in our article “CRISPR Beyond Human Health: The New Investing Frontier for Gene Editing“.
It could even turn plants and microorganisms into biofactories producing medicine or high-value material and molecules. Such a concept is often described as synthetic biology. You can find more information about publicly-listed companies in that sector in our article: “Top 5 Synthetic Biology Public Companies“.
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