Precision Therapies could 6x from 500 billion to 3 Trillion by 2030

The Old Therapeutic Method

Until recently, most of the pharmaceutical industry has been focused on finding new chemical molecules that could be used as drugs. The idea is to find chemical compounds affecting body and cells.

This has often been a difficult method. At first, the pharmaceutical industry could rely on traditional medicine to isolate the active molecules from plants and other natural sources. This is where medicine like Aspirin came from.

Progressively, they had to find entirely new molecules. This meant a lot of trial and error. As a rule of thumb, identifying only one medical treatment could take as much as 10,000 candidate molecules. Such a process is naturally very time-consuming and expensive.

In a nutshell, this approach can be described as “a solution looking for a problem”. You start with the chemistry and determine if it can do something in the body. And ideally, not kill the patient…

Source: Why do 90% of clinical drug development fail, and how to improve it?

Most of the time, these drugs would only treat the symptoms. You can administer painkillers to remove the pain signal, but it does not solve the cause of the pain. We knew what a cancer cell looked like and what might kill it, but not why it turned cancerous.

This forced doctors to shoot in the dark, hoping to find something that worked.

Another problem with this method is that active molecules tend to have more than one biochemical effect. So, while it might have the intended effect on, let’s say, the lungs, it might also impact the heart, liver, or brain.

This is why most medicines have a long list of “side effects”. This is because it is rather rare for an active drug to have only the desired effect.

The New Approach

The old way made sense decades ago, as we knew so little about the body’s mechanisms. Before 1953, we even did not know DNA was a double helix. It took up to the 1970s for PCR to be discovered and even longer for it to be routinely done at an acceptable cost. The human genome was not sequenced until 2003, and the project did cost billions.

The difference is that the cost of full genome sequencing is below the $1,000 mark today.

So, it is only now that we are getting a reasonable picture of what is happening in the cells of our body. We finally understand properly how the DNA “database “turns into the RNA “coding instruction,” which turns into active proteins.

Source: Ark Invest

The first consequence of all this knowledge is that we now understand the root cause of many diseases. A defective protein, a missing gene, or a specific hormone not doing its job properly. Instead of blindly modifying the body chemistry and hoping for the best, we know what’s wrong.

The second consequence is allowing the pharmaceutical industry to turn its head to the classical approach. It can now start with what is wrong in the body and aim at fixing it.

Starting from the problem and looking for a solution.

New Tools

The last decade has also seen a lot of biochemical tools move from the research lab to medical research or commercialized treatments:

• Monoclonal antibodies
• Customized gene editing
• RNA silencing (RNAi)
• mRNA vaccines
• Tissue-specific targeting
• Stem cells
• Protein degraders
• Immunity modulators
• Gene and protein-based diagnostic
• Tissues 3D printing

All of these treatments are often brought together under the umbrella of “Precision Therapies”. In opposition to the “old way” of chemical-based untargeted therapies.

Monoclonal antibodies have been the first. mRNA vaccines have made a dramatic entry during the pandemic. The others will be as important and transformative.

The Precision Therapies Market

Currently, precision therapies are a $500B market, according to an estimate by Ark Invest. It is not anymore just an idea or a potential medicine, but the driving force behind most of the pharmaceutical sector growth in the past decade.

(the exact market value of precision therapies can vary greatly between estimates, depending on what is considered precision therapies)

In the same study, Ark Invest estimates this market will grow to $3T by 2030. This would be a 6x growth in just the next 7 years.

A few sub-sectors are the driving force behind this growth

Gene editing

A lot of rare diseases are caused by a defective or missing gene. The disease’s root cause is deep in every cell and comes from a missing biochemical function. This meant there was nothing for a chemical drug to “activate” or “suppress”. Only restoring the missing function in every single cell can cure the disease.

Early gene editing has been the focus of ex vivo, correcting the cells in a lab and re-injecting them into the body. Newer methods will focus on in-vivo therapies, editing the gene of every targeted cell directly in the body.

mRNA

This is the most known by the broader public due to the mRNA vaccine against Covid-19. But vaccines are far from the only potential application of mRNA. The most promising one is actually cancer therapy. You can read more about it and other mRNA applications in our article “The Next Application for mRNA Technology: Cancer Therapies”

Synthetic Biology

While gene editing modifies existing genes, synthetic biology adds entirely new genes and long DNA sequences. So, this is the natural next step for gene therapies. You can read more about it in our article “Top 5 Synthetic Biology Public Companies“.

Targeted Protein Degraders (TPD)

Sometimes, diseases are caused by the presence of incorrect proteins and not by missing genes. TPD can reduce this protein number and address syndromes solvable nether by drugs or by gene editing. This is an emerging field really studied only since 2016, but with already some candidate medicines in phase II of clinical trials.

Liquid Biopsy / Molecular Cancer Diagnostic

Most cancers have specific genetic sequences distinguishing them from healthy cells. The problem has been for a long time to manage to detect these changes without having to sample parts of the affected organs directly. In addition, the sooner a cancer Is detected, the more chances to survive it.

Liquid biopsy uses the most powerful genetic detection methods to find cancer markers in the blood. This can allow for routine cancer checks and early detection at a low cost.

You can read more about the leading company in liquid biopsy, Grail, in our article about its mother company, “Illumina vs Pacific Bioscience: Choosing the Next Generation Genome Sequencing Company”

Lastly, full genome analysis to detect pre-existing vulnerability to specific cancer types can help to know in advance which patients need more regular monitoring.

A Selection of Precision Therapies Companies

Without doing a detailed analysis of each company, we can mention a few companies that are leading the charge in turning precision therapies into multi-billion dollar blockbuster treatments (some of them have been mentioned earlier and analyzed in more detail in the corresponding articles).