Gene therapy using the CRISPR tool promises to revolutionize medicine – eliminating diseases at their source, in the genes. But how many diseases are really genetically determined, what does the pharmaceutical industry cover today, and will we even need drugs in the future? A brief overview of what is already possible, what is still science fiction – and why drugs are still very much alive.
When we talk about genetically conditioned diseases, we are not just talking about rare cases like cystic fibrosis or Huntington's disease. Most of the diseases that plague us in life – cancer, diabetes, heart disease, depression – have at least a partial genetic basis. It is estimated that as many as 70 to 80 percent of all chronic diseases have some connection to genetics.
This does not mean that our genes are the only culprits. Environment, lifestyle, and chance also play a large role in most diseases. But the fact remains – without genes, diseases would not exist, or they would be quite different.
While rare diseases are usually caused by a mutation in a single gene (there are about 8,000 such diseases), more common diseases are much more complex. They involve multiple genes, along with environment, diet, stress, and habits. These are called polygenic diseases – and they are the biggest health challenges of our time.
Pharmacy: huge, diversified and (for now) indispensable
Most of the drugs we know and use today do not address the cause of the disease, but rather alleviate symptoms or slow its progression. For example, drugs for type 2 diabetes help regulate blood sugar, but do not correct the genetic defect that may contribute to the disease.
The pharmaceutical industry currently covers a large part of patients' needs - it is estimated that 70 to 90 percent of all people with diseases that also have a genetic component are already receiving some form of treatment. However, for rare, monogenic diseases, coverage is much less - for most of these 8,000 diseases, there is no cure at all or only experimental therapy is available.
This is not because science is not capable. Rather, it is because developing a drug for a rare disease is financially risky, extremely expensive, and uninteresting from a market perspective. Developing a gene therapy can cost hundreds of millions of euros, and it only needs to treat a few hundred people in the world.
CRISPR: the technology that cuts DNA and opens a new chapter
CRISPR is a tool that allows for precise DNA editing – like molecular scissors that cut out the wrong piece of genetic code and replace it with the correct one. Theoretically, it could eliminate the cause of disease, not just its consequences. In practice, however, we are still at the beginning of the journey. Although, thanks to AI, things now seem simpler than they were a few years ago.
We have seen the most progress so far in the treatment of rare genetic diseases and some cancers. Therapies such as Zolgensma for spinal muscular atrophy already exist – but they cost more than two million euros per patient. Moreover, only a few are approved in Europe, mainly for diseases that caused by a single gene.
For more common diseases, the situation is much more complex. These diseases are not the result of a single error, but networks of genetic and environmental factorsAnd CRISPR – for now (we don't know what tomorrow will bring) – is not precise and safe enough to effectively and reliably treat them.
There are also ethical and legal constraints. Gene editing in embryos (so-called germline therapy), which would allow the elimination of hereditary diseases before birth, is banned in most countries. And even if it were allowed, the question of who decides what is genetically “normal” would still be unresolved.
Why CRISPR won't (yet) replace pharmaceuticals
Although it seems like CRISPR could replace drugs, in practice it is not so simple. The technology has enormous potential, but it works mainly in very specific cases. Most diseases that we treat today require a different approach - long-term, gradual, often combined.
The pharmaceutical industry will not simply back down either. Large companies such as Pfizer, Novartis or Gilead are already investing in CRISPR researchNot because they are threatened by the technology, but because they want to be part of the next phase of medical development. In the future, we will likely see a hybrid model: some patients will receive gene therapy, while most will continue to use drugs.
Even for diseases that CRISPR can theoretically treat, medications will often still be needed – to control symptoms, prevent complications, or improve quality of life.
What about Slovenia?
Slovenia has well-developed genomic diagnostics, which means we can detect genetic changes associated with diseases. We also have several research groups involved in the development of gene therapies. However, access to CRISPR treatment is (and will remain) limited – mainly due to price, regulation and dependence on foreign countries.
Our pharmaceutical companies, such as Krka and Lek, are focusing on generic drugs, which remains crucial for the healthcare system. Gene therapy is not something that will soon become part of everyday healthcare practice – neither in Slovenia nor elsewhere in the world. Although things are changing very quickly precisely due to the development of artificial intelligence, implementation, namely mass implementation, will take at least a good decade. Regardless of some accelerated discoveries in the coming years.
Conclusion: CRISPR is not the end of pharma, but its new partner
Gene therapy is the future, but a future that is coming slowly and selectively. CRISPR will change the way some diseases are treated – especially rare and genetically simple ones. But most patients will need drugs that alleviate symptoms, improve quality of life and enable day-to-day management of the disease for a long time.
Rather than replacing pharmaceuticals, CRISPR will complement them. And if the technology becomes safer, more accessible, and cheaper in the coming decades, it could play a crucial role. Until then, drugs will remain the mainstay of healthcare—even for genetic diseases.
