How biotechnology and gene therapy are transforming medicine

Half a century ago, most medicines were produced through chemical synthesis in laboratories or extracted from animal organs. For example, to produce insulin for patients with diabetes, it was necessary to use the pancreases of pigs and cows. This led to shortages of medicines and sometimes caused severe allergic reactions.

Biotechnology has completely transformed this approach. Scientists have learnt to use living cells, bacteria and natural mechanisms to create a new generation of medicines. Today, medicine can do more than simply suppress the symptoms of disease; it can precisely correct faults at the molecular and genetic levels.

How living cells became medicine factories

The main principle of medical biotechnology lies in reprogramming living organisms. Scientists take a human gene responsible for producing the required protein and insert it into the DNA of an ordinary bacterium or yeast cell.

The microorganism then begins to function as a miniature factory, producing with high precision the exact protein that a person needs.

Drugs based on protein molecules

Thanks to recombinant DNA, doctors have gained access to a supply of pure human proteins. Today, this technology makes it possible to produce safe insulin, growth hormones, interferons to protect against viruses, and blood clotting factors for patients with haemophilia.

The creation of monoclonal antibodies marked a major breakthrough. In the human body, these proteins act like high-precision keys that locate and block foreign objects. Biotechnologists have learnt to create artificial antibodies targeted at specific proteins found in tumours or inflammatory processes. This makes it possible to treat severe forms of cancer and autoimmune disorders with minimal damage to healthy tissue.

Genetic instructions instead of attenuated viruses

Previously, to create vaccines, doctors had to cultivate attenuated or inactivated viruses. Modern vaccine platforms work differently.

Messenger RNA (mRNA) technology sends a temporary genetic instruction to human cells. The cell reads this ‘recipe’, independently assembles a harmless fragment of a viral protein and presents it to the immune system. The body’s defences recognise the enemy and prepare for a future encounter with the infection without actually coming into contact with the live pathogen. International expert organisations note that mRNA platforms are versatile and enable the development not only of preventive vaccines but also of therapeutic vaccines against cancer.

Cell-based products and living medicines

Cell therapy takes the concept of treatment to a whole new level. In this case, the role of the medicine is played not by a chemical tablet or a protein solution, but by a living, functional human cell.

How CAR-T therapy works

The CAR-T method is being actively developed in oncology and haematology. The essence of the method lies in the individual training of the patient’s own immune cells.

The treatment process consists of several consecutive steps:

blood is taken from the patient and T-lymphocytes, which are responsible for the immune response, are isolated from it;
in a specialised laboratory, a gene for a new receptor is inserted into the lymphocytes’ genome using a safe viral vector;
the modified cell is equipped with a specialised ‘radar’ capable of recognising a specific protein on the surface of the cancerous tumour;
millions of these modified cells are cultured in an incubator and reintroduced into the patient’s bloodstream.

Once inside the body, these cells independently locate tumour structures and destroy them. Medical regulatory bodies classify CAR-T as a high-category personalised living medicine.

Gene therapy and DNA editing

Many serious diseases arise from single errors in the human genetic code. If a single gene functions incorrectly or is missing altogether, the body ceases to produce an important protein. Gene therapy aims to correct precisely this root cause of the disease.

Replacing a defective gene

Conventional gene therapy uses an approach known as ‘re-coding’. If a person has a specific damaged gene, doctors take an inactivated virus, remove its own pathogenic material and insert a healthy copy of the human gene into it.

The virus acts as a microscopic taxi. It penetrates the patient’s cells and delivers the functional gene there, which then begins to produce the required protein.

This approach has proven effective and has received official approval for clinical use in the treatment of a number of serious conditions:

spinal muscular atrophy;
haemophilia types A and B;
certain genetic forms of blindness and retinal dystrophy;
severe forms of primary immunodeficiencies.

Precision editing using CRISPR

CRISPR technology works differently. It can be compared to a built-in text editor. This tool is capable of locating a specific section within a DNA strand comprising many billions of bases, precisely cutting it, removing an erroneous base or replacing it with the correct nucleotide sequence.

The official approval of the drug Casgevy for the treatment of sickle cell anaemia and beta-thalassaemia marked a clinical breakthrough. Scientists extract the patient’s blood stem cells, correct the genetic error using CRISPR ‘molecular scissors’, and return the edited cells to the bone marrow. As a result, the body begins to produce healthy red blood cells.

Laboratory successes with CRISPR in cell cultures often make for sensational headlines in the press. However, researchers urge a clear distinction to be made between experimental studies and ready-to-use medicines. A successful experiment on cells in a test tube does not guarantee that the method will be safe for a living person.

Why the journey from the laboratory to the chemist’s takes years

The development and manufacture of biological medicines differ fundamentally from the production of simple pharmaceutical tablets. An aspirin or paracetamol molecule can be easily described by a precise chemical formula and synthesised at any chemical plant.

A biological product is produced by living cells. Its properties, purity and safety depend on hundreds of parameters: cultivation temperature, the composition of the culture medium, purification methods and transport conditions.

Safety and risk control

International expert committees require strict quality control at every stage of the production of biological products.

Specialists check the following parameters:

the absolute purity of raw materials from foreign bacteria and viruses;
the accuracy and stability of the insertion of new genes;
the absence of off-target DNA breaks when using CRISPR technologies;
the body’s immune response, to prevent anaphylactic shock;
long-term effects to rule out the risk of mutations developing over the years.

Economics and accessibility of innovations

The main obstacle to the widespread adoption of biotechnologies remains their high cost. The development and production of a single dose of personalised gene or cell therapy entail enormous expenditure.

For this reason, the scientific community is working on standardising processes, developing biosimilar medicines and finding simpler ways to deliver genetic material.

Biotechnology provides medicine with powerful tools to target the root causes of diseases. However, every new method must undergo a full course of controlled trials to prove its absolute safety for humans.

FAQ Answers to frequently asked questions

What is medical biotechnology in simple terms?

It is the use of living cells, bacteria, proteins and genes to create medicines, vaccines and treatments for complex diseases.

Is all gene therapy considered experimental?

No. Some gene therapy treatments have successfully completed all phases of clinical trials and are officially used in hospitals to treat spinal muscular atrophy, haemophilia and retinal disorders.

What is the difference between gene therapy and CRISPR editing?

Gene therapy delivers an additional healthy copy of a gene into the cell. CRISPR editing precisely locates a mistake in the cell’s own DNA and corrects it on the spot.

How does CAR-T therapy work?

The patient’s own immune cells are collected, genetically modified in the laboratory to recognise the tumour, and returned to the bloodstream to fight the cancer.

Why can’t CRISPR breakthroughs from the laboratory be used on humans straight away?

In a test tube or on cell cultures, it is difficult to assess the overall impact of the method on the entire body. Years of research are required to ensure that no accidental damage is caused to other healthy genes.

Why are biotechnological medicines so expensive?

Their production requires working individually with living cells, highly complex sterile conditions, expensive equipment and lengthy clinical safety trials.