Biotechnology and DNA: The duo paving the way for the era of customization in health and agriculture

On April 25, 1953, American biologist James Watson and British theoretical physicist Francis Crick published an article in the journal Nature describing their model of the double helix structure of DNA (deoxyribonucleic acid). This provided a fundamental understanding of how genetic information is stored and transmitted in living organisms, leading them to claim they had discovered "the secret of life." In 1962, Watson and Crick were awarded the Nobel Prize in Medicine (along with physicist Maurice Wilkins) for this discovery. A finding that would not have been possible without the pioneering research in X-ray diffraction by British crystallographer Rosalind Franklin, who in 1951 obtained a key image ("Photograph 51") showing for the first time the aforementioned helical structure of DNA.

The discovery of the double helix structure of DNA opened new perspectives in fields such as genetics, molecular biology, or medicine, laying the groundwork for the development of modern biotechnology. Biotechnological advances rooted in DNA have transformed numerous areas such as health or the environment, as demonstrated by the use of new genomic editing techniques in crops, resulting in crops more resistant to diseases, pests, and adverse weather conditions.

Next-generation DNA sequencing has a significant impact on personalized medicine, genomic research, or the identification of genetic diseases. Understanding DNA allows for the development of personalized treatments based on patients' genetic profiles, such as gene therapies: by modifying DNA within the patient's cells, it is possible to correct or compensate for defective genes, opening new doors for the treatment of genetic disorders and serious illnesses.

Pharmacogenomics combines pharmacology and genomics to develop safe and effective drugs tailored to an individual's genetic composition. This not only increases the effectiveness of medications but also reduces the risk of adverse reactions, optimizing pharmacological treatments. These are just a few examples of how DNA has revolutionized the foundations of biotechnology and placed us on a promising horizon. On the occasion of World DNA Day, we analyze how some of AseBio's partners are working on the latest biotechnological innovations rooted in DNA.

CRISPR Technology and the Biomedicine Revolution

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary technology in molecular biology that enables precise and efficient gene editing. The CRISPR/Cas9 system employs an enzyme called "Cas9" along with a guide RNA to target specific DNA sequences within a genome. Once the enzyme binds to that sequence, it can cut the DNA at that location. This allows for both gene insertion and deletion, providing a powerful tool for biological research and the potential to treat genetic diseases or develop crops, among other applications.

Genome editing has the potential to address real challenges for farmers and the planet, such as reducing the need for pesticides and the use of energy, land, and water, as well as developing crops more resistant to diseases and the effects of climate change. In agriculture, this genome editing process typically aims to enhance a beneficial trait within an organism or eliminate an undesirable trait. The latest advances in plant breeding techniques are used to improve seeds, achieving greater efficiency and specificity than ever before.

"With just over 10 years of existence and awarded the Nobel Prize in 2020, CRISPR technology has revolutionized biomedicine: it has made it possible to democratize the use of genome editors, greatly accelerating their development, and has paved the way for therapies for a wide range of genetic and oncological diseases, some of which are already approved for therapeutic use in patients. Now the challenge is integrating genome editing tools with the ability to write new therapeutic messages into the patient's genome," explain from Integra Therapeuctics, a biotechnology company developing genome editing tools to optimize the effectiveness and safety of advanced therapies.

In this regard, the FiCAT platform (Find and Cut-and-Transfer) stands out, which solves current technical limitations in terms of safety, efficiency, and precision for the development of advanced therapies. "It combines the precision of CRISPR-Cas9 molecular scissors, capable of cutting the patient's cell DNA where needed with an accuracy that no engineering tool can match, and the transfer efficiency of modified transposases, which are proteins that nature has evolved to perform this function of copying and pasting a complete therapeutic message into the DNA."

"By making this combination, we have managed to maintain efficiency in gene writing, but very precisely at a single position in the genome that we can control and change depending on the solution we want to find for each disease," explain from Integra Therapeutics.

FiCAT is in the regulatory preclinical phase. In 2023, very promising ex vivo and in vivo data were presented at the Global Synthetic Biology Conference in the United States and Advanced Therapies Europe. "We have a platform prototype that works very well in the laboratory and have implemented it to be able to produce advanced therapy products, both gene therapy and cell therapy. The forecast is to start clinical trials in 2026.

Genomic Medicine: Laying the Foundations for the Present and Future of Healthcare

It is undeniably a fundamental piece today and indispensable tomorrow. Genomic medicine is not only setting the trend currently but is also shaping up as a central pillar of the future of healthcare," argues Guillermo Pérez Solero, founder and CEO of ADNTRO Genetics es una start-up española biotecnológica cuyo objetivo es facilitar la comprensión de los datos genéticos y servir como herramienta entre la parte clínica y de laboratorio.

a Spanish biotechnological startup whose goal is to facilitate the understanding of genetic data and serve as a bridge between the clinical and laboratory aspects.

"The possibility of customizing medical treatments based on each individual's genetic composition is a revolution that is transforming our approach to health. This personalized approach is evident in the way complex diseases such as cardiovascular diseases are being addressed. Renowned institutions like the American Heart Association (AHA) already recognize and endorse the integration of algorithms based on polygenic risk scores (PRS) to refine diagnoses and optimize clinical treatments. These advances are not merely incremental but represent a qualitative leap towards an era where medicine is as unique as each patient's DNA," he adds.

ADNTRO has developed a tool for healthcare professionals that can easily analyze results for an individual regarding diseases of interest. This tool, aimed at healthcare professionals, facilitates the interpretation of patients' genetic data for better guidance in clinical diagnosis and as supplementary information for the physician. This includes interpretation algorithms that can help healthcare professionals better understand the implications of certain genetic variants on their patients' health and is available for both genotyping (arrays) and whole genome sequencing, following ACMG guidelines.

The current landscape shows an increasing number of countries developing strategies for integrating genetic testing into national healthcare systems. An example of this is Spain and the recent announcement by the Ministry of Health of the new Common Catalog of Genetic and Genomic Tests of the National Health System.

The catalog, currently comprising 672 tests, will continue to be progressively expanded due to its flexible nature, with a commitment to ensuring more homogeneous and equitable access to these tests for all patients in need. "The catalog includes prognostic and predictive biomarker analyses, essential for precision medicine, especially in the field of cancer, where genetic and genomic tests play a crucial role in diagnosis, prognosis, and treatment selection. This approach not only improves diagnostic accuracy but also the efficacy of therapies and, consequently, the quality of life of patients," highlight positive aspects from ADNTRO.

The company also values other steps such as the "Genetic Analysis Map" carried out in Spain within the framework of the National Health System. "These developments indicate a significant commitment from Spain towards integrating genetics into healthcare, aiming for improved equity in access to these essential tests, regardless of patients' geographical location within the country."

In conclusion, biotechnological applications rooted in DNA are manifold and open up new frontiers in the field of health, such as genomic medicine, essential for advancing a more personalized, preventive, and effective approach to healthcare. The discovery of the double helix structure of DNA has since that historic moment contributed to a deeper understanding of the underlying biology of diseases and, through tools like biotechnology, improving diagnoses and treatments.