Innovation on the horizon: how big pharma investment is driving the development of oligonucleotide-based medicines

The mission of the OLIGOFATSX project is to develop oligonucleotide-based medicines to improve the quality of life of patients affected by rare diseases for which there is still no solution. The scientists involved in the project firmly believe that the future of personalised precision medicine lies in the successful combination of different technologies, including oligonucleotides;

And we are not alone: global industry is investing and researching in the development of oligonucleotide-based medicines.

This blog will explore how these investments are driving the advancement of oligonucleotide research and what impact they could have on the future of medicine.

Introduction to oligonucleotide-based medicines

Oligonucleotide-based medicines are a promising technology that has gained ground in recent years in the field of medicine. These drugs use small nucleic acid molecules to interact with DNA or RNA to regulate gene expression and treat various diseases.

Large pharmaceutical companies have invested heavily in the research and development of oligonucleotide-based medicines, driving innovation and the advancement of this technology. Thanks to these investments, important advances have been made in the understanding of molecular biology and the identification of new therapeutic targets.

The benefits of these investments have resulted in the approval of several oligonucleotide-based medicines by international regulatory agencies, opening up new opportunities for the treatment of rare and complex diseases.

Despite progress, there are still challenges to overcome, for example in terms of accessibility and cost. However, the promise of oligonucleotide technology remains significant, and its clinical application promises to continue to expand in the future.

How big pharma has driven innovation in oligonucleotide-based drug development

The oligonucleotide-related pharmaceutical industry pivots on a complex universe of patents and trade secrets as startups and specialised companies, always under the watchful eye of big pharmas in search of new opportunities.

In recent years, we are witnessing a scenario in which several companies, including GSK, AstraZeneca, Isis Pharmaceuticals, Rexahn Pharmaceuticals, Alnylam and Wave Life Sciences, have reached agreements to collaborate in the development of these therapies.

GSK has paid $170 million upfront to secure its place at the forefront of oligonucleotide research for years to come. AstraZeneca and Isis Pharmaceuticals have also announced a collaboration to develop specific oligonucleotide delivery methods. Rexahn Pharmaceuticals, meanwhile, has acquired an oligonucleotide drug delivery platform from Ohio State University. Alnylam and Isis have signed an agreement to extend their decade-long partnership in the development of ribonucleic acid-based therapies.

In addition, Sylentis, a subsidiary of PharmaMar Group, has recently inaugurated the first pilot plant in Spain for the manufacture of oligonucleotides under GMP standards to cover internal needs and act as a CMDO for other pharmaceutical companies that need to manufacture and analyse oligonucleotides.

All this also boosts ancillary industries, which are needed to produce raw materials, release nanotech drugs or develop new production and scale-up processes.

And, like any high-tech area, it requires strong value chains that prevent stock, technology gaps and produce imbalances in balances of payments.

We are facing a silent technological revolution where entrepreneurial initiatives aimed at not missing windows of opportunity are essential for a country.

Large pharmaceutical companies are playing a key role in the innovation and development of oligonucleotide-based medicines. Thanks to their large investments in research and development, these companies have made significant advances in the understanding of oligonucleotide biology and their clinical application.

The latest research study on the global Oligonucleotide Therapeutics market reveals that the global market reached a value of USD 4252.28 million in 2022. The market is expected to reach USD 10893.76 million by 2028, at a CAGR of 16.97% during the forecast period.

Pharmaceutical companies have invested in optimising the deliverability of oligonucleotides, improving stability and reducing toxicity, making these drugs more effective and safer.

In addition, large pharmaceutical companies have worked in collaboration with scientists and academics to accelerate the development of oligonucleotide-based medicines.

However, barriers to entry in the pharmaceutical industry are relatively high.

In addition to complying with general laws and regulations, companies in this industry must have the prescribed operating conditions, including legally qualified pharmaceutical technicians, engineering technicians and relevant technical workers; it must have factory buildings, facilities and a hygienic environment suitable for the production of pharmaceuticals; it has an organisation, personnel and necessary instruments and equipment capable of carrying out quality control and quality inspection of the medicines produced; there are rules and regulations to ensure the quality of medicines… to name but a few requirements of the sector.

The pharmaceutical industry is a high investment industry, its investment in new product development is high, the cycle is long, the risk is great. Therefore, new entrants often need a long time to get started and face great financial pressure. Pharmaceutical technology in the pharmaceutical industry is difficult, the equipment requirements are high, the process route is complex and the requirements of the production environment are very strict.

Thus, achieving effective delivery of oligonucleotide therapeutics in many tissues remains a major translational challenge.

Due to their physical and chemical properties, unmodified free oligonucleotides are not only eliminated by the body shortly after drug administration, but also present the risk of toxic and off-target side effects. Therefore, nucleic acid drugs need chemical modification and an appropriate delivery system to achieve a therapeutic effect.

Although challenges remain, such as optimising delivery and reducing production costs, the future of oligonucleotide-based medicines is clearly promising.

With the support of major pharmaceutical companies, this technology has the potential to transform the way many diseases are treated, potentially improving the quality of life for millions of people around the world.

Benefits of large pharmaceutical companies’ investments in oligonucleotide-based drug innovation.

Large pharmaceutical companies have invested significantly in oligonucleotide-based drug innovation because of the benefits they offer. These drugs are able to modulate gene expression in a highly specific way, making them a promising tool for the treatment of genetic diseases, cancers and infectious diseases.

In addition, oligonucleotide-based drugs have a high selectivity, which means that they can act on a specific molecule without affecting other molecules in the body.

This reduces side effects and improves the safety of treatment. Thanks to investments by large pharmaceutical companies, significant advances have been made in oligonucleotide-based drug research and development, leading to a greater understanding of the technology and enabling the creation of more effective and safer therapies.

In summary, investments by large pharmaceutical companies in oligonucleotide-based drug innovation have driven the development of new therapies and opened up new opportunities for the treatment of complex diseases.

Future perspectives for oligonucleotide-based drug innovation and clinical applications

Oligonucleotide technology offers great promise for the development of new medical treatments and therapies, and this has prompted heavy investment by large pharmaceutical companies.

These investments have driven innovation in the medical-scientific field and have enabled progress in the development of oligonucleotide-based medicines.

The future prospects for oligonucleotide-based drug innovation and clinical applications are very promising. It is hoped that these drugs will be able to treat diseases for which there is currently no treatment or solution.

In short, the investment of large pharmaceutical companies in oligonucleotide-based drug innovation has opened up new opportunities for breakthroughs in disease treatment and has generated great expectations in the scientific and medical community.

Oligonucleotide technology offers significant promise for the development of new medical treatments and therapies, which has prompted major pharmaceutical companies to invest heavily, opening up new opportunities and driving innovation within the medical-scientific field.

In summary, oligonucleotide technology holds significant promise for the development of new medical treatments and therapies. Large pharmaceutical companies have invested heavily in this technology, which has driven innovation in the medical-scientific field and opened up new research opportunities.

The benefits of these investments are clear, as oligonucleotide-based medicines have proven to be effective in the treatment of a variety of diseases.

However, there are still challenges to be overcome in terms of the clinical application of this technology. Despite this, the outlook for the future is promising and innovation in oligonucleotide-based medicines is expected to continue to advance in the coming years.

OLIGOFASTX is setting a milestone in the sector, not only from the point of view of research and development, but also because of the way in which we are carrying out the project in a transversal manner and with the best Spanish companies specialising in each of the stages of the project. We are convinced that the medium to long-term results will be efficient in improving the quality of life of patients affected by the rare diseases we are researching.

Sources of information

In Spanish:

  1. El Farmaconalisis –
  2. El Economista –
  3. Noticias de la ciencia y la tecnología –
  4. Diario económico –

In English:

  1. Fierce Biotech –
  2. Precision Oncology News –
  3. Biospace –
  4. Genetic Literacy Project –
  5. Seeking Alpha –
  7. 7. GSK pays $170M upfront to continue to ride oligonucleotide wave for years to come | Fierce Biotech
  8. AstraZeneca and Isis Pharmaceuticals to Co-Develop Targeted Oligonucleotide Delivery Methods | Fierce Biotech
  9. Rexahn Pharmaceuticals In-Licenses Novel Oligonucleotide Targeted Drug Delivery Platform from Ohio State University | Fierce Pharma
  10. Alnylam and Isis Form New Agreement, Extending Decade-Long Partnership for Leadership in RNA Therapeutics | Fierce Pharma
  11. Wave Life Sciences and GSK Announce Collaboration to Drive Discovery and Development of Oligonucleotide Therapeutics Focusing on Novel Genetic Targets | News & Events | Goodwin Procter (

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