Nucleic Acid Therapies – A Medicinal Revolution

Nucleic acid therapeutics are changing healthcare by offering transformative potential to treat diseases by targeting their genetic blueprints. Instead of treating symptoms, these therapies have the potential to address the disease root cause and potentially cure the previously incurable. Big investments are being made with Sweden emerging as a vibrant hotspot.

Addressing the disease root cause
After decades of intense research, nucleic acid therapeutics have now become increasingly common in clinics, with the mRNA COVID-19 vaccines as perhaps the most widely recognized example. These therapies utilize the building blocks of our genetic code, nucleic acids, that work inside the cells to affect the gene expression by inhibiting, adding, replacing or editing at DNA or RNA level. By doing so, nucleic acid therapeutics can achieve long-lasting or even curative effects.

“We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes”
- James Watson, Nobel price winner for discoveries regarding nucleic acids structure

The development of nucleic acid therapies is rapidly evolving driven by scientific breakthroughs and major investments in R&D activity. There are over 45 approved nucleic acid therapies  worldwide to date and over 2 800 in development, addressing diseases such as various cancer types, Parkinson’s and Arthritis. In 2023, there are at least 10 new therapies that could be up for approval in US and Europe¹.

Source: Triathlon analysis, based on FDA & EMA approvals for gene therapies, oligonucleotides, siRNA & mRNA


More than just favourable circumstances
Being an emerging and promising field, the clinical translation is however dependent on managing the associated challenges and considerations by developers and researchers. These are related to complex ethical considerations, high costs and new reimbursement models, delivery challenges, among others. To address the challenges, interdisciplinary collaboration is paramount - between and within academia, healthcare and industry. Major investments are being made around the world, including infrastructural investments in Sweden to accelerate the development.

Presenting a potential healthcare paradigm shift
Nucleic Acid Therapies offer a groundbreaking potential. New therapies are continuously approved providing life-changing treatments. Today’s unimaginable could become tomorrow's standard of care.


¹Alliance for Regenerative Medicine

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