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.