[1] The institute centers around research in the fields of organic chemistry, biochemistry and neighboring disciplines, mostly oriented at applications in medicine and environment.

Its most significant results are acyclic nucleotide phosphonate antivirals (ANPs) discovered by Antonín Holý at IOCB in collaboration with Erik De Clercq from the Rega Institute, which revolutionized[5] the development of antiviral drugs used in the treatment of HIV and hepatitis B.

Its prodrug forms tenofovir disoproxil fumarate and tenofovir alafenamide fumarate developed by Gilead Sciences were used in multiple drugs used for the treatment of HIV/AIDS and chronic hepatitis B, e.g. Viread (approved in 2001), Truvada (2004), Atripla (2006), Complera/Eviplera (2011), Stribild (2012), Genvoya (2015), Odefsey (2016), Descovy (2016), Vemlidy (2016), Biktarvy (2018), Symtuza (2018).

[3][10] At IOCB originated also other nucleoside compounds which became approved drugs: Decitabine used in the treatment of acute myeloid leukemia and Azacytidine for myelodysplastic syndrome, both discovered by Alois Pískala, and an acyclic nucleoside analogue duvira, 9-(2,3-dihydroxypropyl)adenine (DHPA), discovered by Antonín Holý, clinically used in treatment of infections caused by the herpes simplex virus.

The original institute was established under the Faculty of Chemistry of Czech Technical University in Prague and has been renamed multiple times.