Russian Drugmaker and University Win Grant to Build CRISPR-Based Hepatitis B Cure

A project by Russian pharmaceutical company R-Pharm and Sechenov University to build a manufacturing platform for treating chronic hepatitis B using CRISPR-Cas9 genome-editing technology has won a grant in a Russian Science Foundation competition held in memory of academician Yevgeny Velikhov. The competition, held on the president's instructions, aimed to develop new bioeconomy technology for pharmaceuticals and healthcare.

The project involves building a scalable platform that packages CRISPR-Cas9 complexes targeting the hepatitis B virus into membrane nanoparticles, intended to deliver the gene editors throughout the body to fully eliminate the virus. CRISPR-Cas9 is a targeted genome-editing technology, sometimes called "molecular scissors," based on the Cas9 protein, which cuts DNA at a site the system is directed to.

According to R-Pharm, up to 200 copies of the editing complex can be loaded into a single nanoparticle, compared with 20 to 60 for comparable approaches, which the company says improves efficacy. The drug is designed to have reduced off-target activity and increased specificity for infected liver cells, and is intended as a single administration, which could lower the cost of treatment. "Genome editing is today one of the most dynamic areas of global biotech," said Vladimir Andrianov, R-Pharm's director for partner relations. "There are only a handful of end-to-end projects of this level in the world, and in Russia this is the first experience of building a full-cycle platform."

The Russian Science Foundation grant will fund the project's move from laboratory development to a pilot industrial prototype. The company says the platform lets the basic construct stay the same when targeting a different disease, with only the guide component needing to be reconfigured, opening the way to personalized therapy protocols.