Precision gets U.S. patent for in vivo gene editing program

Precision BioSciences has been issued a U.S. patent that encompasses the engineered meganucleases utilized in the company’s lead in vivo gene editing program designed to effectively cure chronic hepatitis B.

The patent, which expires in March 2042, recognizes a highly conserved target sequence present in both HBV covalently closed circular DNA (cccDNA) and integrated HBV DNA. Precision was granted patents in Europe and Hong Kong earlier this year with similar composition of matter claims.

PBGENE-HBV — which utilizes lipid nanoparticle technology from Acuitas Therapeutics — is the first and only potentially curative gene editing program to enter clinical investigation that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA.

Precision shared phase 1 low-dose and safety data back in August. Cohort 1 of the ELIMINATE-B trial consisted of three patients, each of whom received three planned administrations of PBGENE-HBV dosed approximately eight weeks apart. While one patient achieved durable undetectable levels of HBsAg (a marker for active infection) seven months after dosing, the other two patients in Cohort 1 demonstrated antiviral response after each dose administration but returned to baseline levels of HBsAg. In Cohort 2, consisting of three patients, no adverse events above Grade 2, no serious adverse events, nor dose-limiting toxicities were observed.

Precision recently began enrolling and initiated dosing in Cohort 3 to test the next higher dose level. The company says it plans to provide further data updates this year.