PROVIREX Genome Editing Therapies GmbH

PROVIREX GmbH is a privately-held biotech company developing first-in-class advanced therapy medicinal products (ATMPs). PROVIREX is a spin-off of the Leibniz Institute for Virology (LIV) and is based in Hamburg, Germany. PROVIREX’s technology is based on error-free genome editing for eradication of life-threatening persistent virus infection. PROVIREX employs site-specific recombinase-based genome surgery technology to precisely excise chromosomally integrated viral genomes (proviruses). The major advantage of site-specific recombinases is that they function independently of host DNA repair. Therefore, in contrast to other gene editing technologies, they act with nucleotide precision producing completely error-free recombinants. The engineered recombinase Brec1 excises HIV-1 proviruses with high specificity from the genome of infected host cells. Brec1 activity has unparalleled and lasting antiviral effects in vitro and in vivo. Consequently, plasma viral load declines to levels below the detection limits of the most sensitive assay systems. No viral rebound has been observed. PROVIREX’s proprietary Brec1 technology is currently entering clinical testing as a first-in-human phase Ib/IIa clinical trial (HIVcure) aiming to remove HIV-1 from infected subjects using gene editing of the patient’s peripheral blood (hematopoietic) stem cells. HIVcure is being carried out at the University Medical Center Hamburg-Eppendorf (UKE) in collaboration with the Leibniz Institute for Virology (LIV). In this first phase, which was financed entirely from public funds, the treatment of the patient cells is carried out externally by the DRK Blutspendedienst Baden-Württemberg - Hessen gGmbH. For the next phase of the study, PROVIREX is building new clean rooms in Hamburg in order to be able to treat the patient cells there itself. In addition, PROVIREX will conduct research on a direct delivery of Brec1.