Error-Free Genome Editing
PROVIREX Genome Editing Therapies GmbH is a privately-held biotech company developing first-in-class advanced therapy medicinal products (ATMPs). 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.
The following video schematically depicts the therapeutic mechanism of Brec1.
On the road
PROVIREX Genome Editing Therapies GmbH and Pantherna Therapeutics GmbH are developing lipid-based delivery systems for mRNA-directed genome editing of lymphocytes for the treatment of HIV and other life-threatening persistent viral diseases.
PROVIREX and Pantherna Therapeutics have entered into a research collaboration. Based on Pantherna’s proprietary PTXxLNP delivery technology both parties will develop and evaluate novel lipid-based mRNA formulations for the transfection of CD4+ lymphocytes. PTXxLNP is a promising candidate for the non-viral delivery of PROVIREX’s proprietary antiviral genome editing tool Brec1.
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 Heinrich Pette Institute – Leibniz Institute for Experimental Virology (HPI). Original press release
Company foundation of PROVIREX Genome Editing Therapies GmbH
Karpinski et al. (2016). Directed evolution of a recombinase that excises the provirus of most HIV-1 primary isolates with high specificity. Nat Biotechnol. 34:401-409.
Buchholz & Hauber (2016). Antiviral therapy of persistent viral infection using genome editing. Curr Opin Virol. 20:85-91.
Meinke et al. (2016). Cre Recombinase and Other Tyrosine Recombinases. Chem Rev. 116:12785-12820.
Hauber et al. (2013). Highly significant antiviral activity of HIV-1 LTR-specific Tre-recombinase in humanized mice. PLoS Pathog. 9:e1003587.
Buchholz & Hauber (2013). Engineered DNA modifying enzymes: components of a future strategy to cure HIV/AIDS. Antiviral Res. 97:211-217.
Buchholz & Hauber (2011). In vitro evolution and analysis of HIV-1 LTR-specific recombinases. Methods. 53:102-109.
Sarkar et al. (2007). HIV-1 proviral DNA excision using an evolved recombinase. Science. 316:1912-1915.
Who we are
PROVIREX is a spin-off of the Heinrich Pette Institute – Leibniz Institute for Experimental Virology (HPI) and is based in Hamburg, Germany. Our team combines profound expertise in biotechnology (particularly genome editing), virology, cell biology, drug development and commercialization.
PROVIREX Genome Editing Therapies GmbH
20251 Hamburg, Germany