Epigenetically Enhanced HIV Targeted CRISPR/Cas through T-cell specific Lipid Nanoparticles

A new public, private partnership between the University Medical Center Utrecht (UMCU) and biotech companies NanoCell Terapeutics, QVQ, and RiboPro is launching a two year project to tackle hidden HIV. The goal is to develop a new gene-therapy stragtegy that can eliminate HIV from the infected cells by removing the virus hidden deep inside the human genome.

HIV remains a major global health challenge, with over 38 million people living with the virus and yearly 1,3 million new infections worldwide, which is expected to increase. Current treatments allow people to live longer, healthier lives but do not cure the infection. People with HIV must take medication to keep the virus under control. This lifelong treatment is costly and can cause side effects, and if therapy stops, the virus comes back. Innovative solutions are urgently needed to truly cure HIV and reduce the long-term burden on individuals and healthcare systems.

This project explores a promising new approach using lipid nanoparticles (tiny fat-like particles) that specifically target T-cells where HIV hides. These nanoparticles will deliver two key components into the cells: a gene-editing tool called CRISPR/Cas (which acts like molecular scissors to cut DNA) and epigenetic enhancers (molecules that help expose the hidden virus DNA). By unmasking and then snipping out the dormant HIV genetic material from infected cells, the team hopes to completely remove the virus from those cells.

Over the two year project, the team will develop special antibodies that guide the nanoparticles to T-cells that can harbour HIV, create efficient formulations to package the CRISPR/Cas RNA and helper molecules, and demonstrate in lab experiments (including ex vivo tests) that this method can effectively eliminate HIV. These deliverables will pave the way for a future cure for HIV, offering hope to millions worldwide.