Our Technology for HIV

Gene-Modified Cell Therapies for HIV

HIV


Antiretroviral therapy (ART) has changed HIV-infection from a death sentence to a chronic disease. However, ART requires life-long therapy that is expensive and has the risk of significant side effects. In addition, drug resistance is growing requiring new and often more costly products. From a person-centered approach, life-long therapy can be challenging and, unfortunately, stigma and discrimination remain strong throughout the world. More than 40 percent of the 36.7 million people who need therapy do not have access to it. More than one million people die from HIV every year and more than 1.8 million become newly infected. A cure and preventive or therapeutic vaccine could transform the lives of millions of people.

It has been proven that gene-editing to knockdown the expression of CCR5 — a door HIV needs to enter and kill CD4+ T cells — in autologous human stem cells (HSC) combined with transplantation can lead to a cure of HIV. However, the approaches currently available require an expensive and risky ablation of the immune system. Even with that drastic intervention, an insufficient number of gene-modified cells survive to achieve durable control of HIV.

We have pioneered a novel approach that we believe will allow sufficient engraftment of the gene-modified HSC to eliminate the need for ART.

In addition, we are evaluating potential game-changing approaches to immunotherapy that might be effective as preventive and/or therapeutic vaccination.