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, which requires new and often more costly products.  From a patient-centered approach, life-long therapy can be challenging, and unfortunately, stigma and discrimination remain strong throughout the world.  More than 40 percent(40%) 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 a 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 vivo and in vitro data supporting the scientific foundation of the approach has been presented at the annual meeting of The American Society of Gene and Cell Therapy in May 2020.

Increased Engraftment of Gene Modified HSPCs Overexpressing ALDH1 In Vivo

Poster Presentation Link Here Slide Presentation Link Here

Gene Modified CD34+ Cells with Increased ALDH1 Expression Confers In Vitro Protection Against Cyclophosphamide

Poster Presentation Link Here Slide Presentation Link Here

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