December 12, 2023

Cell and Gene: Imagine A World Without HIV – Could Gene Therapy Deliver A Cure?

Jeff Boyle on Cell and Gene

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Cell & Gene

Addimmune Summary

Addimmune, the HIV division of American Gene Technologies, believes that the time for transformative HIV treatment change is now. While antiretroviral therapies (ART) are effective, challenges remain with non-adherence and treatment failure leading to ongoing transmission risk for many patients.

Why Gene and Cell Therapy?

This modality has proven successful against other diseases. In HIV, the goal is a functional cure: durable viral control without ARTs. Natural resistance to HIV exists in people with CCR5 gene mutations. The “Berlin patient” demonstrated a functional HIV cure through transplantation of bone marrow containing this mutation. Researchers now seek to leverage gene and cell therapy to achieve this goal more safely and widely.

Closing the Door: Targeting CCR5

Various strategies are being explored to inhibit CCR5, the protein allowing HIV to infect healthy cells. These include RNA silencing, ribozymes, intrabodies, intrakines, and CRISPR gene editing.

Added Protection: Targeting the HIV Genome

Researchers are seeking to directly target the HIV genome to block replication. Silencing viral genes like Tat and Vif, which are crucial to the virus’s lifecycle, holds immense promise.

Combined Gene and Cell Therapy for Sustained Immunity

HIV depletes CD4 T cells that are vital for fighting infections. Using combined gene and cell therapy, researchers are fortifying these cells by blocking CCR5 and activating them in the lab. This approach aims to create a robust, long-lasting immune defense against HIV.

The Promise of Gene Therapy

Gene and cell therapy has transformative potential, offering a functional cure that may eliminate the need for lifelong HIV treatment. While challenges remain, Addimmune is optimistic about its ability to overcome them, liberating people with HIV from current treatment burdens.