CytoDyn Inc. announced a new, preclinical study in nonhuman primates that will evaluate the potential use in HIV of a gene therapy based on the experimental monoclonal antibody leronlimab. The research will be led by Oregon Health & Science University (OHSU) researcher Jonah Sacha, Ph.D., also serves as a CytoDyn scientific advisor. The study is funded by a five-year grant of up to $5 million to OHSU from the National Institute of Allergy and Infectious Diseases of the National Institutes of Health (NIH).

The grant will fund the development and preclinical research of a single-injection gene therapy that codes for the leronlimab protein sequence and which will be delivered via an adeno-associated virus (AAV) vector. The study will examine if this gene-therapy approach could provide the potential for functional cure, sustained viral suppression to people with HIV without requiring them to take medications for the rest of their lives. Leronlimab has demonstrated it can pharmacologically mimic a CCR5 deficient donor by occupying available CCR5 molecules.

Leronlimab is a protein, and the goal of the research at OHSU is to create a gene therapy that expresses the gene encoding the leronlimab protein. This gene therapy will also require a new delivery modality. The grant will fund the design of synthetic novel AAV vectors specific for T and B cells, with the goal of facilitating long-term expression of leronlimab by the body's own T and B cells.

A novel AAV vector may also support the future development of other anti-HIV approaches, including CRISPR-Cas9, chimeric antigen receptors, and broadly neutralizing antibodies, as it may be able to deliver these therapeutics to the relevant immune cell type. The desired outcome of this research is the development of a safe and effective single injection that suppresses HIV replication long-term, eliminating the need for frequent dosing with antiretroviral therapy.