NIH grant for research into functional cure for HIV

pharmafile | July 12, 2022 | News story | Manufacturing and Production  

The NIH has granted Oregon Health and Science Univerity (OHSU) a $5 million grant for the research of its one-time injection of a gene therapy based on leronlimab, for a functional cure of HIV. The researchers will evaluate the experimental drug’s potential as a gene therapy in nonhuman primates.

The injection could prevent people who have HIV from having to take daily antiviral pills for the rest of their lives.

The research will be led by Oregon Health & Science University researcher Jonah Sacha, PhD, who also serves as a scientific adviser to CytoDyn, the biotechnology company developing the drug, called leronlimab. In an earlier study, Sacha and colleagues found leronlimab completely prevented nonhuman primates from being infected with the monkey form of HIV, a result indicating that the drug holds promise as a potential pre-exposure drug, to prevent human infection from the virus that causes AIDS.

The study now being led by Sacha aims to design a way to offer leronlimab as gene therapy. Sacha and colleagues will explore how to contain the coding sequence of the experimental drug, inside a lab-made form of the adeno-associated virus, an approach that gene therapy researchers call an AAV vector.

This resulting therapy would then be injected inside the body, where muscle cells would make leronlimab long-term.

“This grant will fund the development and early study of leronlimab as a potential single-injection gene therapy,” said Sacha, professor at OHSU’s Vaccine and Gene Therapy Institute and Oregon National Primate Research Centre. “If this approach works as hoped, it could provide a functional cure for HIV, meaning it could suppress HIV enough that patients would no longer need to take daily antiviral pills for the rest of their lives.”

Worldwide, nearly 38 million people live with HIV, and around 73% of them receive treatment. Of the 37.9 million people living with HIV around the world, around 23.3 million (62%) are receiving antiretrovial therapy, and have the virus under control. Poverty, gender inequality, and HIV stigma and discrimination are significant barriers to HIV prevention in many countries.

Ana Ovey

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