One-time gene therapy injection could provide HIV treatment that may last a lifetime
A new pre-clinical study in nonhuman primates will evaluate an experimental drug’s potential use as a gene therapy that could prevent people who have HIV from having to take daily antiviral drugs for the rest of their lives.
The research will be led by Oregon Health & Science University researcher Jonah Sacha, Ph.D., who also serves as a scientific adviser to CytoDyn, the biotechnology company developing the drug, called leronlimab. The study is funded by a five-year grant of up to $5 million that was recently awarded to OHSU by the National Institute of Allergy and Infectious Disease, which is part of the National Institutes of Health.
“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 Center. “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."
In an earlier study, Sacha and colleagues found leronlimab completely prevented nonhuman primates from being infected with the monkey form of HIV. That result indicated leronlimab held promise as a potential pre-exposure drug to prevent human infection from the virus that causes AIDS.
Now, this study 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. The resulting therapy would then be injected inside the body where muscle cells would make leronlimab long term.
Leronlimab is a monoclonal antibody that blocks HIV from entering immune cells through a surface protein called CCR5. The drug has demonstrated it can mimic a CCR5-deficient donor by occupying all available CCR5 molecules, but this would require a new method for delivery as a gene therapy. Viral vectors have been used to deliver antigens from specific pathogens for decades.
In this project, researchers will design a synthetic AAVvector to enable the long-term production leronlimab inside the body. The goal is to develop a safe and effective single injection that suppresses HIV replication and eliminates the need for life-long antiretroviral therapy.
“Currently, patients often take multiple antiretroviral pills daily,” said Sacha. “Removing this pill burden could not only improve patients’ quality of life, but remove problems with adherence.”
Rhesus macaques at OHSU’s Oregon National Primate Research Center that have been exposed to a monkey version of HIV will be given a single AAV injection that contains leronlimab. Researchers will monitor the nonhuman primates for years to assess the safety and efficacy of this approach.
https://news.ohsu.edu/2022/07/08/one-time-gene-therapy-injection-could-provide-hiv-treatment-that-may-last-a-lifetime
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