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.
According to a new report published by Allied Market Research, titled, “Gene Therapy Market,” The Gene Therapy Market Size was valued at $6.0 billion in 2020 and is estimated to reach $46.5 billion by 2030, growing at a CAGR of 22.8% from 2021 to 2030.
Increase in the prevalence of chronic disorders, rise in government support, and ethical acceptance of gene therapy for cancer treatment drive the growth of the global gene therapy market. On the other hand, high cost of gene therapies restrains the growth to some extent. However, the presence of advanced healthcare infrastructure is anticipated to pave the way for lucrative opportunities in the industry.
“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.”
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 AAV vector 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.
This research is supported by the National Institute of Allergy And Infectious Diseases of the National Institutes of Health under Award Number R01AI166969. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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