Genetic Treatment Holds Promise for Blocking HIV Transmission During Breastfeeding

In a groundbreaking development, researchers at The Herbert Wertheim UF Scripps Institute have unveiled a novel gene therapy approach that could offer long-lasting protection against HIV-1 infection in newborns. This breakthrough, published in the prestigious journal Nature (DOI: 10.1038/s41586-025-09330-2), represents a significant advancement in preventative medicine and could potentially eliminate mother-to-child transmission of HIV.

The innovative strategy utilizes adeno-associated virus (AAV) vectors to deliver broadly neutralizing antibodies (bNAbs) directly to infants at birth. The gene therapy was evaluated in a primate model, and nearly 90% of newborn macaques exhibited robust expression levels of 3BNC117 following a single intramuscular injection. When challenged with simian-HIV, the vast majority of treated neonates were protected from infection, and this protective effect endured for multiple years.

The research heralds a paradigm shift in infectious disease prevention by leveraging in vivo gene delivery to establish passive immunity durably. Safety considerations were promising, with no adverse effects attributable to the gene therapy detected throughout the extensive monitoring period.

The study's findings were based on research conducted on macaques and were detailed in the article "Determinants of successful AAV-vectored delivery of HIV-1 bNAbs in early life" by Martins, M.A., et al. The vision of an HIV intervention administered once at birth, providing enduring protection, could redefine global HIV prevention strategies for newborns at risk through breastfeeding.

However, the path forward includes rigorous clinical trials in human infants to validate safety, dosing parameters, and long-term efficacy. The viral vector system's inherent stability and minimal cold chain requirements propose an ideal solution for deployment in remote or low-resource environments.

Prominent organizations and foundations supporting the development of gene-based HIV prevention strategies in human newborns include global health philanthropy groups and programs focusing on HIV/AIDS prevention and treatment. Generally, funding and support often come from international institutions like the Bill & Melinda Gates Foundation, the Global Fund, and governmental research agencies, although the provided data does not explicitly name them for this specific approach.

The image credits for the article are by Stacey DeLoye. This gene therapy approach may circumvent the logistical complexities of current ART regimens while empowering healthcare systems in underserved areas. The success of the study will depend on continuing scientific rigor, ethical clinical development, and equitable distribution worldwide.

Genetic Treatment Holds Promise for Blocking HIV Transmission During Breastfeeding