Breakthroughs Blog

Last week, Gilead Sciences announced that it has expanded the scope of its voluntary licensing agreements with six manufacturing partners, who are already manufacturing the recently approved twice-yearly lenacapavir for HIV prevention, to include investigational once-yearly lenacapavir. Following the unprecedented Phase 3 trials for twice-yearly lenacapavir, an ongoing Phase 3 trial is evaluating a once-yearly option that could provide a full year of prevention with just one annual dose, further expanding prevention options and addressing persistent barriers to uptake and adherence. Establishing these agreements at this early stage helps manufacturers, regulators, health systems, and communities prepare for the broad, rapid, and equitable scale-up of the once-yearly option, if it is eventually approved. The World Health Organization (WHO) has updated its malaria guidelines to include Coartem® Baby, the first antimalarial developed specifically for newborns and young infants weighing between two and five kilograms. This patient group previously had no medicine specifically designed for their unique needs, leaving them to be treated with modified doses of formulations intended for older children, raising the risk of treatment failure or toxicity. The drug was developed by Novartis and partner Medicines for Malaria Venture (MMV), with financial support from the European & Developing Countries Clinical Trials Partnership and the Swedish International Development Cooperation Agency. WHO’s inclusion of Coartem® Baby in the guidelines informs and enables malaria-endemic countries to introduce the treatment in national malaria programs, supporting broader access. After receiving prequalification by WHO and approval from Swiss and Ghanian regulatory authorities, Coartem® Baby has so far been rolled out in 11 countries. The Coalition for Epidemic Preparedness Innovations (CEPI) recently announced that it is awarding $3.9 million to support research projects aimed at improving understanding of the Bundibugyo ebolavirus behind the ongoing outbreak in the Democratic Republic of the Congo. Specifically, the funding will support modelling and analytic teams led by the Institut National de Recherche Biomédicale, Imperial College London, and Makerere University School of Public Health. These teams will collect, integrate, and analyze outbreak data to guide the outbreak response; inform the design of fast, efficient vaccine clinical trials; and support longer-term preparedness against similar outbreaks in the region. To ensure long-term applicability, the partners have placed a strong emphasis on capacity strengthening and open sharing of data and methods, so that the tools and knowledge these projects generate can be utilized in future outbreaks in the region. 

Last week, the World Health Organization (WHO) prequalified multi-dose vials of the maternal vaccine against respiratory syncytial virus (RSV), enabling rollout in lower-income countries. RSV poses a severe global threat, especially for children under five and newborns, in particular. The Gates Foundation supported the development of a multi-dose version of Pfizer’s Abrysvo maternal RSV vaccine, which is administered to pregnant people in their third trimester, protecting newborns against infection and greatly reducing babies’ risk of being hospitalized with RSV. Multi-dose vials simplify the logistics of vaccination, allowing health care professionals to vaccinate more people with fewer vials, which need to be stored and transported in ultra-cold conditions. The prequalification now enables international organizations, like Gavi, the Vaccine Alliance, which has already put implementation plans in place, to procure them for rollout in low-income countries. A research team has developed a nanobody-based antivenom that could provide a more flexible and effective approach for treating snakebites, which kill tens of thousands of people globally and disable hundreds of thousands more. Traditional venom is made by processing antibody-filled blood from horses injected with small doses of venom, but the quality of these antivenoms can vary, they can cause severe allergic reactions, they are expensive to produce, and they offer inconsistent protection across snakebites from different species. Nanobodies, tiny versions of antibodies, can move through tissue more quickly and reach venom before it can cause permanent damage. In a new study, all mice treated with the nanobody-based treatment survived. The treatment also prevented the most severe effects of the venom, is not expected to cause unwanted side effects, and remained intact at high temperatures, which is key in the resource-constrained rural settings that face heightened risk. Further studies will be needed to optimize the dose and ensure safety and efficacy in humans, but the study suggests that nanobody-based antivenoms could be a promising approach for further study. Immunoscript Life Science will receive up to $8.9 million from the Coalition for Epidemic Preparedness Innovations (CEPI) to further development of its thermostable next-generation self-amplifying RNA (saRNA) vaccine platform, which could help improve access to RNA vaccines globally. mRNA vaccines have demonstrated significant promise as a tool against emerging disease threats because of the speed, flexibility, and adaptability with which they can be produced, but their requirement for ultra-cold temperatures for storage and transportation has proven a barrier to access in low-resource and remote settings. The saRNA vaccine platform, now owned by Immunoscript, is an alternative technology that could help overcome challenges associated with the cold chain. With CEPI’s funding, the company will conduct an early-stage trial in people in India to assess the safety and immune response of the technology. 

Fungi have existed for hundreds of millions of years, long before dinosaurs walked the Earth, but fungal infections have become a rapidly growing challenge for human health in recent decades.Fungal diseases affect more than 300 million people each year, disproportionately impacting people in low-resource settings and those with weakened immune systems, including people living with HIV, cancer patients, and transplant recipients. Most fungal infections affect superficial tissue like the skin, nails, or hair and can cause chronic illness and productivity loss. Though less common, invasive fungal infections can spread throughout the body and are associated with high mortality.Why fungal diseases are becoming harder to ignoreThe rising incidence of fungal infections is driven by a variety of factors, including conflict, poverty, urbanization, pollution, travel, and climate change, which increase exposure, heighten vulnerability, and expand the reach of dangerous fungal pathogens. At the same time, advances in medicine mean more people are living longer with conditions that weaken the immune system, increasing the number of people at greater risk. Growing antifungal resistance is also making infections harder to treat and further limiting already scarce treatment options.Despite the mounting burden, fungal disease remains underprioritized, underdiagnosed, under-surveilled, and underfunded, making it one of the world’s most neglected global health threats.Why innovation is falling behindFungal infections receive less than 1.5 percent of all infectious disease research and development (R&D) funding, contributing to sparse pipelines for both therapeutics and diagnostics. Closing these gaps will require greater investment in R&D, particularly for tools designed for resource-constrained settings and targeting the pathogens that pose the greatest threat to human health. Stronger regulatory and procurement systems and international collaboration will also be essential to ensure emerging tools are affordable and accessible worldwide.Despite persistent gaps, several promising innovations demonstrate the potential for R&D to transform the global response to fungal disease.Promising advances in antifungal treatmentThere are currently only four classes of antifungal medicine available, many of which are associated with serious side effects or require lengthy, hospital-administered treatment courses. The pipeline of new antifungal therapies also remains thin.The World Health Organization (WHO) reported last year that only nine antifungal therapies were in clinical development to address WHO’s designated top four priority pathogens, including three in Phase 3 development. Although the Phase 3 trial for one of those candidates has since been terminated, another candidate, olorofim, developed by Shionogi and F2G, recently reported positive Phase 3 trial results. If approved, it could become the first antifungal treatment with a novel mechanism of action for that infection in more than 20 years.Earlier-stage research is also yielding promising advances. Researchers recently discovered that an antibiotic could target a priority fungal pathogen by switching it back from its invasive form to a less harmful form. This antibiotic could help existing antifungal drugs target dangerous fungal pathogens, allowing lower, less toxic doses of those drugs to be used.Together, these advances offer hope of a new generation of antifungal therapies.The future of fungal diagnosticsDiagnostic tools for critical fungal pathogens exist but with significant limitations, including invasive sampling requirements, long turnaround times, limited pathogen coverage, and inadequate sensitivity. Many also depend on sophisticated laboratories and highly trained staff, making them difficult to deploy in resource-constrained settings globally that bear a disproportionate burden of fungal disease.The MycoMEIA® assay developed at Johns Hopkins University and commercialized by Pearl Diagnostics represents a recent advance in antifungal diagnostics. The test received US Food and Drug Administration clearance in 2025. It uses a urine sample, which is much simpler and less invasive for patients than current options, and provides results in just a few hours.Another promising research development illustrates where fungal diagnostics may be headed next. Researchers at the Mayo Clinic and Zepto Life Technology recently reported early results from a new diagnostic platform that was able to identify 18 different fungal pathogens with a single test. While still in development, such approaches could not only streamline diagnosis for health care providers and patients, but also reduce costs and resource requirements, making them well-suited for resource-constrained settings.Closing the innovation gap Fungal diseases and antifungal resistance are attracting greater international attention, but the response still lags behind the scale and pace of this growing threat. Increased investment in the R&D of fit-for-purpose innovations, paired with stronger regulatory and health systems and global coordination, will be crucial to ensure new diagnostics and treatments emerge from the pipeline and reach the patients who need them most. 

Last week, Cepheid announced that its Xpert Hemorrhagic Fever Panel test has received Emergency Use Listing from the World Health Organization, enabling the technology to be deployed to help identify suspected cases and support outbreak containment in the rapidly growing outbreak of Bundibugyo Ebola in the Democratic Republic of the Congo. The molecular panel test detects and identifies Ebola virus (multiple strains, including Bundibugyo), Crimean-Congo hemorrhagic fever, Marburg virus, and Lassa virus, providing results in around an hour. The test runs on Cepheid’s GeneXpert diagnostic technology system, which is already widely used across Africa, helping enable rapid implementation. Cepheid previously donated an initial shipment of tests cleared by the US Food and Drug Administration through the Department of War, and the new listing now enables further deployment. A Phase 2 clinical trial led by the Serum Institute of India found that an experimental monoclonal antibody for dengue virus was safe, well-tolerated, and provided evidence of efficacy in a Phase 2 clinical trial. Dengue affects more than 200 million people each year and is a growing global threat due to the expanding range of the virus-carrying mosquito species. There are several licensed vaccines for dengue but no licensed antiviral drugs. The experimental antibody previously neutralized all four serotypes of dengue virus in animal studies and demonstrated safety in a Phase 1 trial in healthy adults. Researchers at the National Institutes of Health (NIH) have discovered rare antibodies that could inform the development of a therapeutic or preventive intervention for alpha-gal syndrome, an emerging tick-bite-associated condition behind a growing number of cases in the United States and globally, which can lead to allergic reactions to a molecule in red meat and other animal products. The discovery was made through malaria research—the scientists were originally trying to find antibodies that could target alpha-gal (a sugar molecule) on malaria parasites as a potential strategy for malaria prevention. In laboratory tests, the rare antibodies successfully hindered human allergic antibodies from binding to red meat allergens. There are currently no specific treatments or preventions for alpha-gal syndrome.