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Weekly Rundown: Once-weekly HIV pill clears Phase 3 trials. Microbubble-based genetic medicines, pharma deals, trial misses, and more led the news this week. DDN editorial team The currently FDA-approved oral pill from Gilead must be taken once daily. Welcome to the Weekly Rundown where the DDN editors cover this week's top biotech and pharma news. A once-weekly HIV pill just cleared two Phase 3 trials. Gilead Sciences and Merck announced this week that their investigational once-weekly single-tablet HIV regimen, islatravir/lenacapavir, met its primary efficacy endpoint at week 48 in both the ISLEND-1 and ISLEND-2 Phase 3 trials. The combination pairs Merck's islatravir, a next-generation nucleoside analog that blocks HIV replication through multiple mechanisms including reverse transcriptase translocation inhibition, with Gilead's lenacapavir, a first-in-class capsid inhibitor that disrupts HIV at multiple stages of its lifecycle. In ISLEND-1, the once-weekly tablet was non-inferior to Biktarvy, Gilead's current daily standard-of-care regimen, in virologically suppressed patients. In ISLEND-2, it was non-inferior to a broad range of daily antiretroviral regimens. Safety was comparable across arms with no new concerns identified. Both companies plan to file the Phase 3 data with regulatory authorities globally. If approved, islatravir/lenacapavir would become the first long-acting oral HIV treatment taken once weekly, a meaningful shift in a field where daily dosing has long been the norm and where adherence remains one of the most persistent barriers to sustained viral suppression. - Andrea Corona SonoThera raises $125M in series B for microbubble-based genetic medicines. The vast majority of gene therapies are delivered via viral vectors, which have safety risks that include liver toxicity and immune reactions. SonoThera is trying a different approach based on the invention from its Chief Scientific Officer, Steven Feinstein, who developed ultrasound contrast agents called microbubbles to image the heart. The company is now using these microbubbles to deliver genetic medicines to any organ of choice by using ultrasound to create a path and then subsequently pop the bubbles to open cells for the genetic material to enter. In a Series B funding round, SonoThera just banked $125 million from big players in the space like Otsuka Pharmaceutical, Johnson & Johnson, and Leaps by Bayer. The funding will help SonoThera move forward with its proprietary platform that supports gene editing and gene silencing with DNA and RNA therapies. Their lead programs target Duchenne muscular dystrophy - where their therapy will become the first to deliver the full-length dystrophin gene - and autosomal dominant polycystic kidney disease (ADPKD). "Despite remarkable scientific progress, many diseases remain beyond the reach of today's genetic medicines. We founded SonoThera to take a fundamentally different approach, with a platform designed to broaden the therapeutic possibilities of the field," said Kenneth Greenberg, cofounder and CEO at SonoThera in the press release. - Allison Whitten Mass photometry helps researchers investigate the large and dynamic molecular assemblies that drive HIV integration and influence antiviral drug development. GSK adds trio of lung cancer assets in $10.6B deal. GSK said on Tuesday that it has agreed to acquire Nuvalent for $10.6 billion, adding three lung cancer programs in a deal the drugmaker says fits its strategy of buying late-stage assets with validated targets and the potential to outperform existing therapies. Zidesamtinib (NVL-520), a highly selective ROS1 inhibitor, and neladalkib (NVL-655), a next-generation ALK inhibitor, are two late-stage, potential best-in-class candidates for treatment of non-small cell lung cancer (NSCLC). Both have received FDA breakthrough therapy and orphan drug designations and are under regulatory review with target decision dates in late 2026. The third asset, NVL-330, is a potential best-in-class HER2 inhibitor currently in Phase 1 trials for HER2-altered NSCLC. GSK said the lead drugs could launch as soon as 2026 if approved and have multi-blockbuster potential, citing pivotal data presented at the IASLC World Conference on Lung Cancer and the American Society of Clinical Oncology annual meeting showing durable responses, improved tolerability, and enhanced activity against brain metastases. The deal, one of GSK's largest in years, strengthens its lung cancer franchise as large drugmakers seek to replenish pipelines ahead of looming patent expiries. - Bree Foster Sanofi to discontinue late-stage autoimmune trial. On Wednesday, Sanofi announced the end of their Phase 3 MOBILIZE trial testing riliprubart, a complement C1 inhibitor, in chronic inflammatory demyelinating polyneuropathy - a rare neurological disease in which the immune system attacks the myelin sheath that surrounds neurons - after an independent data monitoring committee determined that the study is unlikely to prove efficacious. The news comes after encouraging Phase 2 results were announced in 2024. Sanofi stated in the news release that they will evaluate whether to continue other ongoing studies of the drug in the VITALIZE Phase 3 trial, which compares riliprubart to intravenous immunoglobulin treatment. The disappointing results add to a string of setbacks for Sanofi's drugs last year in psoriasis, chronic obstructive pulmonary disease (COPD), and multiple sclerosis. - Allison Whitten Dual Lassa-rabies vaccine shows early promise in human trial. Researchers at the University of Maryland School of Medicine have reported promising early clinical results for a single vaccine designed to protect against both Lassa fever and rabies, two deadly viral diseases that disproportionately affect parts of Africa. In a first-in-human trial published in Nature Medicine, 54 adults received two doses of the investigational vaccine, known as LASSARAB, or a licensed rabies vaccine, with interim data showing no serious adverse events and robust antibody responses against both Lassa virus and rabies. The study comes as Lassa fever - which has no licensed vaccine and is classified as a priority pathogen by the World Health Organization - continues to cause thousands of deaths each year in West Africa, while rabies remains almost universally fatal once symptoms appear. The candidate can also be freeze-dried, simplifying immunization efforts in regions with limited healthcare infrastructure. Further follow-up and larger trials are planned to assess the durability of protection and advance the vaccine toward broader clinical testing. - Bree Foster Fragile mRNA molecules depend on protective carriers to transport them into cells. Novo Nordisk discloses cyberattack involving clinical trial patient data. Novo Nordisk disclosed a cybersecurity incident this week in which unauthorized parties gained access to a limited number of internal IT systems and copied certain non-public data externally, including pseudonymized information tied to patients participating in some of its clinical trials. The exposed data categories may include patient ID numbers, sex, year of birth, biomarkers, health, immunogenicity data, and lifestyle factors such as body mass index (BMI) and smoking status. The company said the data is not directly linked to patients by name or other direct identifiers, and that identifying any individual from the exposed information would require access to additional data that was not part of the breach. Novo Nordisk has taken certain internal IT systems temporarily offline, launched an investigation with external cybersecurity experts, and is in contact with relevant authorities. Core business operations remain unaffected. The incident raises a pointed question for the industry: As clinical trial datasets grow richer and more interconnected, how well are the systems housing them protected? - Andrea Corona Add Drug Discovery News as a preferred Google source to see more of our trusted coverage.

SonoThera raises $125M to advance gene therapy. 11 June 2026 Key Takeaways * SonoThera raised an oversubscribed $125 million Series B financing round. * The funding was led by Vida Ventures with participation from major healthcare investors. * The company is developing a nonviral, ultrasound-mediated genetic medicine platform. SonoThera Inc. has secured a $125 million funding round to advance the development of a nonviral, ultrasound-mediated platform for gene therapy. This marked a major change for a significantly evolving industry, focusing on alternative delivery strategies and circumventing the multimillion-dollar manufacturing problems and safety concerns associated with the use of the more established viral vector therapies. A strong syndicate of life science investors and strategic pharma partners led the round. Lead investors on this round included Perceptive Advisors and ARCH Venture Partners, with greater commitments from existing investors, including F-Prime Capital, Venrock, Alexandria Venture Investments and previous seed investors. The round attracted strategic investments from the venture arms of some of the world's largest drug companies: Eli Lilly and Company, Bristol Myers Squibb and Johnson & Johnson Innovation, another indication of the major drug companies' interest in the clinical feasibility of the SonoThera platform. Solving the viral vector obstacles. For over a decade, the commercialisation of gene therapies has relied almost exclusively on modified viruses, primarily adeno-associated viruses (AAVs), to deliver therapeutic genetic material into a patient's cells. While highly effective at cellular entry, viral vectors carry steep operational and clinical baggage. The production of recombinant AAV1 at scale may be prohibitively slow and costly. AAV-based therapies in clinical settings risk provoking immune and inflammatory responses, precluding reapplication if therapeutic activity later declines. Viral vectors cannot transport large or complex sequences due to their limited payload capacity. SonoThera is disrupting this model with a fully non-viral delivery platform that pairs microbubble technology with focused ultrasound. Rather than employing a virus, the company loads a broad spectrum of genetic payloads: DNA, RNA and gene editing. complexes within microbubbles. Upon IV injection, these microbubbles flow benignly through the blood. Microbubbles and sound waves: the sonic edge. The real magic happens when clinicians apply localised ultrasound waves directly to the target organ. Once the microbubbles have entered the sound field, the incident ultrasound pressure wave causes the microbubbles to rapidly oscillate in size, or stable cavitate. This mechanical activity leads to a temporary, microscopic rupture of the walls of the blood vessels and the cell membranes around them, a process known as sonoporation. Doctors inject the genetic payload directly into the intended tissue at millimetre precision, avoiding harm to healthy organs. Stopping the ultrasound causes cell membranes to rapidly reseal, trapping the payload inside. "The promise of genetic medicine has long been throttled by the vehicles we use to deliver it," said Kenneth Greenberg, PhD, co-founder and CEO of SonoThera. "Our ultrasound-guided platform allows us to bypass the liver-heavy filtering that destroys most non-viral therapies, directing the treatment exactly where it is needed." Strategic roadmap: liver, kidney, and beyond. The $125 million influx of capital is earmarked to transition SonoThera from a preclinical discovery platform into a robust clinical-stage development enterprise. The primary objectives for the funds include: * IND-Enabling Studies: Finalising the required toxicology and safety profiles to submit Investigational New Drug applications to the U.S. Food and Drug Administration. * Clinical Trial Initiation: Launching Phase 1 human trials for its lead therapeutic programmes, which focus on genetic diseases impacting the kidneys and the heart. * Payload Expansion: Scaling engineering efforts to utilise the platform's massive cargo capacity for complex, multi-gene therapies and large-scale CRISPR gene-editing systems that cannot physically fit inside a virus. Because the system relies on standard diagnostic ultrasound hardware paired with proprietary transducers, it integrates seamlessly into existing hospital infrastructures, offering the long-term potential to deliver highly advanced gene therapies in standard outpatient clinical settings. 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