Welcome to the second iteration of the Weekly Rundown! In this new column, the DDN editorial team will bring you short summaries of the latest industry news every Friday. We’ll keep you up to date on everything you need to know in drug discovery, development, and beyond.
If there’s anything in particular you’d like to see in this new column, we welcome your thoughts and feedback at: editor@drugdiscoverynews.com
NIH warns scientists not to mention mRNA in grants
Scientists said that representatives from the National Institutes of Health (NIH) have urged them not to reference mRNA vaccine technology in their grant applications. The NIH is one of the world's leading sources of research funding for basic biomedical research, with current grants supporting the investigation of mRNA vaccines for preventing and treating diseases like HIV and cancer. Recently, an mRNA vaccine for pancreatic cancer showed positive results in an NIH-supported Phase 1 trial (1). These attacks on mRNA vaccine technology follow its successful deployment in the COVID-19 vaccines, which, despite their life-saving effectiveness, became the focus of various rumors and conspiracy theories. In 2023, the Nobel Prize in Physiology or Medicine was awarded to Katalin Karikó and Drew Weissman for developing mRNA vaccines against COVID-19. – Dika Ojiakor
A big week for H5N1 avian influenza research
The avian influenza virus (H5N1) has rapidly spread among wild birds and dairy cattle in the US and has infected some humans. This week, scientists made significant advances in research aimed at understanding and developing new treatments against the virus.
- Researchers showed that the flu antiviral baloxavir (Xofluza) decreased viral levels in mice infected with H5N1 to a greater extent than the standard treatment, oseltamivir (Tamiflu). However, neither of these commonly used FDA-approved antivirals was effective in treating severe H5N1 infections, according to a new study (2).
- Prior exposure to older flu strains could help prime the immune system to produce antibodies against H5N1, researchers reported in a new study (3). The finding suggests that younger individuals may benefit more from vaccination than older individuals if infected with H5N1.
- Researchers at Cidara Therapeutics have developed CD388, a new antiviral drug with the potential to offer broad protection against both seasonal and pandemic flu strains, including H5N1 (4). CD388 is a stable variant of zanamivir (Relenza), an FDA-approved neuraminidase inhibitor, which was engineered to extend the drug’s half-life. In preclinical models, the drug enhanced zanamivir’s effectiveness against influenza A and B. CD388 is currently being tested in a Phase 2b trial to evaluate its potential for universal flu prevention during the 2024-2025 flu season. – Dika Ojiakor
Allogeneic T cell immunotherapy reduces a severe complication
Allogeneic bone marrow transplants commonly lead to graft versus host disease (GvHD), a life-threatening condition in which the transplanted donor cells attack the recipient’s tissue. When the donor is not related to the recipient, GvHD occurs in around 60 to 80 percent of patients. Orca Bio announced that their Phase 3 trial of allogeneic T cell immunotherapy in blood cancers more than doubled the rate at which patients survived to the one-year endpoint without developing chronic GvHD, compared to standard allogeneic stem cell transplants (78 percent versus 38 percent, respectively). In addition, overall survival with their so-named Orca-T therapy was 94 percent compared to 83 percent with the standard treatment. – Allison Whitten
Recall of embolization devices linked to four deaths
The FDA recalled Medtronic’s Pipeline Vantage embolization devices, which are used to divert blood flow and treat brain aneurysms. Following 17 injuries and four patient deaths, the FDA identified the recall as the “most serious type” and called for the immediate removal of unused devices from the 027 model and updated instructions for the 021 model. The FDA noted that there have been reports that the flexible, braided tube of the device did not successfully attach to the blood vessel wall and led to thrombosis, stroke, and death. The adverse events happened more frequently in females under 45-years-old. – Allison Whitten
FDA-approved drug shows promise in aggressive childhood brain cancer
High-grade gliomas are aggressive brain cancers that can be difficult to treat. They are also the leading cause of cancer-related deaths in children. Researchers recently showed that the FDA-approved cancer drug avapritinib shrunk tumors in mouse models and in three out of eight patients with high-grade gliomas that have mutations in the PDGFRA gene (5). The eight pediatric and young adult patients tolerated the drug well, and these positive initial data led to a Phase 1/2 clinical trial assessing avapritinib in children with advanced relapsed or refractory tumors with PDGFRA mutations. – Stephanie DeMarco
Advances and setbacks in Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an inherited and progressive condition where muscles degenerate due to mutations in the dystrophin gene. While there were a number of exciting results pertaining to DMD presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference this past week, the field also learned the sad news of a patient's death.
- Avidity Biosciences reported in topline data from their Phase 1/2 EXPLORE44 trial that their antibody oligonucleotide conjugate (AOC) drug, del-zota, led to an increase in dystrophin protein production and exon skipping, a decrease in creatine kinase levels, and positive safety and tolerability outcomes in people with DMD amenable to exon 44 skipping.
- Dyne Therapeutics announced long-term data from their ongoing Phase 1/2 DELIVER trial in people with DMD who are amenable to exon 51 skipping. Their drug, DYNE-251, had a positive safety profile and led to improvement over baseline in some functional endpoints such as stride velocity and an ambulatory assessment.
- Regenxbio recently released positive interim data from their AFFINITY DUCHENNE trial of their gene therapy RGX-202. Their therapy continues to exhibit strong safety data with no reported serious adverse events.
- Sarepta Therapeutics released a statement that a young man with DMD had died after receiving treatment with the company’s gene therapy Elevidys. His death, due to acute liver failure, is the first one reported out of the more than 800 people who have been treated with this drug. So far, Elevidys is the only gene therapy approved for DMD. – Stephanie DeMarco
Eight countries may run out of HIV medications soon
The World Health Organization (WHO) announced in a press conference that eight countries are at risk of running out of HIV medications in the coming months as a result of the Trump administration’s pause on USAID funding, including Haiti, Kenya, Lesotho, South Sudan, Burkina Faso, Mali, Nigeria and Ukraine. Patients with HIV require these life-saving drugs to control their infection, and the New York Times recently reported that women and children in South Sudan have already died as a result of no longer being able to access medication. Director-General Tedros Adhanom Ghebreyesus said that the ultimate consequence could be over 10 million new HIV cases and three million HIV-related deaths. On Tuesday, US District Judge Theodore D. Chuang ordered the reinstatement of USAID electronic systems and stated that shutdown of the agency was likely unconstitutional. – Allison Whitten
A Thursday evening approval for Alnylam
The RNAi therapeutics company, Alnylam announced that their drug Amvuttra (vutrisiran) received FDA approval for treating people with the cardiac form of the rare disease transthyretin-mediated amyloidosis (ATTR-CM). It acts by knocking down expression of the transthyretin protein, which in ATTR-CM becomes misfolded and accumulates in multiple organs, leading to fatal cardiomyopathy. Phase 3 trial results published in the New England Journal of Medicine last August showed that the drug reduced the risk of cardiovascular events and death from any cause (6). Amvuttra had already been approved to treat the inherited neurological form of the disease. – Stephanie DeMarco
References
- Sethna, Z. et al. RNA neoantigen vaccines prime long-lived CD8+ T cells in pancreatic cancer. Nature (2025).
- Jones, J.C. et al. Baloxavir improves disease outcomes in mice after intranasal or ocular infection with Influenza A virus H5N1-contaminated cow’s milk. Nat Microbiol (2025).
- Garretson, T.A. et al. Immune history shapes human antibody responses to H5N1 influenza viruses. Nat Med (2025).
- Döhrmann, S. et al. Drug-Fc conjugate CD388 targets influenza virus neuraminidase and is broadly protective in mice. Nat Microbiol (2025).
- Mayr, L. et al. Effective targeting of PDGFRA-altered high-grade glioma with avapritinib. Cancer Cell (2025).
- Fontana, M. et al. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med 392, 33-44 (2025).
