Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Peptide vaccine could slow return of pancreatic and colorectal cancer
A novel peptide vaccine developed by researchers at several US universities and Elicio Therapeutics showed encouraging results against pancreatic and colorectal cancer in a Phase 1 trial published in Nature Medicine. Cancer vaccines, which are gaining momentum as a cutting-edge approach in oncology, can be broadly grouped into those used for preventive and therapeutic purposes. The new peptide vaccine does not prevent cancer, but the team showed that it could help prevent recurrence. Twenty months after receiving the vaccine, two thirds of patients showed activated T cells against KRAS (Kirsten rat sarcoma virus) -mutated cells, which correlated with a relapse-free survival rate. “This gives hope for people with pancreatic and colorectal cancer who have been out of effective treatments when their disease returns,” said Eileen O’Reilly, a pancreatic cancer specialist at Memorial Sloan Kettering Cancer Center who co-led the study, in a press release. – Allison Whitten
UK Biobank identifies 1.5 billion genetic variants in landmark study
The UK Biobank (UKB) has completed whole-genome sequencing (WGS) for 490,640 participants, marking a major expansion of its already extensive dataset, which includes genetic, health, demographic, imaging, proteomic, and metabolomic data. Building on previous SNP genotyping and whole-exome sequencing (WES), the WGS effort identified approximately 1.5 billion genetic variants, revealing a far richer picture of genetic variation than WES or imputed arrays. This enhanced resolution enables deeper insights into disease mechanisms and improved opportunities for drug discovery, including target validation, safety assessment, patient stratification, and drug repurposing. This expanded dataset also enables the detection of rare structural variants and the evaluation of their phenotypic impact. For example, 15 individuals were found to have a 5,200-bp deletion in the MIP gene, which is strongly linked to cataracts. The scale and depth of this dataset will continue to fuel biomedical research and therapeutic development for years to come. – Bree Foster
FDA restricts gene therapy Skysona due to increased blood cancer risk
The FDA has tightened restrictions on bluebird bio’s gene therapy Skysona following a sharp rise in blood cancers among clinical trial participants. Approved in 2022 for boys aged four to 17 with the life-threatening neurological disorder cerebral adrenoleukodystrophy (CALD), the one-time lentiviral therapy will now be restricted to patients without a suitable stem cell donor. At approval, blood cancers such as myelodysplastic syndrome and acute myeloid leukemia had been reported in four percent of treated patients, but as of July 2025, the incidence had risen to 15 percent (10 of 67 participants), with one treatment-related death. Cancers emerged between 14 months and 10 years after therapy and were linked to vector insertions in proto-oncogenes like MECOM and PRDM16, as well as potential contributions from the conditioning regimens used. Despite these risks, the FDA has acknowledged the therapy’s potential to slow CALD progression in patients with no other options but warned that further cases of malignancy remain possible. – Bree Foster
Bayer bets big on Kumquat’s KRAS inhibitor
In a deal worth up to $1.3 billion, Bayer has partnered with Kumquat Biosciences to gain exclusive rights to their drug targeting the G12D mutation in the KRAS gene. This mutation is known to drive tumor growth in the lung, pancreas, and colon. The announcement detailed that Kumquat will initiate and complete the Phase 1a study, and Bayer will then complete development and commercial activities. Kumquat’s investigational drug remains behind a KRAS G12D drug, zoldonrasib, from Revolution Medicines, who recently released results in April from their Phase 1 study. That data showed that zoldonrasib achieved an objective response rate of 61 percent and a disease control rate of 89 percent. – Allison Whitten
Experimental antibody offers hope against sepsis in preclinical trials
Researchers at the University of Virginia (UVA) and the University of Michigan have developed a monoclonal antibody that may offer a breakthrough treatment for sepsis and other life-threatening inflammatory conditions. Over 48.9 million cases of sepsis are reported globally each year, leading to approximately 11 million deaths. In preclinical studies, the antibody blocked the immune overreaction that drives sepsis, commonly referred to as a cytokine storm, without suppressing overall immune function. The therapy also showed promise in treating conditions such as acute respiratory distress syndrome and ischemia-reperfusion injury. Alongside the antibody, the team is advancing a companion diagnostic tool, PEdELISA, that enables rapid monitoring of immune activity. With $800,000 in new funding from Virginia Catalyst, clinical trials are set to begin at UVA Health and Virginia Commonwealth University. – Andrea Corona
FDA launches PreCheck program to strengthen US drug manufacturing and reduce import dependence
The FDA has unveiled a new initiative aimed at boosting domestic pharmaceutical production by streamlining regulatory oversight and accelerating the construction of US-based drug manufacturing facilities. The move responds to growing concerns over the country’s dependence on foreign sources for active pharmaceutical ingredients (API), with only 11 percent of API manufacturers for FDA-approved drugs based in the US. The program, created under Executive Order 14293, introduces a two-phase process offering early agency engagement and faster review timelines to encourage local production. It arrives amid rising trade tensions, as President Trump has continued to threaten tariffs on imported drugs to further incentivize onshoring. A public meeting on the program is scheduled for September 30 at the FDA's Maryland campus. – Andrea Corona
