Objective: The article examines the objective, findings, and clinical context described in FDA Updates Potency Rules for Cell and Gene Therapies. Approach: Life
The FDA’s final guidance, published November 2, replaces its 2011 guidance on potency testing for cell and gene therapies.
The guidance recommends a science- and risk-based Potency Assurance Strategy spanning raw materials, manufacturing processes, and laboratory testing.
The framework integrates potency assurance throughout development, production, commercialization, and postapproval monitoring rather than relying primarily on finished-product testing.
The guidance addresses potency assessment challenges involving short shelf lives, viral vector therapies, and patient-specific treatments.
The recommendations allow manufacturers to select controls and analytical methods based on product characteristics and risks; they do not establish a new approval pathway.
Guinevere Lageveen-Kammeijer studies glycosylation and its changes in diseases such as cancer, using CE-MS-based glyco(proteo)omics.
Her group aims to reveal previously hidden molecular details and extend glycomic analysis toward individual cells.
She expects single-cell glycomics to reveal cellular heterogeneity and sees separation science, including CE-MS, as important for resolving molecular complexity.
She emphasizes that greater sensitivity and more data do not automatically produce better science; measurements and computational patterns require validation and biological interpretation.
A CE experiment using prostate-specific antigen as a model glycoprotein revealed CE's ability to separate glycoforms with differently linked sialic acids and redirected her PhD toward prostate cancer
Objective: Identify seasonal chemical patterns in Scottish honey using phenolic-focused, untargeted ion mobility liquid chromatography–mass spectrometry. Approa
Untargeted ion mobility liquid chromatography–mass spectrometry distinguished seasonal chemical profiles in 39 Scottish and commercial honey samples.
Paired spring and summer samples from approximately nine Aberdeenshire apiaries enabled a more direct assessment of seasonal variation.
Spring and summer markers included galangin, quercetin, sebacic acid, tricoumaroyl spermidine, suberic acid, and luteolin-7-glucuronide.
Heather honey markers included 3-phenyllactic acid, abscisic acid, 4-hydroxycinnamaldehyde, and kaempferol-related compounds.
The exploratory models were intended for marker discovery, and independent validation and broader sampling are needed before routine authentication use.
Rising cancer incidence, expanding biomarker menus, and staffing shortages are increasing IHC workload and straining pathology laboratories.
David Newell identifies order intake, instrument loading, and unloading as key IHC workflow failure points.
Batch-based ordering, batching habits, and inconsistent instrument use can delay starts and create avoidable instrument idle time.
Newell reports that extending daily IHC staining-area coverage from 8 to 9.5 hours and changing workflows increased capacity from 120 to 180 slides per day without new equipment.
The article recommends end-to-end workflow review and cross-functional collaboration to identify bottlenecks and plan for future laboratory needs.
Objective: To explain why high diagnostic accuracy alone does not establish the clinical value of AI in digital pathology and to propose a framework for evaluat
High diagnostic accuracy alone does not establish the clinical value of AI in digital pathology.
The authors propose a six-stage framework spanning tissue preparation, image acquisition, model performance, laboratory workflow, clinical decisions, and patient outcomes.
Differences in tissue processing, scanners, and laboratory procedures can affect image quality and AI performance, making external validation and local testing important.
In a study involving 28 pathology experts, incorrect AI recommendations overturned previously correct assessments in approximately 7% of consultations.
The conceptual framework has not been validated prospectively and calls for ongoing monitoring of AI performance after implementation.
Objective: The article examines the objective, findings, and clinical context described in Lonza to Acquire Five US CDMO Sites from Merck KGaA. Approach: Transa
Objective: The article examines the objective, findings, and clinical context described in FDA Investigates Malignancy Risk Linked to Zolgensma Gene Therapy. Ap
Objective: Assess liver gene correction and safety after LNP-delivered base editing in a patient with alpha-1 antitrypsin deficiency (AATD). Approach: Study and
A 66-year-old man with Pi*ZZ alpha-1 antitrypsin deficiency received a single intravenous infusion of the investigational base-editing therapy YOLT-202.
Ten weeks after treatment, liver biopsy showed correction of 54% of targeted SERPINA1 alleles by Sanger sequencing and 57% by Illumina sequencing.
Testing found no detectable unintended editing at nearby DNA positions or predicted off-target sites.
Circulating alpha-1 antitrypsin rose from 0.15 g/L before treatment to 0.7 g/L at 10 weeks, and functional testing confirmed elastase inhibition.
Larger studies and longer follow-up are needed to determine whether the molecular improvements provide lasting protection against liver and lung disease.
PSS risk strata were based on the most severe organ dysfunction measurements during the first 12 hours after PICU admission.
Hydrocortisone began at median 4.7 hours in high-risk and 4.1 hours in low-risk patients, so some classification measurements may have followed treatment initiation.
The authors’ letter notes that treatment-related changes in organ dysfunction could affect PSS classification, but whether reclassification occurred or influenced estimates is uncertain.
The propensity model included 12-hour PRISM III scores and refractory shock, covariates that may include posttreatment information and may not be suitable baseline confounders.
The letter proposes analyses using shared pretreatment eligibility for an admission-time decision or pretreatment scores among eligible, untreated children at 12 hours.
Objective: To assess changes in early and overall neonatal mortality in Flint, Michigan, after the January 2024 launch of the Rx Kids cash-transfer program. App
Rx Kids launched in Flint in January 2024, providing a $1,500 prenatal payment and $500 monthly payments during an infant’s first year; participation reached 98%.
From 2021 through 2024, Flint’s early and overall neonatal mortality rates were 7.9 and 10.2 per 1,000 live births, significantly higher than rates elsewhere in Michigan.
In 2025, Flint’s early neonatal mortality rate fell 74% to 2.0 per 1,000 live births, while the rate elsewhere in Michigan was 3.3.
Flint’s overall neonatal mortality rate fell 50% to 5.1 per 1,000 live births in 2025, while the rate elsewhere in Michigan remained relatively stable at 3.9.
The study found no statistically significant geographic differences in either mortality measure in 2025, but provisional records and small event counts limit interpretation.
Objective: Assess the 52-week safety and clinical outcomes of motugivatrep 0.3% ophthalmic suspension, used alone or with existing dry eye therapies, in adults
In this 52-week, open-label, single-arm phase 3 study, 162 adults with dry eye disease received motugivatrep 0.3% 4 times daily alone or with existing therapies.
Treatment-emergent adverse events occurred in 45% of patients, and treatment-related adverse events occurred in 15%; most treatment-related events were mild.
No severe or serious treatment-related adverse events or deaths were reported, and 3 serious adverse events were considered unrelated to treatment.
Patient-reported symptoms and corneal staining improved during treatment, while tear break-up time increased; tear secretion decreased from baseline.
The uncontrolled, unmasked design and enrollment of mostly Japanese patients with relatively mild corneal staining limit attribution of improvements and generalizability.
Objective: The article reports the FDA approval of pimicotinib for adults with symptomatic TGCT when surgery may worsen functional limitations or cause severe m
The medical examiner classified Floyd’s death as a homicide caused by cardiopulmonary arrest complicating law enforcement subdual, restraint, and neck compression.
The autopsy found no life-threatening injuries or distinctive traumatic evidence of airway or neck damage, but those absences do not exclude functional breathing impairment.
Floyd’s hospital-admission blood contained fentanyl at 11 ng/mL and methamphetamine at 19 ng/mL, alongside substantial cardiovascular disease.
Toxicology and cardiac findings were plausible contributors to cardiopulmonary arrest, but they do not establish that drugs and disease fully explain the death.
Floyd’s ability to speak indicates that his airway was not completely obstructed while he spoke, but does not establish adequate breathing or oxygenation.
Objective: To assess the diagnostic performance of metagenomic and targeted next-generation sequencing (NGS) of bronchoalveolar lavage fluid in patients with su
In a retrospective study of 941 patients with suspected lower respiratory tract infections, both metagenomic and targeted NGS had greater diagnostic yield than conventional microbiological testing.
Metagenomic NGS sensitivity was 90.6%, compared with 73.4% for targeted NGS, but the methods were evaluated in separate patient groups.
Targeted NGS identified 54 cases of Mycobacterium tuberculosis, including 42 missed by conventional testing, and detected Pneumocystis jirovecii when silver staining was negative.
After targeted NGS results, 22.6% of patients required antimicrobial treatment escalation, and 66.2% showed clinical improvement, similar to 66.7% with initially appropriate treatment.
Both sequencing approaches had low specificity, and the retrospective, single-center study could not establish that sequencing-guided treatment caused improved outcomes.