Fabry disease is a lysosomal storage disease tied to the X chromosome and caused by pathogenic variants in the GLA gene encoding galactosidase A. It is characterized by progressive accumulation of galactosidase A substrates, including Gb3 and lyso-Gb3, mainly in the kidney, heart and nervous system.

WAVES, an algorithm designed to extract menstrual-cycle metrics from physiological signals such as basal body temperature, which oscillates with sex hormones, shows how different parameters change with age and helps determine whether each person maintains a stable individual pattern or personal footprint. A study based on data from 5,674 cycles from 753 women demonstrates through this tool that age is associated with higher temperatures, shorter cycles, and greater irregularity. In addition, several metrics show within-person stability, suggesting they could serve as personalized health markers.

A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.

Korro Bio Inc. has announced the selection of KRRO-111 as a development candidate for the treatment of alpha-1 antitrypsin deficiency (AATD), a genetic disorder most commonly caused by a single missense mutation in SERPINA1.

Type 2 diabetes is marked by insulin resistance coupled with insufficient insulin secretion due to early β-cell dysfunction and progressive loss of β-cell mass. Pdx1 and MafA, critical for maintaining β-cell function, are progressively reduced under metabolic stress and in patients, driving disease progression. Researchers at the University of Pittsburgh have reported efficacy data demonstrating successful pancreatic delivery of GPX-002, an AAV-Pdx1/MafA construct, in HFD mice.

Harbour Biomed has released preclinical data for LET-003, a next-generation ACVR2A/2B-targeted monoclonal antibody with potential for the treatment of obesity. Activin receptors ACVR2A and ACVR2B play critical roles in regulating muscle-fat metabolic homeostasis.

Fractyl Health Inc. has received clinical trial application authorization in the Netherlands to initiate a first-in-human phase I/II study of RJVA-001, the first clinical candidate from the company’s Rejuva Smart GLP-1 gene therapy platform.