Upbeat phase II news lately from Spyre Therapeutics Inc. in inflammatory bowel disease (IBD) perked not only the company’s stock but also wider interest in the buzzing space, where developers big and small are trying to tackle such chronic bedevilments as Crohn’s disease and ulcerative colitis (UC).

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.

As generic versions of its blockbuster drug Ofev (nintedanib) start to hit the market, Boehringer Ingelheim GmbH looks to have swerved the patent cliff, with European and Japanese regulators both approving a potential replacement, Jascayd (nerandomilast), this week. At its monthly meeting, the EMA’s Committee for Medicinal Products for Human Use also recommended Novartis AG’s Vijoice (alpelisib) be given conditional approval in the treatment of PIK3CA-related overgrowth spectrum disorders.

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.

Posting another win for its triple G agonist, Eli Lilly and Co. rolled out top-line phase III data from Triumph-1 showing that all doses of retatrutide met primary and key secondary endpoints for obesity, with participants losing on average between 19% and 28.3% of body weight and significantly improving their cardiovascular risk factors.

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.

Vincentage Pharma Co. Ltd.’s once-daily oral small-molecule GLP-1 receptor agonist, VCT-220, met the primary endpoints in a pivotal phase III trial in overweight or obese patients in China. Based on the top-line data, the Chengdu, China-based company plans to submit an NDA for chronic weight management to China’s National Medical Products Administration.

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.