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.

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.

Eli Lilly and Co.’s buyout of Ventyx Biosciences Inc. for $1.2 billion at the start of the year brought to the forefront NLR family pyrin domain containing 3 (NLRP3) inhibitors on which a handful of developers have been working, and research in the space continues to roll out, as with the paper published March 26 in Nature that delved into mechanisms that rev up the NLRP3 inflammasome.

Nvision Imaging Technologies GmbH raised $55 million in a series B financing round led by Abbott Laboratories for its quantum-enhanced sensing platform, Polaris, which uses quantum technology to boost the MRI signal of sugar-based imaging agents to enable real-time measurement of metabolism on standard MRI systems. Sella Brosh, CEO and co-founder of Nvision, told BioWorld that the funds are extremely important as the company is scaling the Polaris systems to many more sites worldwide, which is a capital intensive process.

In a deal that could be worth up to $1 billion for Suzhou Siran Biotechnology Co. Ltd. (Siranbio), GSK plc licensed exclusive worldwide rights to the siRNA oligonucleotide SA-030, which has recently entered phase I trials for cardiometabolic disease.

Metis Techbio Co. Ltd. is seeking a potential HK$2.11 billion (US$270 million) raise through a stock sale May 13, marking the largest biotech raise on the Hong Kong Stock Exchange this year to date. Hangzhou, China-based Metis is an AI-based nanoparticle drug formulation and delivery-focused company. Synthetic lethality-based cancer drugmaker Impact Therapeutics Inc., of Shanghai, plans to debut on the same day with a US$117 million IPO.