Data from the All Women clinical trial, which looked at the Allegra transcatheter aortic valve replacement (TAVR) system compared to balloon-expandable valves (BEV) in women with aortic stenosis, showed that Allegra delivered lower mean gradients and less prosthesis-patient mismatch than BEVs. The data presented at the EuroPCR conference in Paris was the first randomized head-to-head comparison of different TAVR platforms conducted exclusively in women.

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.

Antibody-drug conjugates (ADCs) with a dual payload, which deliver two distinct cytotoxic agents via a single antibody, are emerging therapeutics developed to address the limitations of classic ADCs. Primelink Biotherapeutics (Shenzhen) Co. Ltd. recently presented data for their dual-payload ADCs, highlighting PLB-015, which carries a TOP1 inhibitor and an ATR inhibitor with an anti-HER2 antibody and is designed to inhibit the DNA damage response activated by cancer cells when harmed.

Netherton syndrome is a rare disease caused by loss of activity of the lympho-epithelial Kazal-type-related inhibitor (LEKTI) protein, which in turn is caused by mutations in its encoding gene, SPINK5. This deficiency leads to the triggering of the kallikrein (KLK) signaling cascade resulting in skin barrier dysfunction, inflammation and atopy. At the recent Society for Investigative Dermatology meeting, Biocryst Pharmaceuticals Inc. presented early data on BCX-17725, a KLK5/KLK14 inhibitor fusion protein developed to restore LEKTI functioning in patients with Netherton syndrome.

Transforming acidic coiled-coil-containing protein 3 (TACC3) is a core member of multiprotein complexes that regulate microtubule- and centrosome-related processes. Its aberrant expression is found in several cancer types with poor prognosis, thus highlighting it as a candidate therapeutic target. Researchers from Beijing Konruns Pharmaceutical Co. Ltd. have presented data for KC-1101, a TACC3 inhibitor for treating aggressive cancers with centrosome amplification.

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.

Werner syndrome helicase (WRN), belonging to the RecQ helicase family, represents a synthetic lethal vulnerability in MSI-H cancers, providing a strong rationale for therapeutic inhibition. Researchers from Simcere Zaiming Pharmaceutical Co. Ltd. reported the discovery and preclinical characterization of ZMS-4084, a novel WRN inhibitor.

Increasing evidence supports metabotropic glutamate mGlu7 receptor as a promising target in psychiatric disorders. Neurosterix Pharma Sarl has presented data on NTX-819, a potential first-in-class, potent and selective negative allosteric modulator (NAM) of mGlu7. NTX-819 was tested in vivo in rats using behavioral tests that are relevant to disorders such as obsessive-compulsive disorder (OCD), agitation and mania/psychosis.