X-linked Alport syndrome is an inherited kidney disease caused by pathogenic mutations in the COL4A5 gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable COL4A5 restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting.

Circio Holding ASA and Aavigen GmbH have entered a research collaboration focused on developing circVec-enhanced AAV vectors engineered for targeted, low-dose gene therapies for heart diseases.

AAV-based therapies for Duchenne muscular dystrophy (DMD) have shown efficacy, but have limitations such as poor delivery to target tissues and toxicity associated with the vector. Gemma Biotherapeutics Inc. has developed a gene therapy candidate, GB-703, which uses a new myotropic, integrin-binding AAV capsid containing a codon-optimized, deimmunized hybrid payload.

Defects in antigen presentation lead to resistance to cancer immunotherapy, where type I conventional dendritic cells (cDC1s) are crucial drivers of antitumor immunity and their presence is tied to favorable responses and better outcomes. Intratumoral delivery of adenoviral vector, Ad5-PIB, encoding PU.1, IRF8 and BATF3 reprograms tumor cells into cDC1-like antigen-presenting cells and has shown synergy with immune checkpoint blockade (ICB) therapy at exerting antitumor immunity. Asgard Therapeutics AB has developed AT-108, a lead candidate developed for durable efficacy.

Sangamo Therapeutics Inc. discussed gene regulation approaches for neurodegenerative diseases when presenting findings on their clinical candidate ST-506 for the treatment of prion disease.

Researchers at the University of London and collaborating institutions have developed a gene and cell therapy approach that enables sustained systemic frataxin protein delivery, improving motor performance and tissue pathology, and supporting a promising translational strategy for long-term disease stabilization in Friedreich’s ataxia patients.

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.

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.

The American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL) have announced the public launch of CGTxchange, an AI-enhanced clearinghouse and marketplace built to help reactivate cell and gene therapy programs that have been shelved despite strong scientific and clinical evidence.

Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.