Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing.

Idefine, The Kleefstra Syndrome Foundation, has established a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to treat Kleefstra syndrome, a rare neurodevelopmental disorder caused by changes or loss of the EHMT1 gene, which plays a critical role in brain development and function.

Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development.

Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.

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.

New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies.

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.

New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies.

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.