Arrhythmogenic cardiomyopathy (ACM) is a severe genetic cardiac disorder caused by mutations in some desmosomal genes. The most frequently affected gene in patients with ACM is PKP2, the loss of which provokes desmosomal instability that leads to activation of downstream disease processes ultimately resulting in life-threatening ventricular arrhythmia and heart failure.

Epigenetic silencing could prevent the production of proteins that cause pathologies. CHARM (coupled histone tail for autoinhibition release of methyltransferase), a DNA methylation-based editor, suppressed transcription of prion proteins in the brains of mice.

Rznomics Inc. has received clinical trial notification (CTN) from Australia’s Therapeutic Goods Administration (TGA) for the initiation of a phase I/IIa trial evaluating RZ-004, a gene therapeutic candidate for autosomal dominant retinitis pigmentosa with rhodopsin mutation.

Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.

Interius Biotherapeutics Inc. has been granted Human Research Ethics Committee (HREC) approval and clinical trial notification clearance by Australia’s Therapeutic Goods Administration (TGA) to begin a first-in-human trial of INT-2104, its lead in vivo CAR candidate for treatment of B-cell malignancies.

Anew Medical Inc. has announced plans to advance its Klotho gene therapy program for neurodegenerative disorders. Initial data suggest that maintaining elevated levels of Klotho in the body significantly contributes to longer, healthier life spans, while individuals with depleted or lower than normal levels of Klotho are more susceptible to neurodegenerative disorders.

Exsilio Therapeutics emerged from stealth mode on June 25, 2024, with $82 million from a series A financing that was co-led by Novartis Venture Fund and Delos Capital. The company plans to use naturally occurring, mobile genetic elements to integrate therapeutic genes at a defined location in the genome, making it safer than random integration, which can cause tumor formation.

Spur Therapeutics Ltd., formerly Freeline Therapeutics, has announced new data from its GBA1 Parkinson’s disease research program. In a subset of Parkinson’s disease patients with mutations in the GBA1 gene, such mutations lead to a deficiency of the glucocerebrosidase (GCase) enzyme and the accumulation of harmful substrates.

4D Molecular Therapeutics Inc. has obtained IND clearance by the FDA for 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with geographic atrophy.