An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood, a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model.

Opus Genetics Inc. has entered a strategic partnership with the Global RDH12 Alliance to advance Opus’ gene therapy program for patients with vision loss due to retinol dehydrogenase 12 (RDH12) gene mutations.

Voyager Therapeutics Inc. has expanded its Alzheimer’s disease (AD) pipeline with the addition of a wholly owned program that modulates the expression of apolipoprotein E (APOE). Using a proprietary intravenous-delivered, blood-brain barrier (BBB)-penetrant Tracer capsid, the product delivers a bifunctional payload.

The U.S. FDA has cleared Aavantgarde Bio Srl’s IND application for AAVB-039, the company’s gene therapy program for Stargardt disease, the most common inherited form of macular degeneration.

The U.S. FDA has granted orphan drug designation to Klotho Neurosciences Inc.’s secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202 or s-KL-AAV.myo) for the treatment of amyotrophic lateral sclerosis (ALS).

Solid Biosciences Inc. has announced approvals of its IND application and CTA by the U.S. FDA and Health Canada, respectively, for SGT-501, a novel gene therapy approach for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a highly malignant, arrhythmogenic channelopathy caused by mutations in the RYR2 and CASQ2 genes.

Arrhythmogenic cardiomyopathy is an inherited disorder that typically manifests in people younger than 40 years and for which only palliative treatments exist. For advanced cases, heart transplantation is the only therapeutic option.

Cellular atlases and omics studies, such as genomics, transcriptomics and proteomics, have become key tools for identifying the diversity of all the elements that make up the cardiovascular system. These approaches help scientists understand how cells, genes and molecules function and interact in both healthy and diseased conditions, revealing critical points where targeted interventions could not only relieve symptoms but potentially reverse the underlying pathology at its origin.

The U.S. FDA has cleared Rocket Pharmaceuticals Inc.’s IND application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-associated dilated cardiomyopathy (BAG3-DCM), a severe form of heart failure.

Cure Rare Disease has successfully completed a pre-IND meeting with the FDA regarding its investigational gene therapy program for limb-girdle muscular dystrophy type 2I/R9 (LGMDR9).