LX-1001

Lexeo Therapeutics' LX1001 gene therapy shows dose-dependent APOE2 expression increase and reduced tau biomarkers in APOE4 homozygote Alzheimer's patients.

Mivelsiran, an siRNA targeting APP, demonstrates durable reduction of APP metabolites in CSF and is being evaluated for cerebral amyloid angiopathy (CAA).

RiboX receives FDA clearance for its Investigational New Drug (IND) application, marking the first such clearance for a circular RNA therapy.

A JAMA Neurology study reveals that deferiprone, while successfully reducing brain iron accumulation, unexpectedly accelerated cognitive decline in early Alzheimer's disease patients.

The FDA is creating a more accessible regulatory environment for cell and gene therapy companies, according to executives at BioFuture 2024.