Aperture Therapeutics Inc. has advanced its matrix metalloproteinase-9 (MMP-9) antisense oligonucleotide (ASO) program, APRTX-003, for the treatment of amyotrophic lateral sclerosis (ALS). This first-in-class RNA-targeting approach program targets chronic neuroinflammation and neurodegeneration.

Researchers at Case Western Reserve University and their collaborators described the use of P7C3-A20, a compound that restores nicotinamide adenine dinucleotide (NAD+) homeostasis, as a potential strategy to reverse Alzheimer’s disease (AD) pathology in advanced Aβ- and tau-driven AD models.

In a recent publication in Molecular Therapy, researchers from Drexel University College of Medicine and UMass Chan Medical School presented a silence-and-replace gene therapy strategy aiming to address both the gain-of-toxicity and loss-of-function components of the disease hereditary spastic paraplegia (HSP).

SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by a wide range of symptoms, including intellectual disability, epilepsy, motor deficits and increased risk-taking behavior.

A review of 2025's noteworthy advances in medical research, including GLP-1 receptor agonists as anti-aging drugs, tumor-agnostic therapies and xenotransplants.