FTO is an α-ketoglutarate-dependent RNA demethylase that regulates N6-methyladenosine (m6A), one of the most abundant epitranscriptomic modifications in mammalian mRNA. Emerging evidence suggests that aberrant m6A signaling may contribute to neuronal dysfunction and neurodegeneration. Researchers at the University of Barcelona and collaborators described the discovery and preclinical characterization of a new series of FTO inhibitors designed to target neurodegenerative disease by modulating m6A RNA demethylation.

At the Alzheimer’s Association International Conference, researchers from Voyager Therapeutics Inc. presented preclinical efficacy data for VY-1706, a blood-brain barrier-penetrant AAV9 gene therapy designed to reduce tau levels in models of Alzheimer’s disease (AD).

Certain cancers that contain organized clusters of immune cells known as tertiary lymphoid structures (TLS) do not respond to treatment as well as expected. Even though they have TLS that support the elimination of cancer cells, they remain resistant to immunotherapy. γ-Aminobutyric acid (GABA), better known as the brain’s major inhibitory neurotransmitter, may play a role in this lack of response by acting as an immunoregulatory metabolite, according to a study led by scientists at Sorbonne Université.

Newronika SpA received CE mark certification for its latest adaptive deep brain stimulation (aDBS) system, which includes the integration of Webbiobank, its proprietary cloud-based neural data platform.

Tuesday brought what was arguably the most anticipated presentation of the 2026 Alzheimer’s Association International Conference when Catherine Mummery, head of novel therapeutics at University College London’s Dementia Research Center, presented data from the phase II Celia trial of tau-lowering antisense oligonucleotide diranersen (BIIB-080, Biogen Inc.). Based on both clinical and biomarker data, “Celia establishes a proof of concept,” Mummery said, that reducing tau may slow the progression of Alzheimer’s disease.

Aggregated α-synuclein (α-Syn) is encoded by the SNCA gene and is one of the key pathological proteins linked to Parkinson’s disease. However, it is still not clear whether or not α-Syn originating in the periphery has a role in the pathology in the brain.

Despite multiple analgesics on the market, current therapeutics often fail at achieving complete pain relief and are usually tied to undesired adverse effects. Recent studies have pointed to adenosine A3 receptor (A3AR) as a promising target for pain management.

Duchenne muscular dystrophy (DMD) is a progressive, genetic (X-linked recessive) neuromuscular disorder caused by mutations to the DMD gene, resulting in the dysfunction or absence of the dystrophin protein. In DMD, muscle regeneration initially depends on the proliferation and differentiation of muscle satellite cells (MuSCs), but their regenerative capacity progressively declines, making repair inefficient and contributing to muscle dysfunction.

Niagen Bioscience Inc.’s proprietary lead small-molecule drug candidate, NB-4168, has been awarded European orphan drug designation and U.S. rare pediatric disease designation for the treatment of ataxia telangiectasia.