Researchers from Voyager Therapeutics Inc. presented preclinical activity data of VY-1706, a blood-brain barrier (BBB)-penetrant gene therapy comprising an adeno-associated virus serotype 9 capsid (AAV9-C9P39) vector encoding primary artificial microRNA (pri-amiRNA) consisting of short-interfering RNA (siRNA) targeting human microtubule-associated protein tau (MAPT) protein.

Remegen Co. Ltd. emerged as a surprise challenger in the generalized myasthenia gravis space, unveiling positive phase III data of its China-approved lupus drug, telitacicept (RCT-18; Tai’ai), in the rare autoimmune neuromuscular disorder at the 2025 American Academy of Neurology conference.

Compared to other forms of prevention, a unique issue for pandemic preparedness is that it is forever unclear what pathogen, exactly, the world needs to be prepared for. There are an estimated 300,000 viruses that infect mammals; add in birds, and the estimate grows to more than half a million. Some of those viruses are much greater threats than others.

Heart failure with preserved ejection fraction (HFpEF) is a disease in which the left ventricle (LV) of the heart shows impaired relaxation during cardiac diastole, often accompanied by structural and functional abnormalities.

Researchers from Sanofi SA reported the preclinical characterization of SAR-446159 (ABL-301), a bispecific antibody construct comprising an antibody targeting α-synuclein fused to an engineered antibody fragment that targets IGF-1R and functions as a blood-brain barrier (BBB) shuttle, known as the Grabody-B platform.

Sepsis-induced coagulopathy (SIC) is a complication of sepsis tied to high mortality in patients. Anticoagulation using a coagulation factor IIa and Xa dual inhibitor might have the potential to improve the treatment of this severe condition.

BDNF is the brain’s most abundant neurotrophic factor, playing a key role in neuronal survival and synaptic plasticity through the activation of the transcription factor CREB, which is essential for driving beneficial effects in neurons. CREB is downregulated in Parkinson’s disease, Huntington’s disease, frontotemporal dementia, Alzheimer’s disease and other neurodegenerative conditions.

The current treatment strategies for neurodegenerative diseases focus on targeting Aβ in Alzheimer’s disease (AD), α-synuclein aggregates in Parkinson’s disease (PD) and anti-tau therapies, which are primarily used in AD but are also being explored for PD. At the 2025 International Conference of Alzheimer’s & Parkinson’s Disease and Related Neurological Disorders, Aditya Iyer, senior RD scientist from Amyl Therapeutics Srl, presented data on an option which could potentially serve as a pan-amyloid therapeutic.

Esophageal basaloid squamous cell carcinoma (EBSCC) is a rare type of esophageal squamous cell cancer (ESCC) characterized by basaloid features and considered to be more aggressive than ESCC. RNA sequencing was performed from 20 pure EBSCC and 11 poorly differentiated ESCC samples; researchers identified chondroitin sulfate proteoglycan 4 (CSPG4) as a potential marker of EBSCC at the mRNA level, which was verified by immunohistochemistry in these same samples.