Spark Therapeutics Inc. has presented a proprietary adeno-associated viral (AAV) vector expressing an artificial miRNA targeting human α-synuclein (α-Syn) mRNA. Accumulation of misfolded and insoluble α-Syn causes neuronal toxicity in preclinical models and has been identified as the underlying cause of synucleinopathies.

Previous work uncovered the role of the complement system in neuroinflammation and synaptic loss in neurodegenerative disorders such as Alzheimer’s disease (AD). The third component, C3, central in all complement activation pathways, has been proposed as a potential therapeutic target in AD.

During the first oral sessions on Translational Drug Discovery at the 19th Alzheimer’s & Parkinson’s Diseases (ADPD) Conference, Beka Solomon from Tel Aviv University presented her work on the therapeutic potential of bone-marrow-derived microglia in Alzheimer’s disease (AD). “After working more than 30 years in immunotherapy, I decided to totally change the subject of the work,” Solomon told the audience.