Satellos Bioscience Inc. recently presented preclinical data for the novel oral small-molecule AP2-associated protein kinase 1 (AAK1) inhibitor SAT-3247, being developed for the treatment of Duchenne muscular dystrophy and other muscle degenerative diseases, at the Muscular Dystrophy Association conference.
Since microglia play a critical role in resolving amyloid pathology and it has been previously demonstrated that microglia function can be induced by TREM2 activation, TREM2 agonism has been considered a promising novel therapeutic approach to delay or prevent the progression of Alzheimer’s disease.
Amyotrophic lateral sclerosis (ALS) is a fatal disease characterized by the death of motor neurons in conjunction with neuroinflammation and deposition of protein aggregates, such as TDP-43, in these neurons and oligodendrocytes. Progranulin is a growth factor that is essential for neuron survival and a regulator of anti-inflammatory responses.
At the 2024 Alzheimer’s & Parkinson’s Diseases Conference this week, Michel Goedert from the MRC Laboratory of Molecular Biology in Cambridge gave the Donald L. Price Memorial Plenary Lecture entitled, “Cryo-EM structures of disease filaments from human brains.” Donald Price was particularly well-known for his work on plaques and tangles in nonhuman aged primate brains and for the development of several animal models of neurodegenerative diseases.
Overall, the story of HIV is one of astounding success. But to declare victory, it will be necessary to develop a vaccine. The opening session of the 31st Conference on Retroviruses and Opportunistic Infections (CROI) 2024 looked back to the failures but also the advances in research, all the steps that over the years brought the basic science knowledge that could bring an HIV vaccine in the future. This year, the former director of the Viral Pathogenesis Laboratory at the NIAID Vaccine Research Center, Barney Graham, was named for the Bernard Field Lecture, where he presented “Modern vaccinology: a legacy of HIV research.”
Researchers from Bugworks Research Inc. presented the discovery and preclinical evaluation of a novel potent and selective cyclin-dependent kinase 7 (CDK7) inhibitor, BWC-5044, in development for the treatment of cancer.
Charcot-Marie-Tooth (CMT) disease is a common neuropathy affecting the peripheral nerves. Compared to Type I, type 2 CMT presents as a slower and more progressive disease tied to axonal dysfunction, accompanied by distal muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.