Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.
Multiple sclerosis (MS) is characterized by persistent inflammation, primarily driven by CNS-resident immune cells like microglia, particularly in the progressive stages. The purinergic P2X7 receptor (P2X7R) is widely expressed on immune cells and is elevated on reactive astrocytes and microglia of MS lesions.
Following a stroke, multiple immune cell types contribute to the neuroinflammatory response, with T cells being among the most consistently associated with both the initial and secondary phases of brain injury.
Blackfinbio Ltd. has obtained IND clearance from the FDA for its novel AAV gene therapy, BFB-101, for hereditary spastic paraplegia type 47 (SPG47), which is caused by changes in the AP4B1 gene. A phase I/II trial will be conducted in the U.S. at Boston Children’s Hospital.
Research of the neuroimmune mechanisms involved in stress-related fear revealed how astrocytes interact with neurons in the amygdala. The study, led by Harvard scientists, also unveiled that this interaction recruited monocytes to the meninges during chronic stress and showed how psychedelic compounds reversed monocyte accumulation in the meninges and reduced fear behavior.
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder affecting approximately 1 in 3,500-5,000 newborn males worldwide. It is characterized by progressive degeneration of skeletal, cardiac and smooth muscle, leading to early mortality. A team of researchers from the Technion Israel Institute of Technology has developed a novel approach to combat fibrosis in DMD.