Camp4 Therapeutics Corp. has received clearance to initiate a phase I/II clinical trial of CMP-002, a potential first-in-class therapeutic for SYNGAP1-related disorder.

Researchers from the Universities of Bologna and Torino recently presented their hematopoietic stem cell gene therapy (HSC-GT) strategy based on microglia-mediated delivery using a lentiviral vector encoding a secretable, cell-penetrating CDKL5 protein (Igκ-TATk-CDKL5).

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.