BBrm

BBrm (also known as BBrm02 or BBrm2) is a proprietary intrathecal formulation of the repurposed macrolide antibiotic azithromycin, developed by Bioblast Pharma (originally licensed from Tel Aviv University) for the treatment of spinal muscular atrophy (SMA). BBrm acts as a translational read-through agent that targets the eukaryotic ribosome to bypass premature termination codons (nonsense mutations) or stop codons, such as the stop codon located at exon 8 of the SMN2 (SMN-del7) transcript. By promoting ribosomal read-through, BBrm increases the expression of full-length, functional survival motor neuron (SMN) protein. Preclinical studies demonstrated proof of efficacy in SMA mouse models, showing increased SMN expression in the brain, spinal cord, and muscle, along with improvements in motor function and survival. Although clinical development was planned, the program was discontinued following Bioblast Pharma's corporate restructuring and merger with Enlivex Therapeutics.

Other names

azithromycinintrathecal azithromycin