BBrm-02

BBrm-02 is a proprietary, intrathecal (spinal) formulation of azithromycin developed as a **read-through therapeutic** for **spinal muscular atrophy (SMA)**. Unlike aminoglycoside antibiotics historically used for stop codon read-through therapy, BBrm-02 is a non-glycoside compound that shows superior efficacy in preclinical models and avoids the prohibitive toxicity associated with aminoglycosides. It enables drug-induced read-through of premature termination codons in the SMN2 gene, facilitating the expression of full-length, functional SMN protein in SMA cells and animal models. This mechanism is therapeutically relevant because all SMA patients have SMN2 and benefit from strategies that induce its functional protein expression. BBrm-02 is currently in preclinical development.[1][4][7][10][16][19][22][24]

Other names

intrathecal azithromycin