BBP-812

BBP-812 is an investigational intravenous adeno-associated virus serotype 9 (AAV9) gene therapy developed for the treatment of Canavan disease, a rare and fatal neurodevelopmental disorder. The therapy works by delivering functional copies of the ASPA gene throughout the body and into the brain, aiming to correct the underlying genetic defect responsible for Canavan disease. In clinical trials, BBP-812 has demonstrated rapid and sustained reductions in N-acetylaspartate (NAA) levels—a key biomarker elevated in Canavan disease—across urine, cerebrospinal fluid, and brain tissue. These reductions are associated with improvements in myelination on MRI scans and progressive gains in motor function milestones such as head control, sitting upright, reaching for objects, and visual tracking. The safety profile of BBP-812 has been generally consistent with other AAV9 gene therapies. The drug has received Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation (FDA/EMA), Rare Pediatric Disease Designation (RPDD), and Fast Track status from regulatory agencies[1][3][4][5][6][7].

Other names

BBP 812BBP812BBP-812