AAV-LSP-MMUT

**AAV-LSP-MMUT** is an experimental **AAV-mediated gene therapy** designed for **isolated methylmalonic acidemia caused by MMUT mutations**. Based on available preclinical references, it is a **liver-directed recombinant adeno-associated viral vector** carrying the **human methylmalonyl-CoA mutase gene** under a **liver-specific promoter**, with the goal of restoring hepatic MMUT enzyme activity, lowering methylmalonic acid levels, and improving metabolic control in MMUT-deficient methylmalonic acidemia. Publicly available information specifically naming AAV-LSP-MMUT appears limited to preclinical conference material, including work presented in a mouse model of MMA and studies evaluating co-administration with Selecta's ImmTOR platform to reduce anti-capsid immune responses and potentially enable repeat dosing. The exact clinical product identity, serotype, sponsor ownership, and whether it ultimately advanced under a different development code are not clearly established from the accessible sources.