Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia

Summary

The SUNRISE trial is a first-in-human (FIH), open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and preliminary efficacy of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. hLB-001 is a liver-targeted, recombinant engineered adeno-associated viral (rAAV) vector utilizing the LK03 capsid (rAAV-LK03), designed to non-disruptively integrate the human methylmalonyl-CoA mutase gene at the albumin locus. The trial is expected to enroll pediatric patients with ages ranging from 6 months to 12 years, initially starting with 3 to 12 year-old patients and then adding patients aged 6 months to 2 years.

Timeline

Start

2021-05-29

Primary completion

2023-01-10

Completion

2023-01-10

Outcome

Outcome not reported

Stopped: “Due to low likelihood of clinical benefit in treated participants.”

Publications

• Bedoyan JK, Morgan T, Sun A, Li H, Gruskin D, Payton M, Chereau F, Swenson ES, Lin Q, Kay MA, Vockley J. First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study. Gene Ther. 2026 May;33(3):345-357. doi: 10.1038/s41434-026-00609-1. Epub 2026 Mar 30.

Drugs

Indications