A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene

计划入组 30 人 · 首次发布于 2026 年 2 月 25 日

Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy

计划入组 4 人 · 首次发布于 2022 年 8 月 18 日

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

计划入组 4 人 · 首次发布于 2020 年 10 月 9 日

P-MMUT-101

MMUT CRISPR/Cas9 gene therapy (AAV8, methylmalonic academia)

MMPT

FDA Clears IND for Gene Therapy Candidate MMA-101 to Treat Rare Metabolic Disorder Methylmalonic Acidemia

Genespire and SR-TIGET Demonstrate Durable Preclinical Efficacy of Liver-Directed Gene Therapy for Methylmalonic Acidemia

Genespire Presents Promising Dosing Data for First-in-Human Gene Therapy in Methylmalonic Acidemia