This study uses a type of gene therapy to address methylmalonic acidemia (MMA), a rare genetic defect that causes problems with digestion of fats and proteins ...
2022年9月20日 · Taken together, these findings support the development of hLB-001 that is currently in clinical trials in pediatric patients with severe forms ...
相关问题
How do you treat methylmalonic acidemia?
2024年2月23日 · hLB-001 is a liver-targeted, recombinant engineered adeno-associated viral (rAAV) vector utilizing the LK03 capsid (rAAV-LK03), designed to non- ...
This is a non-interventional long-term follow-up study of participants who have participated in LogicBio studies for the treatment of MMA and received hLB-001.
Taken together, these findings support the development of hLB-001 that is currently in clinical trials in pediatric patients with severe forms of MMA.
2023年12月12日 · Patients will be followed for a period of 15 years which includes the duration of time that the patient was in the treatment study.
2022年9月20日 · In mice with a humanized liver, treatment with a human homolog of mLB-001 resulted in site-specific genome editing and transgene expression in ...
A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations.
2022年9月20日 · LB-001 is a GeneRide AAV vector designed to provide durable therapeutic hepatic expression of MMUT protein. The expression of MMUT is driven by ...