hLB-001

BIOLOGICAL2 trials

Sponsors

LogicBio Therapeutics, Inc, Alexion Pharmaceuticals, Inc.

Conditions

Methylmalonic Acidemia

Phase 1

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

TerminatedNCT04581785

LogicBio Therapeutics, IncMethylmalonic Acidemia

Start: 2021-05-29End: 2023-01-10Updated: 2024-02-23

Unknown Phase

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

Active, not recruitingNCT05506254

Alexion Pharmaceuticals, Inc.Methylmalonic Acidemia

Start: 2022-07-15End: 2037-01-01Updated: 2026-02-05

Related Papers

First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study

Gene Therapy2026-03-30