Drug Profile
LB 001
Alternative Names: hLB-001; LB-001; mLB-001Latest Information Update: 05 May 2023
Price :
$50
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At a glance
- Originator LogicBio Therapeutics
- Class Gene therapies
- Mechanism of Action Gene transference; Methylmalonyl-CoA mutase replacements
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Phase I/II Methylmalonic acidaemia
Most Recent Events
- 10 Jan 2023 LogicBio Therapeutics and Alexion terminate the first-in-human phase I/II SUNRISE trial for Methylmalonic academia (In infants, In children) in USA (IV), due to low likelihood of clinical benefit in treated participants (NCT04581785)
- 03 Jan 2023 CANbridge Pharmaceuticals completed adeno-associated virus (AAV) sL65 based capsid technology transfer from LogicBio Therapeutics
- 16 Nov 2022 LogicBio Therapeutics has been acquired by Alexion AstraZeneca Rare Disease