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LB 001

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Drug Profile

LB 001

Alternative Names: hLB-001; LB-001; mLB-001

Latest Information Update: 05 May 2023

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At a glance

  • Originator LogicBio Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; Methylmalonyl CoA mutase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Methylmalonic acidaemia
  • 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

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