Drug Profile

BGT OTCD

Alternative Names: AAVLK03hOTC; BGT-OTCD; ssAAV-LK03.hAAT.hcoOTC

Latest Information Update: 11 Mar 2026

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At a glance

Originator Childrens Medical Research Institute; University College London
Developer Bloomsbury Genetic Therapies; University College London
Class Gene therapies
Mechanism of Action Gene transference; Ornithine carbamoyltransferase expression stimulants

Orphan Drug Status

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

Yes - Ornithine carbamoyltransferase deficiency disease
New Molecular Entity No

Highest Development Phases

Phase I/II Ornithine carbamoyltransferase deficiency disease

Most Recent Events

No Data Available

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