Mucopolysaccharidosis I therapeutic - bluebird bio
Alternative Names: HSC LVV-gene-modified stem cells - bluebird bioLatest Information Update: 28 Jun 2023
At a glance
- Originator bluebird bio
- Class Gene therapies; Stem cell therapies
- Mechanism of Action Gene transference; Iduronidase 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
- No development reported Mucopolysaccharidosis I