Drug Profile

Research programme: gene editing therapeutics - Genetix Biotherapeutics

Latest Information Update: 03 Oct 2025

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At a glance

Orphan Drug Status

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

No
New Molecular Entity Yes

18 Sep 2025 bluebird bio is now called Genetix Biotherapeutics
28 Oct 2024 No recent reports of development identified for preclinical development in Unspecified in USA
31 Oct 2022 Preclinical development is ongoing in USA (bluebird bio pipeline, October 2022)

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