Drug Profile

Research programme: gene therapies - bluebird bio

Latest Information Update: 28 Sep 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 No
Available For Licensing Yes

28 Sep 2025 No recent reports of development identified for preclinical development in Inborn genetic disorders in USA (Parenteral)
04 Aug 2021 Gene therapies are available for licensing as of 04 Aug 2021. https://www.bluebirdbio.com/about-us/collaborations
04 Aug 2021 Preclinical trials in Inborn genetic disorders in USA (Parenteral) (bluebird-bio pipeline, August 2021)

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