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Dive into the Biopharma Space

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July 2023: BlueBird Bio Clinical Trial Safety Insight

Category: Gene Therapy Safety

Opportunity: Improved lentiviral gene therapy drug development and genetic testing

Company: BlueBird Bio (See Key Info below)

Product/Asset: SKYSONA (approved rare disease gene therapy)

Issue: Myelodysplastic syndrome (MDS) in rare disease patients from lentiviral integration into proto-oncogenes

Latest trigger event for Insight: Bluebird Bio July 2023 Corporate slide deck

Target Companies: Vendors, CMOs, and biopharma companies with products and services in cell imaging, gene therapy and cell therapy, esp. Using lenti vectors


In July 2023 Bluebird Bio corporate slide deck (See slide below), 5 patients have now reported developing MDS after being treated with SKYSONA. This is a known safety risk. The SKYSONA package insert has a Black Box warning and further details regarding this known safety risk (See below). As of approval on Sept 16, 2023 there were 3 known cases, as reported in the drug label. Now that number has increased to 5. The FDA has taken no additional action because of this as far as we are aware. The package insert provided detailed info about monitoring patients treated with SKYSONA for MDS. However, this issue underscores the need for improved lentiviral vectors for gene therapy and improved methods to detect integration events and clonal expansion of blood cells that can be used to help R&D teams to develop improved vectors and earlier diagnosis of MDS.

See below for more info regarding Bluebird Bio and SKYSONA.

About Bluebird Bio:

Company Type: Public Company

Market Cap (7/18/23): $330,900,000

Location: Somerville, Massachusetts

Cash (Q1 2023): $318,300,000

Burn rate/quarter: $-78,570,000

Technology Focus: Gene therapy and Cell therapy/CAR-T

Latest stage asset: Approved gene and cell therapies

Indications: Beta thalassemia, Cerebral Adrenoleukodystrophy, Multiple Myeloma, & Sickle Cell Disease

Next readout: PDUFA December 20, 2023

Key employees: Subscribe to learn more!


SKYSONA from Bluebird bio, was recently approved on September 16th, 2023 as a one-time gene therapy for treating early, active cerebral adrenoleukodystrophy (CALD). CALD is a genetic disease caused by mutations in the ABCD1 gene that lead to the buildup of very long chain fatty acids (VLCFAs) in the brain. VLCFAs may destroy nerve cells and cause damage to the brain. SKYSONA is made specifically for each patient (patient’s own blood stem cells (i.e., autologous)) and works by adding functional copies of the ABCD1 gene into cells. SKYSONA therefore helps the body break down the VLCFAs, which slows the progression of damage to the brain and slows the decline in neurologic function.


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Article History:

Originally posted 7/18/23 (RB)


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