Following their recent FDA approval and more than a decade of development, biotech firm Bluebird Bio has announced the completion of its first cell collection for their Sickle Cell Disease gene therapy, Lyfgenia. 

Designed for SCD patients aged 12 and older with a history of vaso-occlusive events (VOEs), Lyfgenia modifies patients’ stem cells to produce HbAT87Q, a hemoglobin variant resistant to clumping. The treatment involves the collection and treatment of blood cell precursors, followed by reinfusion via a stem cell transplant – helping limit patient red blood cell sickling and potentially reducing the frequency and severity of VOEs.

The Phase 1/2 trial showed 94% of patients had no severe VOEs post-treatment, with Bluebird Bio planning to conduct Phase 3 and long-term follow-up trials to further evaluate the therapy’s safety and efficacy. 

The OxyDial team is thrilled to see this progress, and extends our congratulations to the Bluebird Bio team. We’re thrilled to see continued advancements and novel therapies for genetic disorders like SCD, and hope to see more innovative treatments that improve the lives of patients worldwide.

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