Pipeline zoals vastgelegd in de Atlas op Aug 25, 2026. Bedrijven passen hun pipelines regelmatig aan — controleer altijd de eigen materialen en regelgevingsdossiers van het bedrijf.
In één alinea
bluebird bio developed approved gene therapies for beta thalassemia, sickle cell disease and a rare brain disorder, using viruses to add working genes rather than editing existing ones. Its scientific record is strong and its commercial history difficult: extremely expensive one-time treatments for small populations turned out to be a hard business, and the company withdrew from the European market and was eventually taken private.
bluebird bio developed lentiviral gene-addition therapies including Zynteglo (beta thalassemia), Lyfgenia (sickle cell disease) and Skysona (cerebral adrenoleukodystrophy). Despite approvals, commercial difficulties led to withdrawal from the European market and, in 2025, acquisition and take-private. Its history is the field's clearest illustration that regulatory success and commercial viability are separate problems.
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Dit profiel is educatieve informatie samengesteld uit openbare bekendmakingen. Het is geen aanbeveling om een effect te kopen, verkopen of aan te houden, en het is geen mening over de vooruitzichten van het bedrijf. Biotechnologie is sterk speculatief en de meeste klinische programma's mislukken.
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Written by The CRISPR Atlas editorial teamLast updated Aug 25, 2026
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