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Bluebird Bio

gene therapy
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory

Skysona: Gene Therapy for Cerebral Adrenoleukodystrophy

by Tanya von Reuss September 19, 2022September 19, 2022

A single dose of the elivaldogene autotemcel will cure a deadly disease.

blood
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory

Zynteglo: Gene Therapy for Beta-thalassemia

by Mark Gubar September 13, 2022September 13, 2022

One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.

multiple-myeloma
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory

Tecvayli: New Drug for Multiple Myeloma

by Mark Gubar August 24, 2022September 8, 2022

Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.

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