A single dose of the elivaldogene autotemcel will cure a deadly disease.
Bluebird Bio
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Zynteglo: Gene Therapy for Beta-thalassemia
One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Tecvayli: New Drug for Multiple Myeloma
Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.