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FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. 0 genotypes. An ex-vivo lentiviral vector (LVV) gene therapy, Zynteglo’s every dose is made by genetically modifying the bone marrow stem cells of the patient to produce functional beta-globin.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Global Roundup: Overland Pharma Launches in China to Provide Access to Breakthrough Therapies

The Pharma Data

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

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