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

Pharmaceutical Technology

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. thalassemia in adult and paediatric patients. Findings showed that 89% of evaluable subjects attained transfusion independence (TI).

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

The Pharma Data

With the launch of Overland, we are creating a robust research and development engine in parallel with the significant growth and opportunity presented by Asia’s evolving biotech industry. A solution could be at hand with Overland Pharmaceuticals. Below is a roundup of some of this week’s interesting news.

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

XTalks

Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. Terence Eagleton, MB BS, Senior Medical Director at the global clinical research organization (CRO) Medpace.

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Innovative Companies Diving into The Neuro Market Ahead of 2021

The Pharma Data

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. which will remain a key shareholder. SciNeuro Pharmaceuticals. in a statement. . Libra Therapeutics.