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

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia 0 genotypes. thalassemia in adult and paediatric patients. Topic sponsors are not involved in the creation of editorial content.

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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. .

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

The Pharma Data

Our focus on partnering to accelerate patient access to innovative medicines underscores our commitment to advancing breakthroughs in biomedicine such as antibody drug conjugates, cell therapy, RNAi and more, Ed Zhang, company co-founder, chief operating and business officer, said in a statement.

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

XTalks

According to Dr. Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases.