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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease.

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

XTalks

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Vertex, CRISPR prep filing for gene-editing blood disorder therapy

pharmaphorum

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. HbF produces normal, healthy red blood cells, rather than the misshapen cells produced by faulty haemoglobin in the two disorders.

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Advances in neuroscience drug discovery

Drug Discovery World

Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery.

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