FDA Approval, Genetic Disease, In-Vivo and Protein

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FDA Approval

Genetic Disease

In-Vivo

Protein

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

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

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.

Drugs

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Clinical Research Informer

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

Advances in neuroscience drug discovery

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