Will a gene therapy that edits multiple genes using CRISPR be approved by the FDA before 2030?
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Atualizado há 7 minutos
26/01/24, 00:13 UTC1/01/30, 07:59 UTC
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In late 2023 the United States Food and Drug Administration (FDA) approved Casgevy, the first ever CRISPR-based gene therapy.
- Casgevy is a gene therapy for sickle cell disease, which is a hereditary disease caused by a mutation in a single gene (i.e. it’s monogenic).
- To count as a YES, a “gene therapy that edits multiple genes using CRISPR” should modify two or more genes in a patient’s genome by using some version of CRISPR (e.g. Cas9, an alternative protein).
- The gene therapy must be for humans.
- Will the FDA approve a multigenic gene therapy using CRISPR technology before 2030?
