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Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease (SCD). But science does not stop.
Excision BioTherapeutics has so far released positive safety data from the first 3 participants living with HIV-1, with no evidence of vector shedding in sexual organ tissue.
New forms of the gene-editing tool could enable treatments for common diseases.
A life-saving gene therapy to treat sickle cell disease has been FDA approved. The first patient to receive the treatment came to Nashville to be part of a clinical trial years ago.
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