Tag: Pharmaceuticals & Drug Trials
2023 was a big year for CRISPR-based gene editing but challenges remain
2023 was an important year for patients with sickle cell disease. Prior to CRISPR, the only cure for the life-long ailment was a bone marrow transplant, which is notoriously dangerous and costly. This month, the FDA approved Vertex’s “Casgevy,” a CRISPR-based therapy for the treatment of sickle cell disease in…
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Researchers developed a gene-editing technology that reduces ‘bad’ cholesterol
In a trial run by Verve Therapeutics, a Cambridge–based biotech company, researchers discovered that a single infusion of a gene-editing treatment called VERVE-101 was able to reduce cholesterol levels in patients. This treatment was tested in individuals with hereditary conditions that made them susceptible to developing clogged arteries and heart…
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