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Source: agupdate.com
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"Half-matched" bone marrow transplants can cure sickle cell disease in 88% of patients
The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185). It was the second approval from FDA for a gene therapy for sickle cell disease.
Several companies are developing Crispr-based therapies for diseases including heart disease, cancer and rare genetic disorders