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- Deaths of two Chinese children receiving CRISPR therapy spark safety debateTwo children in China died after receiving experimental CRISPR-based gene therapies, according to a recent report. The fatalities have unsettled researchers in the United States who are developing…
- Ultragenyx's Angelman syndrome drug fails in Phase 3 trial, showing no benefitUltragenyx announced that its experimental therapy GTX-102 for Angelman syndrome did not demonstrate any benefit compared with a sham control in a large Phase 3 trial. The drug had previously show…