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Jun 14, 2026
In vivo base editing corrected the CHD3 p.R1025W mutation in a mouse model of Snijders Blok–Campeau syndrome, restoring protein levels and rescuing behavioural abnormalities. Intrathecal AAV delivery in nonhuman primates supported translational feasibility.
Jun 08, 2026
Canaccord Genuity initiated Beam Therapeutics with a buy rating and $74 price target, highlighting near-term catalysts. Beam reached an agreement with the FDA on an accelerated approval pathway for its BEAM-302 AATD therapy, which showed early clinical proof-of-concept for in vivo base editing. The company plans a BLA for risto-cel and a pivotal plan for BEAM-302 in early 2026.
May 12, 2026
A review in Genes & Diseases says robust non-clinical safety assessment is critical for CRISPR-based gene therapies. It highlights genotoxic, delivery, and immunological risks and recommends risk-based development programs.
