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.
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.
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.
