- PTC Therapeutics' investigational drug PTC518 demonstrated statistically significant reductions in Huntingtin protein levels in Phase 2 PIVOT-HD study, with 23-39% decreases observed across different dosages over 12 months.
- IDEAYA Biosciences advances multiple oncology programs, with over 230 patients enrolled in potential registration-enabling trial for darovasertib in metastatic uveal melanoma, targeting key readouts by end of 2025.
- uniQure's gene therapy trial has shown promising results, with treated patients exhibiting a significant reduction in the rate of Huntington's disease progression.
- Spinogenix's SPG601 demonstrated a significant reduction in high-frequency gamma band activity, an EEG abnormality associated with Fragile X syndrome (FXS).