uniQure's AMT-162, a gene therapy for SOD1-ALS, has received the green light from the IDMC to proceed with enrollment in the second dose cohort of its Phase I/II EPISOD1 trial.
The FDA granted breakthrough therapy designation to STK-001 for Dravet syndrome, highlighting its potential to improve upon current treatments by restoring NaV1.1 protein levels.
Q32 Bio's bempikibart showed promise in Phase IIa trial for alopecia areata (AA), achieving a 16% mean reduction in SALT score at week 24 compared to placebo.
The FDA has agreed that existing Phase I/II data for uniQure's AMT-130, compared to external controls, can support a Biologics License Application for accelerated approval.
The market for gene and cell therapies targeting CNS disorders was valued at approximately USD 1 billion in 2021 and is projected to grow significantly by 2034.
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