CRISPR/Cas9-based therapies are relatively new, with Intellia’s investigational treatment being the first systemically administered treatment studied in humans. In non-human preclinical studies (e.g., rodents and monkeys), the treatment was evaluated for safe and effective dosing ranges, and Intellia has carefully considered the data from these studies to maximize safety in the clinical trial. In addition, … Continue reading Is CRISPR/Cas9 safe?