Financial PerformanceMarket is valuing the stock at just $260M, $62M below cash, indicating a potential undervaluation.
Medical Advancements100% of patients were VOE-free post-reni-cel infusion, compared to a mean of 4.9 severe VOEs/year in the 2 years before enrollment.
Strategic PartnershipsThe development of extrahepatic in vivo assets will be done in collaboration with Genevant, noting up to $238 million in upfront and contingent payments.