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Serious side effects, including acute kidney injury and thyroid tumors, may occur in rare cases
The combination of biologic exclusivity, ODE, and PRV monetization means that a gene therapy approved for a rare pediatric condition can generate IP-protected commercial revenue plus a substantial cash payment from a PRV sale before a single patient is treated
Also, a favorable ADME and pharmacokinetic profile support strong systemic exposure and a low predicted efficacious dose in humans, and its solid developability profile marked by good stability lays the foundation as a therapeutically effective agent
References: Adrian S, Scherzinger A, Sanyal A, Lake JE, Falutz J, Dub MP, Stanley T, Grinspoon S, Mamputu JC, Marsolais C, Brown TT, Erlandson KM
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