Paige Thompson

Paige Thompson asked about specific benchmarks for target engagement in the upcoming Q1 siRNA readouts, whether current doses are therapeutic, and what levels would indicate potential for higher doses.

Answer

President and Chief Executive Officer Doug Ingram and Executive VP, Chief Scientific Officer, and Head of Research and Development Louise Rodino-Klapac highlighted safety and muscle concentration as key metrics. They expect to see consistency with preclinical data, supporting continued dose escalation and efficient knockdown due to the siRNA mechanism and αvβ6 targeting ligand.

Paige Thompson asked about the upcoming siRNA readouts in Q1, specifically what benchmarks investors should consider for target engagement, whether the initial doses are expected to be therapeutic, and what levels would indicate potential for higher, more effective dosing.

Answer

Doug Ingram, President and Chief Executive Officer, highlighted safety and muscle concentration as the most significant factors to observe in early readouts, suggesting that strong performance in these areas at low doses would imply subsequent knockdown and functional benefits. Louise Rodino-Klapac, Executive VP, Chief Scientific Officer, and Head of Research and Development, added that Sarepta's unique integrin receptor approach for muscle-directed therapies, combined with siRNA potency, supports the potential for best-in-class knockdown and safe dose escalation, aligning with preclinical data.