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In episode 119 of Cell & Gene The Podcast, Host Erin Harris talks to Dr. Norman Putzki, Global Head Clinical Development, Novartis, about the FDA approval of Itvisma, now the only gene replacement therapy approved for children, adolescents, and adults with spinal muscular atrophy (SMA). Dr. Putzki walks us through the six-year development journey behind the STEER and STRENGTH Phase 3 programs. And we explore what the expanded age-range label means for patients who were previously left behind, why intrathecal, fixed-dose AAV delivery represents a pivotal advance for safety, efficacy, and scalability. He details how the Itvisma program is informing Novartis’ broader gene therapy strategy across neuromuscular and CNS diseases, and more.
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