Novartis’s ‘Poster Child’ Zolgensma Shows What Gene Therapy Can Do – And Its Limitations
Long-Term Data Brings Hope To Rare Disease Communities
The clinical development head of Novartis Gene Therapies talks about the progress for SMA patients more than seven years after treatment began, and hopes of boosting outcomes by treating pre-symptomatic patients.
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In this week's podcast edition of Five Must-Know Things: 10 big upcoming US biosimilar launches; Kisqali strikes gold in early adjuvant breast cancer; Zolgensma shows the promise and limitations of gene therapy; Chinese biotechs advancing multiple ADCs; and Scrip Asks about diversity, equity and inclusion in pharma.
New long-term data from Novartis’s Zolgensma and Roche’s Erysdi will help close the gap on Biogen’s market leader Spinraza, but the three therapies look set to develop distinct or even complementary roles as the market matures.
Investors balked at Sanofi plans to raise R&D spending and eat into profits, so CEO Paul Hudson and new R&D chief Houman Ashrafian need to convince doubters that its immunology focused plan will pay off.