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Not yet in human trials, but worth tracking since a domestic product would sidestep the import costs discussed in Topic 4:
Intas Pharmaceuticals (Centre for Cell & Gene Therapy) — preclinical AAV and lentiviral vector development targeting SMA, run alongside parallel programs for Duchenne Muscular Dystrophy, Hemophilia A/B, beta-thalassemia, and Sickle Cell Disease. This is a platform-level investment rather than a single-asset program, meaning Intas is building manufacturing and vector-design capability that could plausibly support multiple gene therapies over time, SMA included.
Why this matters for cost: the single biggest driver of Zolgensma’s price (Topic 4) is that it is imported and manufactured under Novartis’s global cost structure. India’s growing domestic cell-and-gene-therapy manufacturing base — already responsible for the CDSCO-approved CAR-T product NexCAR19 (developed by ImmunoACT and IIT Bombay) — is the infrastructure analysts point to when predicting that an indigenous AAV9-SMN1 gene therapy could eventually reach CTRI-registered trials at a fraction of the imported price, similar to how Natco’s generic Risdiplam undercut Roche’s branded Evrysdi by roughly 97%.
No CTRI registration exists yet for an Intas SMA product — this thread is for tracking announcements, not for treating anything here as an active trial.
References:
Recent developments in gene therapy research in India — https://www.ias.ac.in/article/fulltext/jbsc/049/0037
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