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Curative cell and gene therapies and healthcare system disruption SHARES Introduction Significant scientific advances have created a rich clinical research pipeline, with innovative and potentially curative cell and gene therapies already available (eg, Kymriah ® for acute lymphoblastic leukaemia, Luxturna ® for inherited retinal disease and Zolgensma ® for spinal muscular atrophy). Over the next 10 years, more of these therapies are expected to enter the market for other disease areas and larger patient populations. Potentially curative cell and gene therapies represent a key shift in patient care and will have considerable impact across the patient journey, affecting the healthcare system as we know it and the key stakeholders involved. Their launch is also met with significant debate over the uncertainty of outcomes’ durability and safety over the longer term; and the impact of treatment prices on affordability as more treatments enter the market. We draw from the chara

Charles riverUnited statesCity ofUnited kingdomArtes haderiTanna hillelLev gerlovinRebecca steeleNeil turnerTaxus cardiumHealth working papers noLife sciences practiceAmerican society of geneCharles river associatesLife sciences practice at charles river associatesGeneral practice