Living Medicines With Curative Intent: Small N’s Go Viral

21 November 2017

Article from written by Bruce Booth. Photo ©AP Photo/Elaine Thompson.

“The field of gene and cell therapy has witnessed an unprecedented few months, almost unimaginable only a few years ago. It’s worth pausing to reflect on some of the recent highlights:

  • FDA approval of two chimeric antigen receptor (CAR) T-cell therapy products, Kymriah from Novartis and Yescarta from Kite/Gilead, that have repeatedly shown profound efficacy
  • Positive unanimous recommendation from a group of FDA advisors (and patients) for Luxturna, an adeno-associated virus (AAV2) gene therapy from Spark Therapeutics, for a rare form of blindness, delivered via subretinal injection
  • Continued positive results from ex vivo lentiviral therapy in cerebral adrenoleukodystrophy (CALD) from bluebird bio’s STARBEAM trial, published last month in the New England Journal of Medicine
  • Striking improvements on survival and motor function from a single dose of AveXis’ AAV9 encoding SMA1 in children with spinal muscular atrophy, according to another NEJM paper published this month

These are indeed exciting times – and the pace of innovation seems incredible. Living medicines based on cell and gene therapy have moved into mainstream on the promise of compelling clinical data, and there are literally dozens of companies, big and small alike, advancing the next generation of this genre of therapies.  It’s worth pausing to reflect on how far and fast we’ve come.”

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