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Gene Therapy

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Note from the editor

Twenty-one years ago, Jesse Gelsinger died after receiving an experimental gene therapy in a clinical trial seeking a cure for the rare disease he inherited.

His death reverberated throughout the scientific community, spurring investigations of the University of Pennsyvlania center that conducted the study. Gene therapy development, which had been charging ahead on the promise of replacing faulty genes, slowed considerably.

Two decades later, the technology is again at the forefront of biomedical research, catalyzed by advances in safer delivery of genes to cells.

Two genetic fixes for inherited diseases have won U.S. approval, one for a type of childhood blindness and the other for a fatal neuromuscular condition. By one count, nearly 300 experimental gene-based therapies are in clinical testing, including one aimed at OTC deficinecy, the disease from which Gelsinger suffered.

By 2025, the Food and Drug Administration expects it will be clearing 10 to 20 cell and gene therapy products a year.

Along with progress have come several major setbacks, though. Recently, two cancer cases in a study of a sickle cell gene therapy have renewed safety concerns. Last fall, three patients with a rare neuromuscular disorder died due to liver complications in a trial of another gene therapy, a tragedy that brought with it echoes of Gelsinger's death.

Just a few months before, meanwhile, a hemophilia gene therapy that was widely expected to win U.S. approval was rejected by the FDA, which asked for more data from developer BioMarin Pharmaceutical.

Gene therapy's effects on a healthcare system designed around chronic treatment are also just beginning to be worked out. The two medicines now approved cost $850,000 and $2.1 million per patient, respectively, and those advancing through development are expected to command similar price tags.

Ned Pagliarulo Senior Editor

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Inside the Rapidly Changing World of Gene Therapy

Five years from now, the Food and Drug Administration expects it will be clearing 10 to 20 cell and gene therapy products a year. Yet while the arrival of gene therapy's moment may be clear, its effects on a healthcare system designed around chronic treatment are not.

included in this trendline
  • Trends and challenges in the world of gene therapy today
  • Roche hands $1B to Sarepta in major return to gene therapy deals
  • Why gene editing companies oppose altering of embryos
Our Trendlines go deep on the biggest trends. These special reports, produced by our team of award-winning journalists, help business leaders understand how their industries are changing.
Davide Savenije Editor-in-Chief at Industry Dive.