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Gene Therapy

Note from the editor

Twenty years ago last September, 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. 

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. 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. 

Gene therapy's promise of a cure, meanwhile, can only be hinted at through studies lasting years, rather than decades. Whether other consequences to patient health emerge over time remains a question mark, too.

Science may also move quickly past gene replacement therapy to gene editing via CRISPR and other methods, an approach in which the unkowns are even greater and clinical work has just begun. 

Ned Pagliarulo Senior Editor

The gene therapy era has arrived. So have the challenges.

FDA, expecting a gene therapy boom, firms up policies

Approval of Novartis gene therapy a landmark, for its price as well as its promise

In rebuke of Novartis, FDA making 'statement for the world'

Pfizer lays out gene therapy aspirations

Roche hands $1B to Sarepta in major return to gene therapy deals

BioMarin sets gene therapy sights beyond hemophilia

Gene therapy costs, manufacturing keeping CBER head 'up at night'

Vertex sets sights on DMD gene therapy, joining crowded field

Nationwide Children's, a gene therapy leader, launches manufacturing spinout

Rare disease drugmakers turn to academia to advance gene therapy hopes

Gene editing companies oppose altering of embryos