FDA Grapples with Promise and Perils of Gene Therapy

Pharmaceutical Executive December 3, 2021
Jill Wechsler, Pharm Exec’s Washington Correspondent

The challenges in developing gene and cellular therapies have made headlines over the past year, as biotech companies have launched a wave of development programs despite ongoing concerns about treatment safety and efficacy plus unknown long-term effects. High price tags on these breakthrough treatments, moreover, threaten to block patient access to potentially life-saving cures and treatments. FDA scientists and reviewers are working to advance the field, with new guidances and advice for manufacturers on appropriate testing of treatments to minimize risks to patients and fill gaps in data.

New gene therapies for debilitating rare conditions stand to advance from an FDA collaboration with the National Institutes of Health (NIH) and a cadre of pharma companies, small biotechs and patient groups,...

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