The promise of gene therapy is to cure diseases associated with faulty or missing genes.
Ideally, gene therapies address the root causes of disease with a single curative dose. If they can replace a lifetime of expensive maintenance treatments this may lead to cost savings in the long run. Yet, the high upfront costs, uncertainty surrounding long-term durability, and adverse events have led to some concerns among payers and regulators.
Pharmaceutical firms deploy multiple approaches to pursuing curative gene therapy, including:
Replacing a mutated gene that causes disease with a healthy copy;
Inactivating a mutated gene;
Introducing a new gene into the body.