Enthusiasm for gene therapy remains high this week at the annual J.P. Morgan Healthcare Conference, despite uneven clinical trials and uncertainty over pricing, according to published reports.
The approach, which is still in the experimental stages, treats disease by replacing a broken gene with a functioning one. In best case scenarios, that means a one-time treatment, as opposed to drugs that take months or years before progress begins to show.
Biotech startups are racing to get federal approvals to put gene therapies on the market. Philadephia-based Spark Therapeutics is working on treatment that would address inherited disorders that lead to blindness, while Bluebird Bio, based in Cambridge, Massachusetts, is also eyeing the therapy.
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But it's been a rough few weeks for gene therapy. Bluebird's stock tanked last year after it tested gene therapies for sickle-cell disease and beta thalassemia, with mixed results, while Maryland biotech company GenVec's stock plummeted when enrollment of new patients was halted in an early-stage trial testing its gene therapy for heating loss, due to an independent monitoring board's recommendation of a safety check.
Then there's pricing. One-time gene therapies are expected to cost a bundle, stoking fears about a healthcare system already struggling with rising drug prices.
Still, Pfizer, Novartis, Shire and other companies have recently upped their investments in gene therapy. Investors and gene therapy developers at the conference said they betting that the tricky technology will be worth it in the end.
Twitter: @JELagasse