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Gene therapy: What you need to know

British drugmaker GlaxoSmithKline made headlines last year when it won approval for its gene therapy Strimvelis in Europe. But, due to a small patient population and high price tag, the drug has only been used once. So far, despite higher levels of safety and efficacy than previous iterations, the new wave of gene therapies still face commercial hurdles.

Spark Therapeutics looks set to be the next company to take on this challenge in the U.S. The biotech is currently awaiting approval of its treatment for a rare genetic form of blindness — a potential one-time cure. Yet pricing will be the most closely watched aspect of this therapy, likely serving as an early barometer of what might be sustainable for a pipeline of treatments still in development.

While gene therapy offers the promise of cures and new ways of revolutionizing treatment of genetic diseases, society remains a long way from fully realizing those advances.

  1. A new wave of gene therapies ready to hit US shores

    After decades of setbacks, a slew of next-gen gene therapies are ready to hit the U.S. market, prompting questions about manufacturing and pricing. Read More >>

  2. 5 Trends affecting gene therapy development

    A pricing conundrum and ethical decisions are clouding an already hazy path to market for many gene therapy drugs and providers. Read More >>

  3. Alternative pricing models a fantasy for gene therapies?

    With an approval of Spark Therapeutics' gene therapy for a rare eye disease rapidly approaching, new questions about pricing are being raised. Read More >>

  4. Gene therapy by the numbers

    In a field shaped by small patient populations and eye-popping cost considerations, understanding gene therapy's promise and challenges comes down, in part, to the numbers. Read More >>

  5. Do patients understand the risk of reshaping a genome?

    While many are optimistic about gene editing's ability to cure disease, it seems not enough realize the more dangerous aspects of treatment. Read More >>

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Filed Under: Drug Discovery Clinical Trials
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