Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel Prize-winning technology faces a basic math problem: It¡¯s not profitable to spend millions of dollars making a drug that may only treat one person.
The solution, according to gene editing pioneer David Liu and his colleagues, is to bring everyone involved together ¡ª including patients, hospitals, scientists, drug companies and manufacturers ¡ª to make each step of the process simpler, faster and cheaper. Rather than the current bespoke method where each step is tailored and price tags can top $3 million, a collaborative approach may improve efficiency and allow the science to live up to its potential.
That¡¯s the idea behind the Center for Genetic Surgery, a new nonprofit created by Liu and his fellow scientists at the Harvard University-affiliated Broad Institute. It¡¯s a radical departure from a health care system that keeps the players separate, each with their own bill in a manner that inflates costs. If the process is made more efficient, it could be a model for treating the long tail of rare diseases that the pharmaceutical industry traditionally ignores.
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