Gene Genies: Inside The Revolutionary Biotech That Can Edit DNA Inside Living Humans

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Gene Genies: Inside The Revolutionary Biotech That Can Edit DNA Inside Living Humans
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John Leonard built Intellia Therapeutics with Jennifer Doudna, the Nobel Prize-winning scientist who pioneered gene editing technology. Intellia has figured out how to alter disease-causing genes inside patients, but before any breakthrough treatments come, it must cure itself of financial ills.

John Leonard built Intellia Therapeutics with Jennifer Doudna, the Nobel Prize-winning scientist who pioneered gene editing technology. The company has figured out how to alter disease-causing genes inside patients, but before any breakthrough treatments come, it must first cure itself of its legal and financial ills.ohn Leonard, CEO of Intellia Therapeutics, has just finished an hour-long meeting in the company's Boston headquarters, which is a five-minute jog from the MIT campus.

It sounds pretty grim, and Intellia’s stock has taken a beating. Since the start of the year, Intellia shares are 62% down compared to 23% for the Nasdaq and 24% for Nasdaq’s biotech index. Still Intellia has at least one ace in the hole: The man who runs the company is no stranger to these challenges. Leonard, a medical doctor by training, has a track record which few others can point to in this industry.

The market for treating this disease was $585 million in 2019. But more people probably have the disease and are not being properly diagnosed. Better diagnoses could lead this to become a $14.1 billion market within 7 years, according to a report by London-based consultancy GlobalData. There are currently three FDA approved drugs which slow the progress of the disease, but none of them are permanent cures and patients often end up still needing liver transplants.

“Fortunately, this ruling does not impact the development of CRISPR in anyway,” says Doudna. “Investors are continuing to put money into the space,” she adds.

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