This startup might finally cure sickle cell disease—after a century of racist neglect:
loves rocket launches—and not just the ones that succeed. He makes his team watch all the SpaceX failures, too, when the unmanned rockets explode. “For the first many years, all the rockets crash and there's a lot of failure along the way, and then suddenly, they start working,” he says.
Beam, which was founded in 2017, went public in February and now has a market cap of $1.5 billion despite having no revenues and losing $95 million over the past year. What the company does have is 12 research programs for 10 different rare diseases, including beta thalassemia, another inherited blood disorder, and two types of blood cancer. None of the treatments have been cleared for clinical trials, but the company hopes to file a slew of applications in 2021.
Sickle cell was the first “molecular disease” discovered, revealing how a change in one single amino acid could disrupt blood and oxygen supply to the entire body. The first drug to treat sickle cell, hydroxyurea, wasn’t approved until 1998, even though the disease has been known in the medical literature since 1910, with three more drugs coming to market since 2017. The only cure is a bone marrow transplant, which is limited to a small percentage of patients who have a matching sibling donor.
“I certainly, in my wildest dreams, never imagined this kind of precise capability to edit the genome,” says Dr. Francis Collins of the National Institutes of Health. Beam is also trying another approach to curing the disease: Introducing a second mutation in a different location to override the production of sickle hemoglobin. It mimics a naturally occurring phenomenon in which a person has two sets of sickle hemoglobin genes, but doesn’t show signs of the disease. The reason? A different mutation in the fetal hemoglobin gene, which usually turns off in favor of adult hemoglobin as people age, but remains turned on and producing normal hemoglobin.
“Celgene, gave us $130 million dollars upfront to explore this area of biology and Agios was able to preserve commercial right, and many other important features, so it was a very creative deal,” says David Schenkein, the former CEO of Agios and a general partner at Mountain View, California-based venture firm GV , which invested in Beam.
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