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be able to get fast, objective answers about whether their compounds do what they are supposed to do in animals and people, at the molecular level. And as the company moves on into clinical trials designed to deliver proof worthy of FDA approvals, Global Blood Therapeutics will have no trouble designing experiments to show clear correlations between a drug that performs a certain molecular task, which translates into something meaningful to patients—like being able to climb stairs, live an active life, or live a longer life.
“The way we treat patients, we like to see direct molecular effects on a patient,” Goldsmith says. “We can measure things frequently this way. It will facilitate preclinical studies, and clinical trials. Eventually we should be able to adjust and monitor therapy as needed. With sickle-cell disease, it offers a compelling biological target, a compelling disease with an unmet need, and a compelling clinical/regulatory strategy.”
Work on this strategy has been going on for almost two years within Third Rock, essentially since the West Coast office was formed in 2010, Goldsmith says. He stressed that Global Blood has its sights on a portfolio of genetic blood disorders, and that sickle-cell is just one example. The opportunity to bring the technologies together to produce multiple products is what helped this company earn its chance at successs. “This company has a lot of potential to have great impact, it’s the kind of company Third Rock likes to build,” Goldsmith says.