Excitement over sickle cell now shifts to long slog of delivering treatments, in pivotal year for bluebird and gene therapy
SAN DIEGO — It is likely to take several months before the first new sickle cell disease gene therapies actually reach patients following their landmark approvals last week, according to the companies behind the therapies and the doctors who have been briefed on their rollout.
As exciting as the new treatments are — including the first-ever therapy that uses gene editing — the actual process of consenting patients, making the treatments and delivering them is a complicated slog. And while there are an estimated 20,000 sickle cell patients living in the US who are eligible, only a handful of centers that treat the disease are capable of giving the therapies.
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