With a po­ten­tial cure for lyso­so­mal stor­age dis­or­ders in hand, Avro­bio rais­es $60M to back gene ther­a­py pipeline

In gene ther­a­py, re­spons­es from just one or two pa­tients can have a huge in­flu­ence on an­a­lysts’ opin­ions. Just ask the ex­ec­u­tive team at blue­bird bio, which trum­pets each pa­tient’s progress with de­light — or cal­i­brates a new de­vel­op­ment strat­e­gy when smiles turn to frowns.

So it’s no won­der that Avro­bio is so proud of its very first pa­tient.

At a sci­en­tif­ic con­fer­ence in Japan last fall, re­searchers re­viewed the case of a pa­tient with Fab­ry dis­ease whose blood lev­els of the en­zyme a-Gal A were near ze­ro at the start of treat­ment. With­in 45 days of re­ceiv­ing their gene ther­a­py AVR-RD-01, the pa­tient’s plas­ma a-Gal A ac­tiv­i­ty in­creased in­to the nor­mal range, they said. And 6 months lat­er that hadn’t changed.

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