Gene therapy has generated exceptional excitement and interest over
its potential for over three decades, but actual clinical progress has
been painfully slow and frustrating. Bluebird Bio (BLUE)
(and I am choosing to capitalize the name, even though it is officially
lower case) may be on its way to transforming at least part of the
dreams and hopes of gene therapy into reality.
Bluebird has
developed a platform that management believes will allow the company to
deliver therapies via lentivirus vectors to treat single-gene
rare/orphan disorders. The company's lead compound addresses a small
market, but the second could be a potential billion-dollar therapy. The
risks here are definitely elevated, but Bluebird may be in the early
days of establishing a leadership position in a major therapeutic class.
Read more here:
Bluebird Bio May Turn Dreams Into Reality
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