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Feature Story

Cover Story: Strategy: Rules of competition

How companies can compete with gene therapies that provide a functional cure

By Stephen Hansen, Associate Editor

Competing in gene therapy is unlikely to follow the same rules as competing in other therapeutic categories where — even in Orphan diseases — a better product can steal market share. The reason is that if the first gene therapies to market deliver on the promise of a functional cure, there may not be any patients left to treat.

Investors have poured at least $3 billion into 20 companies developing gene therapies for Orphan indications that have one or more competitors working on the same gene — including $1 billion to bluebird bio Inc. alone. Most of these companies have touted potential benefits of their products over more advanced competitors targeting the same genes.

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