Riding a wave of scientific and investor enthusiasm over gene therapy, a biotech startup hopes to convince Wall Street of its unicorn status when it goes public next week. But reaching that goal will require succeeding where pharma failed and building a business on ultra-rare genetic diseases.
Headquartered in the U.K., Orchard Therapeutics is working on one-time treatments for diseases that each affect fewer than 500 people around the world. The bulk of the company’s pipeline comes from GlaxoSmithKline, which abandoned the field earlier this year after failing to find much of a market for its first approved gene therapy.
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