The London-based company's lead drug to treat adenosine deaminase
severe combined immunodeficiency (ADA-SCID), OTL-101, aims to fix
the disease-causing gene by infusing patients' own stem cells in
their bodies.
The drug is in the late-stage of development, with data expected in
the second half of 2018.
Orchard is planning to file a marketing application with the U.S.
health regulator before the end of next year, Chief Executive Mark
Rothera told Reuters, adding that he expects the drug to be launched
in the United States and Europe in 2019.
After several high profile failures in the late 1990s and early
2000s, gene therapy is gaining momentum with the FDA recently
updating guidance to help speed up development of such treatments.
On Tuesday, the health agency approved Spark Therapeutics Inc's
treatment for a rare form of blindness, marking the first time the
agency has approved a gene therapy for an inherited disease.
ADA-SCID is an inherited disorder that damages the immune system's
ability to fight disease-causing bacteria, viruses and fungi.
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Orchard said the current round of funding was led by asset manager
Baillie Gifford and venture capital fund ORI Capital. Investment
firms Temasek and Cowen Healthcare were among the other big
investors.
The company has also appointed ORI Capital's senior partner, Simone
Song, to its board.
Rothera said the company would revisit its funding needs next year.
"This could include going public," he said, adding that the company
is yet to decide on this.
(Reporting by Divya Grover in Bengaluru; Editing by Arun Koyyur)
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