Orchard Therapeutics has positioned itself to join the commercial-stage gene therapy club with a $110 million series B round. The investment gives Orchard the financial firepower to take its gene therapy against adenosine deaminase severe combined immunodeficiency (ADA-SCID) through the final stage of clinical development and onto the market.
Baillie Gifford and ORI Capital co-led the round. Singaporean wealth fund Temasek and Cowen Healthcare Investments also made significant new investments. And a clutch of other existing and new financiers including Juda Capital, F-Prime Capital and UCL Technology Fund also chipped into the series B.
The closing of the round continues Orchard’s rapid rise. Orchard surfaced in May 2016, at which time it had $33 million in venture funding, leaders who worked on GlaxoSmithKline’s ADA-SCID gene therapy Strimvelis and a clinical-phase rival to the Big Pharma’s product.
Now, Orchard’s treatment for ADA-SCID—the immunodeficiency known colloquially as bubble boy syndrome—is powering through late-phase development. The series B gives Orchard the money to wrap up the study and, if the level of immune reconstitution seen in early development is achieved again, bring it to market.
GSK already has approval to sell its ADA-SCID gene therapy in Europe but has achieved minimal commercial traction, only landing its first commercial use a year after approval. The introduction of Strimvelis was hampered by the need for patients to go to Milan for treatment. Orchard, having seen its rival’s struggles, is planning to make its therapy available from multiple sites. Some of the cash is earmarked for adding manufacturing capacity.
The biotech also sees its use of a lentivirus, not a gamma retrovirus, as giving it the edge in terms of safety, although GSK’s clinical trial program was free from the cases of leukemia that blighted earlier gene therapies based on the technology.
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With ADA-SCID affecting about 15 patients a year in Europe, Orchard will need to look beyond that indication and its home continent. The startup has operations in California and once held aspirations to become the first company to win approval for a gene therapy in the U.S. Getting the ADA-SCID gene therapy, OTL-101, past the FDA would open up a market free from even GSK’s lackluster competition.
Orchard has other programs coming down the pipeline, too. The earlier-stage pipeline features gene therapies aimed at immune deficiencies and metabolic diseases, including X-linked chronic granulomatous disease and Sanfilippo syndrome type A and type B.
The potential of those prospects, and possibly the broader maturation of the gene therapy field, has enabled Orchard to pull off a series B that exceeded expectations.
“The size of this financing greatly exceeds the initial goal. This speaks to the quality of Orchard’s pipeline and to the expertise of the team. We are delighted that a top-tier investor syndicate with international reach has supported Orchard,” Alex Pasteur, a partner at F-Prime Capital, said in a statement.