Orchard Therapeutics Scoops Up $110M in Funding to Advance “Bubble Boy Syndrome” Gene Therapy

Amneal and Impax to Marry, Creating the 5th Larges

Amneal and Impax to Marry, Creating the 5th Larges

Baillie Gifford and ORI Capital co-led the financing with significant new investments from Temasek and Cowen Healthcare Investments, alongside Juda Capital, Pavilion Capital, RTW Investments, Agent Capital, 4BIO Capital and existing investors F-Prime Capital and UCL Technology Fund.

Orchard Therapeutics closed on an oversubscribed Series B financing, raising $110 million. The round was co-led by Baillie Gifford and ORI Capital, with new investments from Temasek and Cowen Healthcare Investments. Juda Capital, Pavilion Capital, RTW Investments, Agent Capital, 4BIO Capital joined existing investors F-Prime Capital and UCL Technology Fund.

Orchard Therapeutics is a gene therapy company focused on rare diseases. Its technical operations are in Foster City, California. On Nov. 30, Orchard opened a second facility in the San Francisco Bay Area, the new site located at 1360 O’Brien Drive in Menlo Park, California. The addition quadrupled its laboratory space.

“Since the start of its technical operations in April 2016, Orchard has made significant progress in building a world-class team of experts for the development, manufacture and delivery of our ex-vivo gene therapy product pipeline,” said Stewart Craig, Orchard’s chief manufacturing officer, in a statement. “Opening of the new Menlo Park facility represents the next step of a phased plan to establish a fully integrated infrastructure for the company’s Technical Operations. Even as we open the new Menlo Park site, we are already evaluating sites for a further custom-built facility in the Bay area to provide another threefold increase in our overall capacity with additional operational capabilities by 2019.”

Orchard’s lead product is OTL-101, a gene therapy to treat adenosine deaminase severe combined immunodeficiency (ADA-SCID). The company expects to progress to a Biological License Application with the U.S. Food and Drug Administration (FDA) in 2018. The program has received a $19 million award from the California Institute for Regenerative Medicine (CIRM). The FDA granted OTL-101 Breakthrough Therapy Designation. Only about 15 patients in Europe are affected by ADA-SCID.

ADA-SCID has been dubbed “bubble boy syndrome,” named after David Vetter, a Texas boy from Texas who managed, for a while, to live outside in a plastic bubble. Born in 1971, he died at the age of 12. The disease causes severe immunodeficiency.

Funds raised from the Series B financing will be used to progress the company’s pipeline, including the global launch of OTL-101. It will also be used to expand its manufacturing infrastructure and business development operations.

As part of the financing, Simone Song, a senior partner at ORI Capital, joins Orchard’s board of directors. “Orchard has quickly established itself as a force in gene therapy,” Song said in a statement. “We are delighted to co-lead this financing and provide our support to deliver multiple life-saving medicines in this pipeline to patients with severe genetic diseases.”

In addition to OTL-101, the company’s pipeline includes clinical and preclinical gene therapies. They include a therapy for X-linked chronic granulomatous diseases (X-CGD) and metabolic diseases, such as MPS-IIIA and MPS-IIIB (Sanfilippo syndrome type A and type B).”

“The financing further empowers Orchard to deliver on our vision to be a leading, global, fully-integrated company that transforms the lives of patients through innovative gene therapy,” said Mark Rothera, Orchard’s chief executive officer, in a statement. “In collaboration with our stakeholders, we are committed to translating our outstanding clinical data into therapies for patients with the greatest need, as quickly as possible. This financing makes meeting this challenge possible.”

The company raised $33 million in a Series A round in May 2016.

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