Fresh from GSK deal, Orchard raises $150M for multifront late-phase gene therapy push

Orchard Therapeutics has raised $150 million (€132 million) to advance three gene therapies toward approval. The series C round continues a transformative year for Orchard defined by the acquisition of GlaxoSmithKline’s portfolio of approved and experimental rare disease gene therapies.

GSK sold its portfolio of gene therapies, including the approved but little-used Strimvelis, to Orchard in return for a 19.9% stake in the biotech, plus milestone and royalty commitments. The transaction turned Orchard into a commercial-stage gene therapy specialist with a clutch of late-phase prospects in its pipeline. And, by extension, redefined its near-term financial requirements.  

Four months after disclosing the deal, Orchard has raised the money needed to support its expanded pipeline. The $150 million round will fund pivotal trials of two gene therapies transferred from GSK and one Orchard had on its books prior to the transformative transaction.

The pivotal ex-GSK programs are treatments for metachromatic leukodystrophy (MLD), OTL-200, and Wiskott–Aldrich syndrome (WAS), OTL-103. OTL-200, an autologous ex vivo lentiviral gene therapy, is designed to treat MLD by transferring the gene that is mutated in patients with the life-threatening metabolic disorder. OTL-103 uses a similar approach to deliver the gene responsible for WAS.

Orchard’s third program, OTL-101, predates the GSK deal but is nonetheless linked to the Big Pharma. Like Strimvelis, OTL-101 is designed to treat the rare, life-threatening disease adenosine deaminase severe combined immunodeficiency. Orchard emerged in 2016 with employees who worked on GSK’s product and plans to improve on it by using a lentivirus, not the gamma retrovirus that underpins Strimvelis.

The presence of three late-phase programs in Orchard's pipeline—plus three more that are around clinical proof of concept—means the company has a shot at becoming an early leader in the nascent gene therapy sector. That potential has proven to be a potent proposition to investors. 

Deerfield Management led the series C with "significant" support from RA Capital Management, Venrock, Foresite Capital, Perceptive Advisors and other new investors. Temasek and Baillie Gifford, the co-leads of Orchard’s $110 million series B, and other existing investors chipped in cash, too.

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