Orchard Therapeutics raises $150m in Series C funding to advance gene therapies for rare diseases

Orchard Therapeutics, a leader in gene therapy specializing in rare diseases, has successfully secured $150 million through a Series C funding round. This substantial capital injection will support the advancement and commercialization of its innovative gene therapies. The funding round was led by Deerfield Management, with significant contributions from other top-tier investment firms such as RA Capital Management, Perceptive Advisors, Venrock, and Foresite Capital.

The fresh influx of capital underscores the industry’s confidence in Orchard Therapeutics‘ potential to address unmet needs in rare diseases through gene therapy. Existing investors including Temasek, Baillie Gifford, RTW Investments, Agent Capital, LP, and Cowen Healthcare Investments also participated, reaffirming their ongoing commitment to the company’s growth and development strategy.

Elise Wang, Principal at Deerfield Management, praised the company’s progress: “Orchard has made an impressive transition from a start-up company to an emerging leader in gene therapy for rare diseases by building a comprehensive, industry-leading portfolio of ex vivo gene therapies and assembling a highly experienced team.”

With this new funding, Orchard Therapeutics plans to accelerate its three most advanced clinical programs—OTL-101 for adenosine deaminase severe combined immunodeficiency (ADA-SCID), OTL-200 for metachromatic leukodystrophy (MLD), and OTL-103 for Wiskott–Aldrich syndrome (WAS). These programs are poised for registration and eventual commercialization. Additionally, the funds will support ongoing clinical and preclinical development across Orchard’s robust pipeline of rare disease gene therapies.

Mark Rothera, President and CEO of Orchard Therapeutics, expressed enthusiasm for the support received: “We are thrilled to have such strong support from both new and existing investors in this financing round. The quality of this investor syndicate is a testament to the confidence we have built among our stakeholders, based on the substantial progress of Orchard’s clinical and preclinical programs since our Series B round last year.”

Frank Thomas, CFO and Chief Business Officer of Orchard Therapeutics, highlighted the strategic importance of the Series C funds: “This financing provides Orchard with additional capital to rapidly progress our most advanced clinical programs to commercialization. We are advancing our pipeline of potentially transformative gene therapies in primary immune deficiencies and neurometabolic disorders to reach patients as quickly as possible.”

Since receiving a $110 million Series B funding and a $19 million grant from the California Institute of Regenerative Medicine (CIRM), Orchard has made significant strides in the gene therapy space. The company’s Strimvelis, an autologous ex vivo gene therapy for ADA-SCID, was approved by the European Medical Agency in 2016, marking one of the first regulatory successes for genetic therapies in the rare disease sector.