Four months after acquiring GlaxoSmithKline’s rare disease gene therapy portfolio, U.K.-based Orchard Therapeutics secured $150 million in an oversubscribed Series C funding round. The funds will be used to advance three late-stage programs that it gained from the deal with GSK.
Four months after acquiring GlaxoSmithKline’s rare disease gene therapy portfolio, U.K.-based Orchard Therapeutics secured $150 million in an oversubscribed Series C funding round. The funds will be used to advance three late-stage programs that it gained from the deal with GSK.
Orchard said the $150 million it gained through the Series C will help drive development and potential commercialization of OTL-103, a gene therapy for Wiskott–Aldrich syndrome (WAS). The rare disease causes children to not produce the proper number of platelets, and the immune system fails to function as designed. WAS patients are at risk of having serious bleeds and life-threatening infections. It occurs in approximately one baby in 200,000 births and affects boys almost exclusively, according to Orchard’s website.
The other two programs that will be the primary beneficiaries from the funding are OTL-101 for treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID), and OTL-200 for metachromatic leukodystrophy (MLD).
If approved by regulatory agencies, Orchard’s OTL-101 would be marketed alongside another ADA-SCID treatment that is in the company’s portfolio. As part of the deal with GSK, Orchard gained Strimvelis, the first autologous ex vivo gene therapy for children with ADA-SCID. Strimvelis was approved by the European Medicines Agency (EMA) in 2016. Children born with ADA-SCID do not develop a healthy immune system so they cannot fight off everyday infections, which results in severe and life-threatening illness. Without prompt treatment, the disorder often proves fatal within the child’s first year of life. ADA-SCID occurs in less than 1 in 200,000 births. The U.S. Food and Drug Administration (FDA) granted OTL-101 Breakthrough Therapy Designation.
In addition to the three late-stage assets, Orchard said the financing will also be used to support further clinical and preclinical development of its rare disease gene therapy pipeline. Other assets that Orchard gained from the GSK deal include one clinical program for beta thalassemia, as well as the licensing rights to three preclinical programs from Telethon/Ospedale San Raffaele upon completion of clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD) and globoid cell leukodystrophy.
When Orchard gained the rare disease gene therapy portfolio from GSK, it gave the larger pharma company a 19.9 percent equity stake, as well as a seat on the board of directors. Additionally, GSK will receive undisclosed royalties and commercial milestone payments related to the portfolio.
The Series C funding was led by Deerfield Management. Other significant new investors include RA Capital Management, Venrock, Foresite Capital, Perceptive Advisors, Cormorant Asset Management LP, ArrowMark Partners, Sphera Global Healthcare, Medison Ventures, Driehaus Capital Management and Ghost Tree Capital Group, LP. Existing investors in Orchard who participated in the Series C round are Temasek, Baillie Gifford, RTW Investments, LP, Cowen Healthcare Investments and Agent Capital.
Chief Executive Officer Mark Rothera said the Series C funding is a testament to the confidence the company has built among its stakeholders “based on the substantial progress of Orchard’s clinical and preclinical programs since our Series B round last year.” That Series B round scored Orchard $110 million in December.
