August 10, 2017
By Alex Keown, BioSpace.com Breaking News Staff
LONDON – Mark Rothera, the former chief commercial officer of New Jersey-based PTC Therapeutics , will now helm the startup Orchard Therapeutics, a clinical-stage gene therapy company.
Rothera will serve as president and chief executive officer of Orchard, the company announced this morning. As CEO, Rothera will bring 28 years of leadership experience to Orchard, including the four years he spent at PTC Therapeutics. It was during his tenure at PTC that he helped take the company from a privately-held operation to a publicly traded one during his first year at the company.
According to his LinkedIn profile, during his four years at PTC, he “developed the strategy and built the global commercial footprint for the launch of Translarna…” Translarna is the first treatment approved for patients with Duchenne Muscular Dystrophy due to a nonsense mutation (a devastating condition that affects young boys mainly). It is now commercially available in nearly 20 countries and is expected to reach patients in over 35 countries in 2016.
Orchard Therapeutics, which launched last year and has already established a U.S. foothold in Foster City, Calif., is developing a platform of ex-vivo autologous gene therapies in serious and life-threatening orphan diseases. The company has collaborative partnerships with University College London, Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH) and the University of Manchester.
Orchard’s lead candidate is ex-vivo autologous lentiviral stem cell gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID). Interim clinical data from this program show significant immune reconstitution and 100 percent survival in 32 patients treated at GOSH and UCLA as of March 2016. As of the end of September, Orchard said the number of patients treated increased to 39 patients.
“Now is an exciting time to be joining Orchard as the cell and gene therapy field is advancing successfully. Gene therapy can have a profound, life-changing effect on patients with severe inherited diseases. The clinical data we’ve seen to date with Orchard’s therapy for ADA-SCID is exceptional, and the company has assembled an impressive R&D pipeline to apply the approach more widely. I’m honored to join such a talented management team and exceptional academic founders as CEO, and I look forward to leading Orchard in the years ahead,” Rothera said in a statement.
Orchard’s drug candidate is expected to challenge Strimvelis, GlaxoSmithKline ’s gene therapy drug recently approved in Europe to treat ADA-SCID. 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 is estimated to occur in approximately 15 patients per year in Europe.
Prior to his time at PTC, Rothera served as global president of Aegerion Pharmaceuticals Inc. and general manager of commercial operations at Shire Human Genetic Therapies for Europe, Middle East and Africa.
Company directors Ben Auspitz and Alex Pasteur, of F-Prime Capital, touted Rothera’s abilities. In a joint statement, they said Rothera is joining Orchard at a key time as the company begins to look at several “major milestones” in its pipeline.
