A 2016 Fierce 15 winner and ultrarare disease startup Orchard Therapeutics has taken on Mark Rothera as its new president and chief.
Rothera, a near 30-year biopharma vet, comes to Orchard from controversial biotech PTC Therapeutics, where he served as its chief commercial officer. He’s also done stints as global president of Aegerion Pharmaceuticals, as well as VP and GM of commercial ops at Shire Human Genetic Therapies for the EMEA region.
Orchard is focused on ex vivo autologous hematopoietic stem cell gene therapy designed to restore normal gene function in primary immune deficiencies, metabolic diseases and hematological disorders.
This tech uses a sample of the patient’s own stem cells, which are modified with a functioning copy of the missing or faulty gene before being transplanted back into the patient’s body. Using the patient’s own cells (autologous) removes the need to search for a matching stem cell donor, which can take months or even years.
The small biotech’s early candidate is an ex vivo autologous lentiviral stem cell gene therapy for severe combined immunodeficiency (SCID), also known as “bubble boy syndrome," as children with the disorder are so susceptible to infection they have to be constantly protected and must live in a sterile environment.
Orchard is also exploring the effects of ex vivo autologous lentiviral stem cell gene therapy in patients with mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo disease type A).
Only about 15 patients in Europe have ADA-SCID, but last year the EU approved the first gene therapy for the disease, GlaxoSmithKline’s Strimvelis (autologous CD34+ cells transduced to express ADA). Patients had to travel to Ospedale San Raffaele in Milan, Italy, as this was the only place where the drug can be manufactured and given to a patient.
But things have not gone quite as planned, and GSK just a few weeks back said, after conducting a review of its rare disease meds, that it is “considering options for future ownership of these assets.”
These assets include Strimvelis, which costs $665,000 per treatment, as well as two other programs in development. The drug has simply not been the commercial success the company hoped for: So far, just two patients have received treatment with Strimvelis, and two more are lined up to receive the gene therapy later this year.
Orchard believes its lentiviral treatment is better than what GSK is offering as the Big Pharma’s tech is based on a gamma retrovirus, which is seen as older technology, and Orchard sees this as being superseded by lentivirus. The biotech also says that its med is potentially a safer therapy, and it hopes to have an easier method of manufacturing and getting to patients.
Orchard has been beefing up its staff over the past year, with GSK/Strimvelis vets Andrea Spezzi and Nicolas Koebel taking on new positions at the biotech: Spezzi as CMO and Koebel as SVP of business operations.
Koebel was at GSK for 10 years, until 2015. In 2011, he was appointed global commercial lead to GSK's rare diseases unit, to oversee the commercialization of GSK's program of autologous ex vivo gene therapies.
Spezzi, meanwhile, served as VP and medicine development leader at GSK's Rare Diseases Unit, after working for Takeda as its global medical director for R&D.
Orchard’s directors Ben Auspitz and Alex Pasteur, of F-Prime Capital, said of Rothera’s appointment: “We are delighted to welcome Mark as CEO of Orchard. He has a patient-centred approach and is focused on delivering access to innovative therapies as early as possible. He joins Orchard at a key time, as we progress a mature clinical pipeline towards several major milestones. Mark brings proven commercialisation and leadership skills, and he will have a great impact on the company during our next phase of growth.”
Rothera added: “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 honoured to join such a talented management team and exceptional academic founders as CEO, and I look forward to leading Orchard in the years ahead.”