Mark Rothera

Aegerion Pharmaceuticals, Inc. operates as a biopharmaceutical company. It engages in the development and commercialization of therapies for patients with debilitating rare diseases. The company was founded by David I. Scheer, Gerald L. Wisler and William H. Lewis in 2005 and is headquartered in Cambridge, MA.

Shire Human Genetic Therapies, Inc. develops medicines for the treatment of genetic diseases. The company was founded on July 7, 1988 and is headquartered in Lexington, MA.

Orchard Therapeutics (Europe) Ltd. researches on transformative gene therapies. Its services include stem cell technology, gene therapy, eradicating orphan diseases, and paediatric diseases. The company was founded by Andrea Spezzi and Nicolas Koebel on September 1, 2015 and is headquartered in London, the United Kingdom.

Orchard Therapeutics Plc is a biopharmaceutical company, which engages in discovering, acquiring, developing and commercializing gene therapies for patients with rare disorders. The company focuses on its autologous ex vivo gene therapy approach on three therapeutic rare disease franchise areas: primary immune deficiencies, neurometabolic disorders, and hemoglobinopathies. Its portfolio includes Strimvelis, a commercial-stage gamma retroviral-based product for the treatment of Adenosine deaminase deficiency (ADA-SCID). The company was founded by Nicolas Koebel and Andrea Spezzi in August 2018 and is headquartered in London, the United Kingdom.

ARM is a Washington, DC-based multi-stakeholder advocacy organization that promotes global initiatives necessary to facilitate access to life-giving advances in regenerative medicine. The organization promotes legislative, regulatory reimbursement, investment, technical, and other initiatives to accelerate the development of safe and effective regenerative medicine technologies. ARM also works to increase public understand- ing of the field and its potential to transform human healthcare. Prior to the formation of ARM in 2009, there was a need for more coordinated and cohesive advocacy representing the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today ARM has more than 140 members and is the leading global advocacy organization in this field.