About the Speaker
Márcia Sellos-Moura, PhD joined Orphan Technologies in January 2016 as Associate Vice President and Program Executive. She brings over 20 years of global R&D and program management experience, with line and cross-functional leadership, with emphasis in preclinical and clinical development, bioanalytical and translational sciences.
Previously, Márcia held a variety of positions of increasing responsibility at Shire and Transkaryotic Therapies and was an integral contributor to the approval of four products and to the development of greater than 20 candidates across their rare disease portfolios. She has authored multiple manuscripts and has been granted several patents throughout her academic and industry career. Márcia received her PhD in Biochemistry, Cell and Molecular Biology and her B.A. in Biochemistry and Chemistry from Cornell University, and a Graduate degree in Biopharmaceutical International Regulatory Affairs from Northeastern University.
Orphan Technologies is dedicated to developing novel therapies to dramatically improve the lives of patients suffering from rare disorders. OT-58, our lead drug development candidate, has been optimized as an enzyme replacement therapy for classical homocystinuria, a genetic disease characterized by debilitating cardiovascular, skeletal, neurologic, and ophthalmologic complications. OT-58 is designed to reduce homocysteine levels via a targeted mechanism of action and may have therapeutic applications in other diseases. For more information, please visit www.orphantechnologies.com
OT-58, Orphan Technologies’ lead drug candidate, is a modified recombinant enzyme replacement therapy in development as an enzyme replacement therapy for patients suffering from the rare disease classical homocystinuria. Classical homocystinuria is a genetic metabolic disease caused by a deficiency in the CBS enzyme leading to elevated levels of the amino acid homocysteine. OT-58 has consistently demonstrated significant reductions in homocysteine levels across multiple models of homocystinuria and has the potential to improve metabolic control, reduce or remove dietary restrictions, and positively impact clinical outcomes. OT-58 is anticipated to enter clinical evaluation in 2018 and has been granted Orphan Status by the US Food and Drug Administration and EMA. In addition, based on its mechanism of action, OT-58 has therapeutic potential in other diseases.