Gene Therapies: Building Facilities, Commercializing Product And Industrializing The Footprint

Situation:

• In 2015, client was a clinical-stage company focused on bringing their gene therapy from the lab into a clinical setting for patients and families devastated by rare neurological genetic diseases. Today, client is a leading, global gene therapy company with one of the first-ever commercialized, FDA, EMA and other regulatory agency-approved gene therapies
• Client was looking to pioneer the industrialization of gene therapies and build a cutting-edge manufacturing facility using modular cleanrooms, emerging bioreactors and single-use technologies for the clinical and commercial manufacture of a life-saving gene therapy for a rare neurological disease. At the time, this disease was the leading cause of infant death from genetic disorders
• Client had a team of less than ten employees with product being manufactured at an academic hospital. Client had no further external or internal manufacturing capability, no facility selected, no facility design, no technical operations infrastructure, no long-term plan or execution strategy and an accelerated timeline of fifteen months from board approval to facility startup

Task: Provide a manufacturing strategy and build a first-of-its-kind gene therapy facility using cutting-edge technologies within 15 months.