Session Details

Commercial, Manufacturing and Clinical Aspects of a Multinational Rare Disease Gene Therapy Trial (Corporate Breakfast Tutorial Hosted By Cell Therapies Pty Ltd)
Thursday, May 30, 2019 07:30 AM - 08:30 AM
Room 220
Chair: Nathan Smith Project Portfolio Manager, Cell Therapies Pty Ltd, Australia

Speakers:

Geoff Mackay Chief Executive Officer, AvroBio, United States

Alexandra Rizzitelli, PhD Cell Therapy Specialist, Cell Therapies Pty Ltd, Australia

David Ritchie, MD, PhD Haematologist & Head of Allogeneic Stem Cell Transplantation, The Royal Melbourne Hospital, Australia

Conducting a multinational rare disease gene therapy clinical trial poses many challenges that must be overcome to ensure a successful clinical study. We will present a case study detailing the different aspects to the implementation of AVROBIO’s phase 1/2 clinical study for their AVR-RD-01 product for patients with Fabry disease in Australia.
Initially, AVROBIO, a leader in lentiviral-based gene therapies, will review their corporate goals, the technology platform, the product, and the disease. Cell Therapies, a contract manufacturer based in Melbourne that supplies the clinical product, will provide details on the technology transfer and product manufacturing. Finally, the clinical aspects and patient considerations of the Fabry disease
study will be outlined by The Royal Melbourne Hospital, who has been treating patients in Victoria for over 170 years.