Abeona Therapeutics Evaluates a Scalable Suspension-based Platform for Production of Viral Vector for MPS-IIIA

Live30 webinars are thirty minute presentations designed to update you on the latest innovations, applications and data in a fast yet interactive format.

Viral vector-based gene therapies are bringing life changing treatments to many patients and families. One investigational viral vector-based gene therapy in development is for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA). This gene replacement therapy uses an AAV capsid to deliver a functional hSGSH gene.

If approved, a scalable upstream production process is needed to provide the necessary manufacturing capacity for this life-changing therapy to reach patients suffering from this disease. In this webinar, new insights into scaling up production of high-titer and high-quality viral vectors at clinical and commercial scales are presented.

Participants will learn:

Abeona Therapeutics’ approach and success with bringing life changing gene therapies to patients.
How the control of key process parameters enabled a scalable vector production process between the 50 L and 500 L scale using AllegroTM STR single-use bioreactors.
Next steps and considerations for scaling further using the Allegro 2000 L STR bioreactor.

Phillip B. Maples, PhD

Vice President, Process Development, Abeona Therapeutics

Dr. Maples has more than 35 years’ experience in international biotechnology, focused on cell and gene therapies, including twelve years at Baxter Healthcare prior to embarking on several business initiatives, exploring clinical and commercial applications of pioneering cell and gene therapies for inherited diseases and cancers. He has worn “many hats” in his career, providing leadership in product development, manufacturing, quality assurance, technical marketing support, regulatory and clinical affairs, but his central focus has been product/process development.

Dr. Maples has teamed to design, construct and or operate more than ten cell and gene therapy processing centers (ex. NIH), gene/cell therapy clinics and GMP manufacturing facilities for advanced biologics. He has held 2 CRADAs with NIH. He has provided professional guidance to universities, private foundations, government bodies, professional societies, public and private companies. Dr. Maples has been on the editorial board of BioProcessing Journal for eighteen years. He has 47 publications, over 170 abstracts and presentations and 17 issued US patents.

Anne MacIntyre

Senior Scientist, R&D Bioprocessing, Pall Corporation

Anne MacIntyre is a Senior Scientist in the Upstream R&D BioProcessing group at Pall Corporation in Westborough, MA. Anne studied Biochemistry at Northeastern University and has over 15 years of experience in mammalian upstream process and product development. Since joining Pall in 2019, Anne has worked primarily on developing gene therapy applications and technologies in the iCELLis™ and Allegro™ STR bioreactors platforms.