Business of regenerative medicine III: an expert panel discussion

Webinar now available on demand. Join our panelists to discuss real-world evidence and accelerating approval, obstacles and aids to translation, and looking into the future of manufacturing and quality control.

We’ve convened six industry leaders to discuss their pain points and the business landscape of advanced therapies. Bringing expertise from around the regenerative medicine ecosystem, Graça Almeida-Porada (Wake Forest Institute for Regenerative Medicine), John Duguid (Vericel Corporation), Niall Carolan (REGENXBIO Inc.), Kelley Rogers (National Institute of Standards and Technology), Evan Snyder (University of California-San Diego) and Martha Lundberg share their unique perspective and insight.

This webinar was recorded on 5 September 2019.

Topics discussed include:

• How will the FDA’s increased support of real-world evidence affect the regulation and approval of regenerative medicines?
• What are the obstacles of translating a gene therapy from academia to clinic?
• Where should investments be made to improve the supply chain in the future?
• Identifying critical-to-quality parameters: what’s working, and what challenges exist?
• How can you optimize your in-process risk mitigation strategy?

Who may this interest?

• Cell therapy developers and CMOs
• Researchers and companies in translational research
• GMP facilities and product vendors
• Professionals in cell manufacturing and process development

Date: Thursday 5 September 2019

Time: 07:00 [PDT] 10:00 [EDT] 15:00 [BST]

Chaired by

Graça Almeida-Porada
Director, Fetal Research and Therapy Program, Wake Forest Institute for Regenerative Medicine

Graça Almeida-Porada is a Professor of Regenerative Medicine and the Director of the Fetal Research and Therapy Program at Wake Forest Institute for Regenerative Medicine (NC, USA). Dr. Almeida-Porada’s research focuses on the development of cellular and gene delivery platforms to treat genetic and immune-mediated diseases. She is particularly interested in improving the outcome of stem cell transplantation and gene therapy in fetal and neonatal patients with genetic disorders, and in developing therapies for children with immune-mediated diseases.

She has been a member of several NIH study sections, she serves as an Editor, or on the Editorial Boards, of several scientific journals and is the co-editor-in-chief of Current Stem Cell Reports. She was inducted into Phi Beta Delta in 2006. She is the co-founder of the International Fetal Transplantation and Immunology Society. Dr. Almeida-Porada holds several patents and has authored more than 200 scientific works including papers, abstracts, and book chapters.

Speakers

John Duguid
Senior Director, Research and Development, Vericel Corporation

John Duguid is Senior Director, Research & Development at Vericel Corporation (MA, USA). Vericel Corporation acquired Genzyme’s Cell Therapy and Regenerative Medicine business (CTRM) in 2014. As a Principal Scientist, Mr. Duguid was responsible for developing, validating and transferring molecular biology assays for rapid microbiology and cell differentiation applications, managing complex projects to implement process changes, and using statistical process control tools to implement process analytical technology for cell therapy products. He has been with CTRM since 1995. Before taking on his current role in R&D, Mr. Duguid managed quality control cell therapy operations at Genzyme (MA, USA) for over 10 years, where he designed and implemented a comprehensive cGMP-compliant raw material program controlling 300-400 parts and participated in 16 vendor audits, directing 4 as lead auditor. He also represented QC during 10 FDA inspections and numerous audits from international regulatory authorities as a subject matter expert in material inspection and release, biopsy accessioning, endotoxin testing, mycoplasma testing, analytical methods, flow cytometry, laboratory failure investigations, assay validation, and data management.

Mr. Duguid received his Bachelor of Science degree in chemistry in 1986 from the University of Michigan (MI, USA) and taught analytical chemistry in 2000 at Northeastern University in Boston (MA, USA). Prior to joining Genzyme, he worked in analytical research at Abbott Laboratories’ Pharmaceutical Products Division (IL, USA) and then as a scientific consultant for Massachusetts biotechnology companies at Arthur D. Little (MA, USA). Involved with the pharmaceutical and biotechnology industries for over 20 years, Mr. Duguid has technical and management experience spanning all phases of the product lifecycle from early research and development through cGMP commercial operations.

Niall Carolan
Vice President, Global Program Management & Supply Chain, REGENXBIO Inc.

Niall Carolan has been at REGENXBIO, Inc. (MD, USA) for 3.5 years, where he is currently VP of Global Program Management & Supply Chain and previously was VP of Manufacturing & Supply Chain. Prior to REGNEXBIO, Inc., he was Head of Biopharmaceutical Technology at GlaxoSmithKline (MD, USA) leading a BDS manufacturing sciences team and a process development lab function for both GSK programs and as a CMO service provider. Prior to GSK, he was Director of Manufacturing Sciences at Human Genome Sciences (MD, USA), with earlier roles in technology transfer, project management and supply chain. Earlier in his career, he held roles in manufacturing management and global technology coordination at Novozymes, and in process engineering at Novo Nordisk (both Denmark). Dr. Carolan holds a PhD in Bioprocessing and a BEng in Chemical Engineering from the Queen’s University (Belfast, Northern Ireland).

Kelley Rogers
Technical Program Director for Biosciences at National Institute of Standards and Technology

Kelley Rogers is the Technical Program Manager for the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) at the National Institute of Standards and Technology (NIST) Office of Advanced Manufacturing (OAM) (MD, USA). Kelley received a PhD in Molecular Biophysics and Biochemistry from Yale University (CT, USA) and a BA in Chemistry from Hendrix College (AR, USA).

Prior joining AMNPO, Kelley was the Technical Program Advisor for Biosciences for NIST’s Material Measurement Laboratory. She was a post-doctoral fellow and staff fellow in the National Institute of Digestive, Diabetes, and Kidney Diseases (NIDDK), at the National Institutes of Health. After completing her fellowship at NIDDK, Kelley worked as a Principal Investigator with DuPont-Merck and DuPont Pharmaceuticals (MI, USA) identifying novel targets for antimicrobial drugs. She has extensive research expertise in bacterial protein synthesis.

In addition to her professional positions, Kelley has been an active advocate for educational issues in Maryland, and for organizations that promote science education for children.

As the Technical Program Manager, Kelley is responsible for technical quality and coordination with NIIMBL, a NIST-sponsored Manufacturing USA institute whose mission is to accelerate biopharmaceutical manufacturing innovation in the United States.

Evan Snyder
Professor, Sanford Burnham Prebys Institute & Dept. of Pediatrics, University of California-San Diego

Evan Y. Snyder earned his MD and PhD in neuroscience from the University of Pennsylvania (PA, USA) as a member of National Institutes of Health Medical Scientist Training Program (MSTP). He had also studied psychology and linguistics at the University of Oxford (Oxford, UK). He completed residencies in pediatrics and neurology as well as a clinical fellowship in Neonatal-Perinatal Medicine at Children’s Hospital-Boston, Harvard Medical School (MA, USA). He completed a research post-doctoral fellowship in the Department of Genetics at Harvard Medical School and became an attending physician in the Department of Pediatrics (Division of Newborn Medicine) and Department of Neurology at Children’s Hospital-Boston, Harvard Medical School. Dr. Snyder was also established his own independent research program at Boston Children’s Hospital, Harvard Medical School and was appointed first as an instructor in neurology (neonatology) and was promoted to assistant professor where he helped launch the stem cell and regenerative medicine fields.

In 2003, after 23 years at Harvard, Dr. Snyder was recruited to Southern California, to Sanford-Burnham Prebys Medical Discovery Institute and University of California-San Diego (CA, USA) (Department of Pediatrics) as a Full Professor and Director of the Program in Stem Cell and Regenerative Biology. He then inaugurated the Stem Cell Research Center (serving as its founding director) and initiated the Southern California Stem Cell Consortium (CA, USA). Dr. Snyder is a Fellow of the American Academy of Pediatrics (FAAP) and was elected to the Association of American Physicians. He was a founding member of the FDA/NIH Stem Cell Working Group to generate guidelines for human transplantation, and served nearly a decade on the FDA’s Biological Response Modifiers Advisory Committee with emphasis on stem cells, as well as serving two terms as Chair of the FDA’s Cell, Tissue, & Gene Therapy Advisory Committee.

Martha Lundberg

Martha Lundberg has over 25 years of scientific experience that focuses on tissue engineering, genomics and regenerative medicine for heart and vascular diseases.