GRP Workshop

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Pre-Conference Workshop
Wednesday, May 3
08:00 - 17:15

The ISCT Global Regulatory Perspectives (GRP) represents the diverse activities and partnerships between international regulatory bodies, industry, clinicians and academia.

The GRP Workshop is an annual one-day program.  Unique to this workshop is the attendance of regulators from North America, Europe, Asia, South & Central America and Australia & New Zealand.  It is a highly interactive program including various hypothetical case studies throughout.

Regulators confirmed to date: FDA (USA), Paul-Ehrlich-Institut (Germany) and National Pharmaceutical Regulatory Agency (Malaysia) 

Supporters

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Program Information

Speaker: Scott Burger, MD, Advanced Cell & Gene Therapy

Moderator:David DiGiusto, PhD, Stanford School of Medicine

Speakers: 

  • Matthias Renner, PhD, Paul-Ehrlich-Institut
  • Michael Havart, PhD, FDA
  • Anne Dupraz-Poiseau, Orchard Therapeutics

This session is a review of different gene modification technologies and regulators’ perspectives on their use – the technologies to be reviewed may include TALENs, ZnFinger, CRISPR, and viral transduction.  Agency speakers will discuss their processes for assessing the use of these technologies in clinical studies.  An industry speaker will discuss their experience with gene modification technologies, use in their company’s product(s), and their regulatory choices in doing so.

Case study to follow

Moderator: Christopher Bravery, PhD, Consulting on Advanced Biologicals Ltd

Speaker:

  • Martha French, BS, Bellicum Pharmaceuticals

Aligning commercial business models with a global manufacturing strategy to ensure supply of genetically modified (GM) cell therapies in all target markets poses a number of issues.  In particular autologous GM-cell products require collection of donor material from patients and then the return of the GM-product back to the patient for administration; a challenge when both the starting material and the final product have limited stability.  This creates a logistical challenge for centralised manufacturing, leading many to explore other options of disseminated manufacture such as regional centres, small centre of excellence manufacturing and even near-patient/bedside manufacturing.  Behind this are systems designed to allow non-routine compassionate use manufacturing such as the EU ‘hospital exemption’.  Systems that allow small-scale manufacturing with limited regulatory oversight pose a commercial risk to those undertaking full development with a view to licensing.   This session aims to explore these issues and consider the commercial risks and opportunities; what will happen when successful products are approved which could be reverse engineered fairly simply by developers in less regulated regions or regions where exemption systems are available?  Will the flood gates open?

Case study to follow.

Moderator: Janet Macpherson, PhD, Royal Prince Alfred Hospital

Speakers:

  • Michael Havert, PhD, FDA
  • James Miskin, PhD, Oxford Biomedica

Viral vectors, particularly gamma retroviral and lentiviral vectors, are well suited to delivering genetic material to cells to offer therapeutic benefit. The wildtype viruses from which these vectors are derived, can replicate efficiently with a high recombination rate, hijacking the infected cells’ own replicative machinery. A conservative approach to prevent the unintended transmission of replication competent recombinant viruses (RCR/RCL), with potential to cause disease in patients has been adopted for over 25 years.

This session will highlight the current regulatory expectations for testing gene-modified patient cell products for RCR/RCL. The speakers will discuss benefits and limitations of the current technology in terms of the testing requirements, and provide some general guidance on permissible testing strategies, including post-release testing. Technology for the detection of RCR/RCL is evolving, to provide more rapid and more sensitive detection of the genetic material. Is this the future? What is the current status and what is the future of RCR testing?

Moderator: Dominic Wall, PhD, FFSc(RCPA), Peter MacCallum Cancer Centre

Speakers: 

  • Azizah Ab Ghani, National Pharmaceutical Regulatory Agency, Ministry of Health Malaysia
  • Peter Olagunju, MBA, ASQ-CQA, bluebird bio

In many cases the new cell-and gene treatments being developed in European and North American markets will have their greatest utility in treating patients in geographically distant markets, sometimes with local agencies who have less framework in place for cell-based treatments. How much reworking should be performed when transferring novel products developed in the West to new markets in the East, often where there are the most pressing unmet needs. Can EU and US agencies facilitate the flow of technologies to meet these demands? How can companies develop products where there is limited local prospect of a market approval, when the intended users will be in an entirely different region, or indeed when the intended recipients may be children whilst the product development has been assessed only in young adults? What lessons can be learnt to ensure that products developed for these emerging (and emerged) economies are efficiently and safely brought to these markets?

Case study to follow.

To view the full program, please refer to the Program Schedule.

Confirmed Speakers and Moderators

  • Christopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd., United Kingdom
  • Scott Burger, MD, Principal, Advanced Cell & Gene Therapy, USA
  • David DiGiusto, PhD, Executive Director, Stem Cells and Cellular Therapeutics Operations, Stanford Healthcare, United States
  • Martha French, BS, Vice President of Regulatory Affairs, Bellicum Pharmaceuticals, Inc.,United States
  • Azizah Ab Ghani, PhD, Senior Principal Assistant Director, Head of Biologics Section, National Pharmaceutical Regulatory Agency (NPRA), Ministry of Health Malaysia, Malaysia
  • Michael Havert, PhD, Gene Therapy Reviewer, FDA, United States
  • Janet MacPherson, PhD, Development Manager, Royal Prince Alfred Hospital, Australia
  • James Miskin, PhD, Chief Technical Officer, Oxford BioMedica, United Kingdom
  • Peter Olagunju, MBA, ASQ-CQA, Senior Director, Commercial CMC Operations, bluebird bio, United States
  • Anne Dupraz Poiseau, PhD, Chief Regulatory Officer, Orchard Therapeutics, Ltd, United Kingdom
  • Matthias Renner, PhD, Deputy Head Non-Viral Gene Transfer Medicinal Products, Paul-Ehrlich-Institut, Germany
  • Dominic Wall, PhD, FFSc(RCPA), Operations Director, Peter MacCallum Cancer Centre, Australia

Organizing Committee

Karen Nichols, Esq. – Co-Chair
VP, Regulatory and Quality, NantKwest, USA
Dominic Wall, PhD, FFSc(RCPA) – Co-Chair
Operations Director, Peter MacCallum Cancer Centre, Australia
Christopher Bravery, PhD, Director, Consulting on Advanced Biologicals, United Kingdom
Scott Burger, MD, Principal, Advanced Cell & Gene Therapy, USA
Karen Edward, BSc, MT(ASCP), USA
Srinivasan Kellathur, PhD, Head, Advanced Therapy Products Unit, Health Sciences Authority, Singapore
Aisha Khan, ABD, MSc, MBA, Executive Director of Laboratory Operations, Interdisciplinary Stem Cell Institute, USA
Janet Macpherson, PhD, Development Manager, Dept of Cell & Molecular Therapies, Royal Prince Alfred Hospital, Australia
Gustavo Moviglia, MD, PhD, Medical Director, University of Maimonides, Argentina
Paula Salmikangas, PhD, Chair, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Finland
ISCT 2017

ISCT 2017