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April 2018
Boston, MA


Pre Conference Morning Workshop A
Monday April 24, 2017

09.00 - 12.00
Workshop A: Understanding the Regulatory Landscape and Expectations of the Gene Therapy Field
Workshop Leader: Gopalan Narayanan, VP, Disruptive Biologics, Voisin Consulting Life Sciences

As the gene therapy space advances and clinical investigations continue to produce promising results, uncertainty about the regulatory frameworks and procedures in place needs to be addressed. Attend this workshop to gain an in-depth understanding of the regulatory landscape at this pivotal time for the gene therapy field.

Attendees Will Learn About:

  • What the regulators are looking for in every stage of development – from preclinical investigations, through clinical development, manufacturability and reimbursement
  • The availability of regulatory incentives for the development of gene therapies in the rare disease space
  • In-depth case studies, guiding you through regulatory strategy and decision making throughout the development process

Gopalan Narayanan, VP, Disruptive Biologics, Voisin Consulting Life Sciences

Gopalan Narayanan

Dr Narayanan provides leadership in the area of complex and disruptive biologics such as Cell and Gene therapies, including guidance on product development and regulatory strategy and in the global effort of rationalising the development process through which such disruptive biologics can be brought to patients faster and more efficiently.

Narayanan was a senior regulator at the UK MHRA for nearly 12 years as an expert medical assessor, and 4 years as Manager of the Biologicals Unit. He was a member of the Committee for Advanced Therapies (EMA/CAT 2009 – 2013), Scientific Advice Working Party (CHMP/SAWP 2009 – 2013) representing  EMA/CAT, Biosimilar Working Party (EMA/BMWP) and Gene Therapy Working Party (CAT/GTWP).

Narayanan qualified as a physician, and has dual specialist postgraduate qualification in general internal medicine and pharmaceutical medicine. He is currently on the Scientific Editorial Board of Human Gene Therapy Clinical Development journal and is a Review Editor of Regulatory Science section of Frontiers in Medicine, and is based in the UK.

Pre Conference Morning Workshop C

09.00 - 12.00
Workshop C: Discussing Novel Technologies With the Potential to Impact the Gene Therapy Field
Workshop Leader: Casey Maguire, Assistant Professor, Harvard Medical School

Evidently, the use of AAV vectors has had a significant impact on the field, as demonstrated by their continued use in the pioneering clinical trials currently reaching maturity. However, these vectors still encounter significant challenges upon injection that impact on the efficacy and durability of transgene expression. Attend this workshop to get up to speed on the latest developments in vector technology.

Attendees will learn about:

  • The degree to which the current suite of AAV vectors are able to affect the expression levels of a transgene at a therapeutic level and comparison with new vectors in preclinical development
  • Next generation delivery technologies with improved tissue targeting and the ability to deliver larger genes to the target tissue
  • Novel technologies which can evade pre-existing antibodies as well as avoid immune response complications associated with re-dosing

Casey Maguire, Assistant Professor, Harvard Medical School

Casey Maguire

Pre Conference Afternoon Workshop B
Monday April 24, 2017

13.00 - 16.00
Workshop B: How will Gene Therapies be Reimbursed?
Workshop Leader: Edmund Pezalla, Consultant, EJ Pezalla Independent Consultant

Multiple frameworks and plans have been proposed in the US and across different geographies, to address the concerns of payers regarding the cost of gene therapies. Attend this workshop to investigate the current reimbursement landscape and the ways in which it will need to adapt in the future.

Attendees will learn about:

  • The complexities of pricing potentially curative therapeutics
  • Comparisons between proposed pricing and reimbursement models
  • Differences between pricing structures in different geographies worldwide

Edmund Pezalla, Consultant, EJ Pezalla Independent Consultant

Edmund Pezalla

Pre Conference Afternoon Workshop D

13.00 - 16.00
Workshop D: Enhancing the Outsourcing Process: How to Utilize Contract Manufacturing Services and the Broader Contract Infrastructure Effectively
Workshop Leader: Scott Burger, Principal, Advanced Cell & Gene Therapy

Gene therapies have made significant progress in the rare disease space, and as commercialization draws closer, particularly in indications like haemophilia which require high doses of therapeutic, the outsourcing and contract service environment becomes more essential than ever before.

Attendees will learn about:

  • The criteria you should be using to adequately assess potential partners to outsource projects to in the gene therapy space
  • The broader contract infrastructure – understand how to improve interactions with CMOs, CROs, contract testing firms and contract shipping and transportation companies
  • Key factors impacting the decision to develop in-house capabilities or outsource services to third parties

Scott Burger, Principal, Advanced Cell & Gene Therapy

Scott Burger