Cell & Gene Therapies for Rare Diseases: Navigating the Pathway to Market
On demand

Cell & Gene Therapies for Rare Diseases: Navigating the Pathway to Market

Tuesday 08:00 PST / 11:00 EST / 16:00 GMT / 17:00 CET
Cell & Gene Therapies for Rare Diseases: Navigating the Pathway to Market

In Association with Phacilitate’s Cell and Gene Therapy World Forum

The promising early clinical data from gene therapies for rare diseases has seen a resurgence in investment and optimism in the field. This On Demand Webinar provides an opportunity to hear from three leading experts at the forefront of developing these potentially curative therapies:

  • Dr Sven Kili, VP and Head of Cell & Gene Therapy Development, GSK, UK
  • Mr Geoff MacKay, CEO, AVROBIO, USA
  • Ms Sue Washer, CEO, AGTC, USA

They share their insights and experiences in navigating the commercialization path, including:

  • What factors are unique to rare disease that need to be considered in terms of demonstrating clinical efficacy and selecting clinical endpoints?
  • The different types of accelerated approval tracks available for rare and ultra rare diseases and what do you need to demonstrate in order to gain approval?
  • Reimbursement price points and how rare disease companies can establish a viable business strategy to support different payment models?
  • How feasible is it to drive down manufacturing costs at small scale for niche patient therapies?

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Sven Kili
Sven Kili
CEO at Antion Biosciences
Sven Kili provides specialist strategic consulting services to innovative Regenerative Medicine companies. His clients include small and medium sized companies from company formation through to clinical development and commercialisation. He was previously the Head of Development for the Cell and Gene Therapy division of GSK Rare Diseases where he led teams developing ex-vivo Gene Therapies for a variety of rare genetic disorders including Strimvelis®, the first ex-vivo gene therapy to be approved for children with ADA-SCID; Wiskott – Aldrich syndrome (WAS); Metachromatic Leukodystrophy (MLD) and Beta-Thalassemia. Prior to this, he was Senior Director, Cell Therapy and Regenerative Medicine for Sanofi (Genzyme) Biosurgery where he led the clinical development, approval and commercialisation activities of the first combined ATMP approval in the EU for MACI®. His team also prepared and submitted Advanced Therapy regulatory filings for Australia and the US, including health technology assessments and he was responsible for late stage developments for Carticel® and Epicel® in the US. Before joining Genzyme, Sven led the cell therapy activities and oversaw all UK & Irish regulatory functions and was the QPPV for pharmacovigilance for the Geistlich Pharma. Sven trained as an Orthopaedic surgeon in the UK and South Africa and since leaving full-time clinical practise has developed expertise Cell and Gene Therapy in clinical development, regulatory compliance, value creation, risk management and product safety, product launches and post-marketing activities. He sits on the board of CCRM in Canada; Xintela – a Swedish Stem Cell company and is the chair of the CGTAC as part of the UK BIA and the VP of the Standards Co-ordinating Body for Regenerative Medicine. Additionally, he still maintains his clinical skills in the UK NHS and serves as an ATLS Instructor in his spare time.
Geoff MacKay
Geoff MacKay
Geoff is an experienced CEO with proven success leading innovative businesses. While CEO of Organogenesis Inc., the company treated 1 million patients with living cell therapies, received the first FDA CBER allogeneic cell-therapy approval and achieved an unparalleled position within regenerative medicine. Geoff was founding CEO of eGenesis, applying CRISPR Cas-9 gene editing to xenotransplantation. Geoff spent 11 years at Novartis in senior leadership positions within the Global Transplantation & Immunology franchise. Past activities include Chairman of the Board of MassBio, Chairman of the Board of the Alliance of Regenerative Medicine, and Advisory Council to the Health Policy Commission for Massachusetts. Through his public and private enterprise work, Geoff is dedicated to applying innovation to reshape and improve the quality of healthcare.
Sue Washer
Sue Washer
President & Chief Executive Officer at Applied Genetic Technologies Cooperation
An experienced entrepreneur with a strong scientific and operational background, Sue brings a decade of experience in pharmaceutical management and research with Abbott Labs and Eli Lilly & Company and more than 16 years of senior management experience with entrepreneurial firms in Florida including three start-ups. At AGTC, Sue has successfully secured investments of more than $91 million from nationally recognized venture capitalists and granting agencies; negotiated and closed on a major collaboration with a top five Biotech company; lead the company to complete critical milestones; and recruited an experienced management team. Her experience as a group leader with Abbott’s Diagnostic Division helped hone her skills in bringing new products and technologies from the lab bench into the market place. Sue is the chair of Southeast BIO, is a member of the Executive Committee of BioFlorida and sits on the board of the Florida High Tech Corridor Council. She has a degree in biochemistry from Michigan State University and an MBA from the University of Florida where she was one of the first graduates from the Warrington College of Business Entrepreneurship program.