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Join 1,000+ attendees from big pharma, biotechs, payers, patient groups, academics & government.

The event will feature 7 tracks of content including:

  • Gene Track
  • Global Market Access
  • Regulatory, Clinical Development / Pricing & Reimbursement
  • Commercial
  • Rare Disease Advocacy World
  • Pitch & Partner
  • Patient groups Seminar

Leading the discussion will be 250+ speakers including:

  • Peter Saltonstall, CEO, NORD

  • Yann Le Cam, Chief Executive Officer, EURORDIS

  • Preston Campbell, President & Chief Executive Officer, Cystic Fibrosis Foundation

  • Katherine High, Co-founder, President and Head of Research and Development, Spark Therapeutics

  • André Choulika, Chairman & Chief Executive Officer, Cellectis

  • Sandy Macrae, President and Chief Executive Officer, Sangamo Therapeutics

  • Hans Bishop, President & CEO, Juno Therapeutics

  • Mark Rothera, Chief Executive Officer, Orchard Therapeutics

  • Ken Mills, Chief Executive Officer, REGENXBIO

  • Karen Aiach, Founder, Chief Executive Officer, Lysogene

  • Mariana Fagnilli, Head of Global Patient Advocacy, bluebird bio

  • Jacqueline Corrigan-Curay, Director, Office of Medical Policy, Center for Drug Evaluation and Research, FDA

  • Chris Austin, Director, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

  • Kenneth Hobby, President, Cure SMA

  • Durhane Wong-Rieger, President & CEO, Canadian Organization for Rare Disorders

  • Steven Kamisky, Chief Scientific Officer, International Rett Syndrome Foundation

  • Pat Furlong, Founding President and CEO, Parent Project Muscular Dystrophy (PPMD)

  • Carmencita D Padilla, Founding Chairman, Philippine Society for Orphan Disorders

  • Douglas Ingram, Chief Executive Officer, Sarepta Therapeutics

  • Harsha Rajasimha, Co-Founder, Organization for Rare Diseases, India

  • Kevin Huang, President, Chinese Organization for Rare Disorders

  • Yukiko Nishimura, President, ASrid Japan

  • Alastair Kent, Ambassador, Genetic Alliance UK

For the full conference agenda & list of speakers, download the brochure >>