Rare Disease/Orphan Drugs

The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. The conference is a place to meet and brainstorm
ways to advance orphan drug development and improve access to life-saving therapies.

Gene Therapy for Rare Disorders is dedicated to overcoming the unique drug development challenges encountered in transforming gene therapy concepts into reality for rare disease patients in need.As the gene therapy field strives to strike the balance between clinical efficacy and product safety in the context of mounting regulatory scrutiny, Gene Therapy for Rare Disorders provides in-depth insights from experts who know how to drive clinical progress, optimize manufacturing, and demonstrate the value of gene therapy products to regulators, payers, and patients.Across four days, this meeting will unite 500+ leading experts in Boston from innovative biotechs, large pharma, academia, and key service provider companies, delivering actionable insights and valuable networking opportunities to accelerate gene therapy progress.