Advancing treatments for lysosomal storage disorders

  • Bigger, Brian (Corresponding participant)
  • Robert Wynn (Participant)
  • Simon Jones (Participant)

Impact: Health and wellbeing, Economic


We developed new treatments for rare childhood lysosomal storage diseases (LSDs) and built commercial capacity in the sector. Our research led to licensing two drugs worldwide for LSDs: Sebelipase Alfa for lysosomal acid lipase deficiency (LAL-D) and Elosulfase Alfa for mucopolysaccharidosis (MPS) IVA (Morquio A syndrome), marketed by Alexion Pharmaceuticals and Biomarin Pharmaceuticals, respectively. As a result, >90 LSD patients in England and >1,000 worldwide have been treated. We were scientific co-founders of Orchard Therapeutics Plc, a company specialising in developing gene therapy treatments for rare diseases. Our research was instrumental in the company securing USD33,000,000 financing, allowing successful launch and growth from 33 to >250 employees across the UK and USA.
Impact date1 Aug 201331 Dec 2020
Category of impactHealth and wellbeing, Economic
Impact levelAdoption

Research Beacons, Institutes and Platforms

  • Lydia Becker Institute