Gene Therapy for Rare Disorders US

March 20-23, 2023

Boston, MA

Visit Premier Research at Gene Therapy for Rare Disorders. As the gene therapy field continues to break records, there are still significant challenges to overcome relating to safety, efficacy, and accessibility. This conference will showcasing the top case studies and strategic learnings from the past year. Key questions will be answered on how best the field can overcome regulatory, clinical, manufacturing and pricing bottlenecks to progress gene therapies into and through the clinic.


Stream – CLINICAL – Designing & Implementing a Safe Clinical Trial

Rare “Plus”: The Operational Nuances to Consider When Designing Gene Therapy First-In-Human Studies

March 21st, 11:30 A.M.


Speaker: Dr. Ken Ndugga-Kabuye

  • Design and safety considerations
  • Site selection considerations
  • Effective patient enrollment and ongoing engagement