Recently Approved Cell-Based Gene Therapies Demonstrate Innovation in Sickle Cell Disease Treatment
This white paper offers a comprehensive primer on the burden of SCD, the patient journey, and current and emerging treatment options.
Planning Real-World Studies to Support Cell and Gene Therapy Evidence Generation Needs
Overcome Cell and Gene Therapy (CGT) study challenges by using strategic real-world data and real-world evidence (RWD/RWE) planning to construct external controls, integrate registries for long-term follow-up (LTFU), and demonstrate product value to regulators and payers.
Navigating the Complexity of Gene Therapy
Learn how UBC reduced patient access hurdles with a tailored case management approach for a lifesaving SMA treatment.
