Cell, Gene Therapy Success Rates Analyzed

A Tufts Medical Center for Biomedical System Design analysis finds that durable cell and gene therapy (CGT) programs for rare orphan conditions and CAR-T/TCR therapies for blood cancers have higher success once they enter the clinic compared to the combined pipeline of all therapies. A Center newsletter says the results are consistent for:

• likelihood of approval from Phase 1, for all therapeutic programs;
• programs in the same therapeutic areas; and
• every development phase except Phase 1 for CAR-T.

“These results illustrate the power of compounding incremental success across nearly every phase for all CGTs,” the analysis says. “They suggest that drug developers and investors should consider the full likelihood of approval when comparing their options, not only the immediate next clinical milestone. Durable cell and gene therapies are demonstrating transformative patient impact; their clinical success rates may also transform drug development and associated financial returns.”