Gene, Cell, And RNA Therapy Landscape Continues To Thrive

As 2024 concludes, molecular medicine continues its steady 6-7% annual growth, while diversifying its therapeutic landscape. Notably, Q4 brought approvals across gene, cell, and RNA therapies: Aucatzyl for acute lymphocytic leukemia, RegeneCyte for blood disorders, and Tryngolza for lipoprotein lipase deficiency. The surge in pre-registration gene therapy programs—up 83% from Q3—signals a maturing field alongside strong early-stage innovation.

Therapeutic applications are broadening, with RNA therapy trial initiations rising 53%, 82% of which target non-oncology indications. Cell therapy trials also show diversification, and the continued expansion of these advanced therapies, combined with strong funding and clinical progress, marks a transformative phase in molecular medicine—one poised to redefine treatment across a growing spectrum of diseases.