26% of all cell and gene therapies in late-stage development in the US are being developed for oncology.
The FDA has backed the use of CAR-T cell therapies for cancer indications.
Novel therapeutic approaches indicate a focus on expanding cell and gene therapy for use in solid tumors.

Regenerative medicine advanced therapy (RMAT) designations are awarded by the FDA to cell and gene therapies that have the potential to treat unmet clinical needs and accelerate their route to the clinic. Approximately 26% of all cell and gene therapies in late-stage clinical development in the US are being developed in the oncology space, and highlighting those that have received an RMAT designation can provide insight into the future directions of this therapeutic modality in cancer therapy says GlobalData, a leading data and analytics company.

James Donagher, MRes, Oncology and Haematology Analyst at GlobalData, comments: “Over half of the cell and gene therapy oncology products, which have been awarded an RMAT, have been CAR-T cells. This highlights the FDA’s recognition of this innovative approach as one of the leading treatment modalities in the future treatment paradigm for cancer.”

Autologous CAR-T cells that target both established (CD-19) and novel antigens (CD-30 and BCMA) have been awarded an RMAT designation, in addition to the most recent designation being awarded to an allogenic CAR-T therapy, ALLO-715.

Adam Pearson, PhD, Senior Oncology Analyst at GlobalData, comments: “The RMAT designations awarded to CAR-T cell therapies indicate a potential shift in the development of the therapy, from targeting more established antigens, such as CD-19 to new targets in novel haematological malignancies. Finally, the most recent RMAT designation for an allogeneic CAR-T, ALLO-715, indicates a shift from autologous to allogeneic CAR-T therapy over time. Allogeneic, or ‘off the shelf’ alternatives, are ready for immediate use and can thus reduce the costs and delays associated with manufacturing, circumventing key obstacles associated with the clinical use of autologous CAR-T cells.”

CAR-T development is mainly restricted to treating haematological malignancies. Novel therapies, namely modified T-cell therapies, tumor infiltrating lymphocyte (TIL) therapy, and other forms of allogeneic cell therapies that can treat solid tumors have now been awarded RMAT designations.

Donagher continues: “Target indications for these novel therapies include solid tumors, which have been difficult to target for CAR-T therapy in part due to their heterogeneity and lack of targetable antigens. These novel approaches promise to bridge the gap and bring cell and gene therapy into the future treatment paradigm for solid tumors.”

Pearson concludes: “The RMAT designation recognizes some of the most innovative forms of cell and gene therapies in development for cancer and is designed to accelerate their route to clinic. The distribution of RMAT designations also hints at potential future treatment paradigms in oncology, namely the expanding use of autologous and allogeneic CAR-T cell therapy, and the use of novel approaches for targeting solid tumors.”